THRIVE Teaming Profiles
This page is designed to help facilitate connections between prospective proposers, which ARPA-H anticipates will be necessary to achieve the goals of the Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) program. Prospective performers are encouraged (but not required) to form teams with varied technical expertise to submit a proposal.
If either you or your organization are interested in teaming, please create a profile via the ARPA-H Solutions Portal linked below. Your details will then be added to this page, which is publicly available.
Please note that by publishing the teaming profiles list, ARPA-H is not endorsing, sponsoring, or otherwise evaluating the qualifications of the individuals or organizations included here. Submissions to the teaming profiles list are reviewed and updated periodically.
THRIVE Teaming Profiles
To narrow the results in the Teaming Profiles List, please use the input below to filter results based on your search term. The list will filter as you type.
| Anna Blakney | University of British Columbia | anna.blakney@msl.ubc.ca | Vancouver, BC, Canada | The Blakney Lab at University of British Columbia develops RNA-based therapeutics and biomaterial delivery systems to enable precision genetic medicines. By tailoring RNA payloads, targeting delivery to specific tissues, and modulating immune responses, the lab aims to create safe, effective, and personalized treatments. | We’re seeking teaming partners with expertise in gene editing technologies (e.g., CRISPR, base editing, prime editing) and personalized manufacturing of genetic medicines. Ideal collaborators will bring innovative approaches to therapeutic design, scalable and flexible manufacturing platforms, or capabilities that enable rapid, patient-specific development and deployment of precision genetic therapies. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Xuan Wang | Virginia Tech | xuanw@vt.edu | Blacksburg, VA | AI for biomedicine | Genetics medicine | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Dipanjan Pan | The Pennsylvania State University | dipanjan@psu.edu | State College, PA | Biosensing and molecular diagnostics as critical enablers in the space of in vivo precision genetic medicines because they address patient selection, real-time monitoring, treatment optimization, and safety. The current focus of the lab is to develop affordable and easily deployable mutation-specific diagnostics, rare allele detection, real-time and longitudinal tracking, dose response guidance and delivery validation, surrogate endpoints and companion diagnostics. | We are interested in partnering with companies developing interventions to halt, reverse, or prevent chronic diseases at the genetic level. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Charlene Son Rigby | Global Genes | charlene.sonrigby@globalgenes.org | San Francisco, CA | Global Genes is a patient advocacy organization. We equip rare disease patients and advocates with tools and training to advance research and activate communities. Our RARE-X platform provides a scaled model for longitudinal data collection to support research and therapy development. It includes >85 disorders and spans >90 countries. With 820+ patient advocacy groups in our Global Advocacy Alliance, we work with patient advocates, industry and academia to build vital ecosystems in rare disease. | We are open to partnering with drug developers, researchers, patient advocates, regulatory experts and other innovative collaborators. We are seeking partners with expertise in therapy development who want to team with patient advocates with expertise and experience in registries, natural history studies, patient-reported outcomes, and patient identification and recruitment. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| John Cooke | Houston Methodist Research Institute | jpcooke@houstonmethodist.org | Houston, TX | Our Center for RNA Therapeutics has faculty innovators in RNA biology and therapeutics. In addition, our RNACore has expertise in the synthesis, purification, encapsulation and validation of mRNA and circular RNA, both research and clinical grade. In our ISO7 clean rooms, our cGMP trained team has made mRNA and circular RNA for clinical development. We can assist mRNA gene editing programs get to the clinic. To our knowledge, we are the only AMC with such capabilities. | Scientists with expertise in mRNA gene editing that need our help (cGMP manufacturing with expertise in regulatory interactions with the FDA regarding RNA therapeutics) | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Yoel Shiboleth | TargetGene Biotechnologies LTD | yoel@targetgenebio.com | Rehovot, Israel | TargetGene is a recognized innovative gene-editing pioneer with our proprietary, best-in-class TGEE platform. TGEE is a super-precise dual-guide-based editing system that is as versatile as CRISPR but without the latter's off-target safety concerns. In-vivo genetic manipulations are exceedingly susceptible to off-target risks. Thus, we are leveraging TGEE's advantages to rewire cell transcriptomes to develop safe and effective cell therapies for cancer, immune-related-, and genetic disorders. | TargetGene is seeking partners participating in development of novel cell and gene-therapies that can benefit from our safer gene-editing platform, including Clinicians, Academia, Delivery-platform developers, Pharma, Patient advocacy organizations and CDMOs. | Module 1: Platform PGMs Design and Development |
| Lina Zelinger | Geneyx | lina@geneyx.com | Israel | Clinical Genetics. We offer a comprehensive (AI based) solution from raw sequencing data to a full clinical report with actionable insight. We provide AI-based, fully customizable and regulation compliant analysis that utilizes numerous annotation sources, phenotype-genotype correlation, pathogenicity prediction tools, automated ACMG guidelines integration, visualization and more. Our solution is sequencing method agnostic and is compatible with genomic, epigenomic and transcriptomic data. | We are happy to connect with any institute and organization that needs high quality and rapid genetic analysis. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Sean Russell | PrimeRA Pharma Partners | info@primerapharmapartners.co.uk | Nottingham, United Kingdom | PrimeRA Pharma Partners is a leading consultancy of regulatory and biotech leaders guiding drug development from discovery to global approvals. We specialize in regulatory science, CMC development, and translational strategy, with deep expertise in cell and gene therapies. Our team has supported IND/CTA submissions, engaged with FDA, EMA, EU national agencies, and MHRA, and advised on expedited programs as well as orphan designations and complex CMC/quality dossiers. | We seek to partner with teams advancing precision genetic medicines where we can help solve complex regulatory challenges, from translation bottlenecks and CMC hurdles to regulatory uncertainty and global alignment. Our goal is to provide practical solutions that keep science moving toward patients. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Jonathan Finn | Basecamp Research | john.finn@basecamp-research.com | Cambridge, MA | Basecamp Research (BCR) designs curative therapeutics using frontier AI models trained on BaseData™, the largest genomics database spanning 10B new genes from 1M+ new organisms. BCR’s EDEN™ family of foundation models solves complex biological design tasks, with a headline focus on solving programmable gene insertion in a single enzyme system - enabling large DNA integration anywhere in any genome, with their first applications in genetic diseases and cell therapies for cancer. | We seek highly motivated teams with expertise in Delivery (safe, effective mRNA/DNA in vivo systems), Investigational Medicine (pre-clinical/regulatory advice, early clinical trials), and manufacturing (rapid production for clinical to commercial scale). | Module 1: Platform PGMs Design and Development |
| Sunitha Malepati | Buffalo Initiative | sunitha@buffaloinitiative.org | Mclean, VA | Buffalo Initiative is an integrated platform for patient-led therapeutic development. We focus on translating preclinical innovations to clinical candidates and ultimately approved medicines for patients. Our initial work is in rare genetically defined neurodevelopmental disorders. The organization is building a scalable patient-led development model and fund that reduces redundancies in nonclinical development, manufacturing and clinical development across rare diseases. | Buffalo Initiative seeks Module 1 partners advancing neurogenetic targets. We bring platform strengths in Module 2 (hub for working with disease-specific patient groups, patient-centric decentralized trial design, master protocols and evidence generation) and aim to extend therapies into Module 3 through equitable distribution beyond clinical studies. As a nonprofit convening hub, we can enable neutral collaboration, data sharing and sustainable pathways for rare disease therapeutics. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| David Peritt | Lupagen, Inc. | DAVID.PERITT@lupagen.COM | Dallas, TX | Lupagen is a clinical stage company with a novel bedside delivery system that improves safety of gene editing of immune cells via an extracorporeal process. | For ARPA-H contractees that are targeting immune cells (T, NK, HSC, etc.) and are concerned about dosing accuracy or off target side effects, we are happy to explore using the extracorporeal system to delivery your particular therapy. | Module 3: Real-world Capabilities Pilots and Scaling |
| Kosta Kleitsas | Dyno Therapeutics, Inc. | kosta.kleitsas@dynotx.com | Watertown, MA | DynoTx pioneers AI-powered delivery and genetic sequence design for transformative medicines targeting eye, muscle, and CNS diseases. We specialize in quantifying the performance of delivery technologies with superior efficiency, target specificity and safety in animal models, including NHPs. We offer capabilities to develop novel delivery technologies, access to leading-edge AAV capsids, and AI methods for sequence optimization of payload function in vivo to accelerate clinical translation. | We seek partners with innovative payloads that are looking to test payload function in NHP, mouse or other relevant models to assess readiness for human translation. We are also looking for partners with manufacturing, clinical development and regulatory expertise. | Module 1: Platform PGMs Design and Development |
| Judee Sharon | KuriBio | judee@kuribio.com | Berkeley, CA | KuriBio is pioneering a logic-gated liposomal nanotechnology platform that can deliver PGMs to a variety of tissue types. Our liposomes are functionalized with integral proteins which enable logic-gated activation of encapsulated cell-free expression systems (CFES) that manufacture or unmask payloads only inside the target cell. This PGM delivery technology solves the problem of targeting specificity and toxicity while dramatically increasing the safety profile of editing technologies. | KuriBio is looking for teammates who are developing PGMs that can be used as payloads for our delivery platform. We are also looking for partners that can assist us with Module 2 and 3 of this ISO. We are open to partners in academia, clinics, companies, patient advocacy groups and any other team that can make use of our technology. | Module 1: Platform PGMs Design and Development |
| Ashley Winslow | Odylia Therapeutics | awinslow@odylia.org | Atlanta, GA | Odylia Therapeutics is a nonprofit focused on developing gene therapies for rare inherited retinal diseases and other under served rare diseases. Our current research includes preclinical and clinical-stage programs in ocular, neurodevelopmental, and overgrowth disorders, with an emphasis on conditions lacking commercial interest but high patient need. | Odylia Therapeutics is seeking partners interested in novel genetic therapies that can benefit from Odylia's expertise in gene therapy, as well as programs looking for help with strategic or regulatory navigation. We have an established network of translational and manufacturing partners, and additional depth in project management, clinical planning, and patient engagement. Our internal pipeline focuses on gene therapies, and we have partnerships involving small molecules, devices, and ASOs. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Elisabeth Gardiner | Tevard Biosciences | egardiner@tevard.com | Boston, MA | Tevard’s tRNA platform technology restores full-length proteins in diseases like Duchenne Muscular Dystrophy and dilated cardiomyopathy caused by nonsense mutations. Tevard’s potent and proprietary suppressor tRNAs readthrough premature stop codons, enabling durable, disease-targeted delivery via low doses of tissue specific AAV. Preclinical data show strong dystrophin and Titin protein restoration with AAV delivery, improved motor function, and excellent safety across multiple models. | Partners who are eager to take on rare disease with a strong interest in nonsense mediated genetic diseases. We seek to team with patient advocacy groups, AAV delivery and non-viral delivery companies, and other groups seeking to test our novel suppressor tRNAs in their unique animal and cellular models. We love to collaborate and we want to help facilitate excellent science in the rare disease space. Let's get this technology to patients!! | Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development |
| Yael Weiss | Mahzi Therapeutics | yael@mahzi.com | South San Francisco, CA | Our focus and expertise is in developing disease modifying therapies for rage genetic neurodevelopmental disorders (including epilepsy, autism and intellectual disabilities). | Companies that have pre-clinical platforms that can be used for delivering therapies to the CNS | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Eerik Kaseniit | Radar Therapeutics | eerik@radartx.bio | Berkeley, CA | Radar Therapeutics develops precision-targeted, programmable genetic medicines using rationally designed sensors that act as a “lock-and-key,” ensuring payloads activate only in the presence of markers unique to the target cell. This specificity enables tunable spatio-temporal control and allows a single sensor to address clusters of diseases in the same cell type. Our sensors are compatible with DNA and RNA delivery, flexibly integrating with diverse payloads and systems. | Radar seeks Module 1 partners developing novel delivery vehicles (e.g., LNPs, PNPs, xNPs, VLPs) and/or therapeutic payloads (e.g., CRISPR editors, ASOs, base/prime editors) to integrate with our cell-type–specific sensor platform. We also seek Module 2 and 3 collaborators with preclinical models, translational expertise, clinical infrastructure, or regulatory and manufacturing capabilities to advance therapies to patients. | Module 1: Platform PGMs Design and Development |
| Jennifer Gori | Prime Medicine, Inc. | jgori@primemedicine.com | Cambridge, MA | Prime Medicine is progressing a diversified portfolio of investigational therapeutic programs organized around our core areas of focus: hematology, immunology & oncology, liver and lung. Across each core area, Prime Medicine is focused on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. | Strategic partnerships that complement our expertise and are focused on advancing genetic medicines to clinic. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Benjamin tenOever | NYU School of Medicine | Benjamin.tenOever@NYUlangone.org | New York City, NY | Through DARPA's HERMES program, we are advancing a novel synthetic RNA vector for safe, precise in vivo delivery of genetic payloads. This platform, enables scalable, low-cost GMP production and executes a transient, self-resolving "hit-and-run" biology to delivery base/prime editors or support targeted gene insertion without immune activation. Proof-of-concept in mice has achieved robust delivery to lung, brain, heart, and/or kidneys through various routes of administration. | Future efforts for our organization include identifying rare diseases with well established advocacy groups for us to focus on as well partners to help with drug development and clinical trials (although some of this can be performed at NYU as well) | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Rob Freishtat | AlphaRose Therapeutics, Inc. | rfreishtat@gmail.com | Austin, TX | The company is currently focused on ASOs and RNA drugs, for their affordability and amenability to optimization with AI and robotics. AlphaRose owns two proprietary RNA platforms. AbcDNA, which could be the safest and most durable ASO backbone yet. And SOT Caller, which is a shRNA epigenetic platform, which is a great tool for loss of function mutations. | We are open to partners who are willing to be bold and push the limits of efficiency in oligo design and delivery. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Terry Jo Bichell | COMBINEDBrain, Inc. | terryjobichell@combinedbrain.org | Brentwood, TN | We are a patient advocate run organization, but we are not your standard patient advocacy group. We are a research-based non-profit run by translational neuroscientists devoted to speeding the path to treatments. We work nationally (and internationally), virtually, across 120 disorders. We work with the leaders of these groups to fill gaps at every stage from diagnosis, through natural history data collection, biomarker identification, endpoint development and clinical trial design. | We are looking for partners with innovative neurological treatment strategies and delivery methods who are committed to including patient advocates at every stage of the process. And, we are looking for expert clinical trial centers who are willing to trial these novel therapeutics while mentoring local physicians through a decentralized patient-friendly trial system. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling, Module 1: Platform PGMs Design and Development |
| Ibrahim Alabri | Arbor Biotechnologies | ialabri@arbor.bio | Cambridge, MA | Arbor develops programmable DNA editors to move from treatment to functional cure—and ultimately prevention. Our wholly owned portfolio of compact, precise CRISPR editors is designed to access >90% of the genome and pair with LNP/AAV/novel non-viral delivery. We match mechanism to disease biology across liver, CNS, and muscle to enable one-time, durable therapies at scale, with a focus on rare, often pediatric, conditions. | Seeking partners to develop and deliver cures for genetic diseases, especially pediatric. We’re looking to team with (1) delivery innovators (engineered capsids, next-gen LNPs, peptide/polymer carriers) to achieve disease-relevant tissue targeting and durable, in vivo editing; and (2) children’s hospitals and rare-disease networks for natural-history cohorts, registries, and biobanks that enable first-in-child trials | Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development |
| Anna Marie Wagner | Transfyr | annamarie@transfyr.ai | Cambridge, MA, MD | Precision genetic medicine (PGM) discoveries and rare disease insights are often lost in translation or silos. At Transfyr, we are building a new paradigm for AI-driven infrastructure to enable the passive observation, capture and exchange of scientific know-how between individuals, robotics, and organizations, like APIs in software. Our multimodal, self-evolving AI tools interpret the complex physical realities of science to make knowledge robust, reproducible, and transferable towards cures. | We seek partners developing PGMs and committed to “one-shot” tech transfer at each “hand-off” from the lab to manufacturing to the clinic. We provide the agentic AI-powered technology stack that (1) observes implementation of science in the physical world, (2) captures tacit knowledge and execution intelligence (incl. long-term), and (3) uplift scientists, manufacturers, and clinicians to troubleshoot and support tech transfer across diseases, sites, and PGM formats in the real world, at scale. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Eunji Chung | University of Southern California | Eunchung@usc.edu | Los Angeles, California, CA | We aim to develop cell-specific targeted therapy for genetic kidney diseases | We are aiming to partner with industry and foundation partners that will catalyze our scale-up and clinical efforts | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Krishanu Saha | University of Wisconsin-Madison | ksaha@wisc.edu | Madison, WI | Developing and clinically applying innovative RNA-based CRISPR therapies for rare diseases in both children and adults. | UW-Madison is open to teaming for all phases of development. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Mark Bathe | MIT | mbathe@mit.edu | Cambridge, MA | Our lab is pioneering a new non-viral delivery platform, DNA-based Virus-like Particles (DVLPs). DVLPs are rationally designed, self-assembled nucleic acids that are (1) AAV in size (~25 nm); (2) amenable to ASO, siRNA, guide RNA, RNP, mRNA, ssDNA, and cssDNA cargo; (3) formulated with targeting ligands such as lipids, sugars, peptides, aptamers, and antibodies; (4) non-immunogenic (no anti-vector IgG); (5) biodegradable; and (6) scalably manufactured. | We are keen to contribute to Module 1 to bring this innovative non-viral vector through pre-clinical testing to the clinic in Module 2 and 3. We seek to address challenging delivery problems such as large cargo delivery to the CNS and cell-specific targeting. We have a potential clinical collaborator in the pediatric rare movement disorder space that could contribute to Module 2 and 3 of a high-risk PGM-2 program. | Module 1: Platform PGMs Design and Development |
| Eric Josephs | Stony Brook University, SUNY | eric.josephs@stonybrook.edu | Stony Brook, NY | Our lab has developed high-throughput screening techniques of variants of CRISPR components for (1) rapid testing of CRISPR activity to identify the most active target sequences; (2) experimental screening of CRISPR activity at nominated or potential off-targets for a given target; (3) gRNA variant generation that, for a given target, can eliminate activity at known off-target sequences while maintaining therapeutic efficiencies; and (4) gRNA variant generation able to discriminate between SNVs. | Looking for academic or industrial partners with potential gene, pathway, or disease targets of interest who want efficient methods to optimize CRISPR components for editing efficiency and increased (personalized or population-level) safety; we're expanding our capabilities to other CRISPR modalities (base and prime editing) and to personalized CRISPR component variant generation based on the presence of specific off-target sequences potentially found in a patient's genome. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Amberlie Clutterbuck | The Emmes Company | aclutterbuck@emmes.com | Rockville, MD | Emmes is a full service CRO focused on accelerating clinical development of new treatments in rare diseases. With over 45 years of experience, we combine our industry knowledge alongside personal experience within rare disease and cell and gene therapy clinical trials, making us a superior research partner for Sponsors looking for a tailored service experience. We are experts in RD Medical Research, Clinical Research, Regulatory, and Bioinformatics specific to Figure 5 in the ISO. | We understand the THRIVE program is disease-agnostic and Emmes’ 45 years of experience has supported therapeutic areas underfunded by industry in need of support. We are looking for partners for Module 1: Platform PGMs Design and Development. Our 125+ clinical studies are focused on advancing cell or gene therapies in therapeutic areas including hematology/oncology, ophthalmology, cardiology, and pulmonology. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Shengdar Tsai | St. Jude Children's Research Hospital | shengdar.tsai@stjude.org | Memphis, TN, USA, TN | St Jude Children's Research Hospital is focused on developing a rapid and scalable platform for in vivo genomic medicines, starting with treatments for patients with bone marrow failure disorders. Our approach use AI-driven lead target discovery that leverage our capabilities to generate large-scale data for understanding the genome editors. Our in vivo therapeutic platforms will include targeted lipid nanoparticles delivering base editors or RNA-guided editors for programmable gene insertion. | Special areas of interest include: 1) in vivo delivery to human HSCs with targeted lipid nanoparticles or other innovative approaches, 2) novel editing platforms for programmable gene insertion, 3) innovative approaches to tune or modulate editor activity without large changes to therapeutic cargo, 4) cost-effective and scalable GMP manufacturing of mRNA and gRNA, 5) patient advocacy groups, and 5) companies seeking to broaden access to rare disease therapies. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Gabrielle Conecker | Decoding Developmental Epilpsies | gabrielle@scn8aalliance.org | Washington, D.C., DC | Decoding Developmental Epilepsies, a global patient organization, serves rare disease families & organizations through education and research including The Inchstone Project. Our multi-disciplinary team is uniquely experienced & poised to collaborate with platform and clinical delivery partners to fully embed an early focus on outcomes prioritized by patients. Our team will help assure a focus on endpoints and meaningful change to assure solutions yield transformative change in people’s lives. | We seek partners advancing platforms and delivery mechanisms who require & value patient advocate leads bringing proven expertise in researching needs and priorities of the most severely impacted pediatric populations with high unmet needs. Our experience is broad & deep, bringing global leadership in FFP measurement of severely impacted populations. We will bring the patient voice to ground new therapies in documented patient needs, priorities, meaningful change, & targeted recruitment. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Minghong Zhong | GeneLancet Biosciences, INC | MINGHONG.ZHONG@genelancetbio.com | Doylestown, PA | GeneLancet (www.genelancetbio.com) advances precision gene editing with its STAR platform (Seek–Tag–Amend–Release). This RNA-guided CRISPR polymerase system uses proprietary chemically ligated guide RNAs (LgRNAs) for scalable, non-cutting, non-permanent editing and modulation. LgRNAs excel over sgRNAs and pegRNAs in product quality (for safety), efficacy and specificity, and scalability (for manufacturing). | We seek collaborations in all CRISPR platforms and are particularly interested in NDD and cancers. Check out our precise and scalable gene editing solutions (https://crisprguidena.com/wp-content/uploads/2025/07/GeneLancetTechnologySeptember2025.pdf) and our guide RNA white paper (https://www.genelancetbio.com/genelancet-guide-rna-white-paper). | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Claudia Lee | Velvet Therapeutics | Claudia.lee@velvettherapeutics.com | Houston, TX | Velvet Therapeutics focuses on the delivery of DNA to the immune system for in vivo reprogramming using a polypeptide nanoparticle. The technology is scalable, without payload packaging limitation and removes the complexity of individualized manufacturing which is the status quo of approved cell therapies. We characterize the delivery efficiency to different immune cell types, develop formulations to create an off-the-shelf injectable solution, and evaluate pharmacokinetics. | Velvet Therapeutics seeks partners who understand rare genetic disease patient needs and have experience with DNA knock in. We look to provide an affordable, easy to distribute solution to deliver DNA for gene augmentation. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Silvia Duarte-Sanmiguel | NeucoreBio | sduarte-sanmiguel@neucorebio.com | Columbus, OH | Neucore Bio, is leading the development of non-viral exosome delivery options for next generation genetic medicines. The company's primary research focus is on non-viral engineered exosomes for therapeutic applications targeting rare genetic diseases and neuromuscular disorders. Central to their research is the proprietary FiXE™ (Fibroblast-Derived Engineering Extracellular Vesicles) platform, which utilizes exosomes to delivery genetic material in a tissue tropic manner to target cells. | We seek partners with validated therapeutic molecular cargos (e.g., siRNA, mRNA, DNA) targeting high-impact diseases. Neucore Bio provides our proprietary FiXE™ platform for precise tissue tropic, non-viral delivery to specific cells. Our goal is to utilize our delivery technology with novel therapeutic payloads to jointly develop breakthrough treatments for high-need petient populations, aligning with ARPA-H's transformative mission. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Jyotika Varshney | Verisim Life Inc. | jo.varshney@verisimlife.com | San Francisco, CA | VeriSIM Life develops hybrid AI–PBPK platforms to predict PK, toxicity, and efficacy across small molecules, biologics, and gene therapies. Our models integrate in silico, experimental, and real-world data to de-risk programs and enhance translational success. Our models further include assess lipid nanoparticle delivery, and enable patient stratification for precision medicine. | We seek collaborators with expertise in genetic medicine platforms (cargo and delivery), GMP manufacturing, and clinical translation of precision genetic therapies. Ideal partners bring capabilities in vector engineering, regulatory navigation, and patient engagement to complement our predictive infrastructure that provides state-of-the-art in silico tools for screening across dose-exposure, efficacy, and safety of such therapeutics. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| John Lewis | Entos Pharmaceuticals | john.lewis@entospharma.com | San Diego, CA | Entos Pharmaceuticals develops genetic medicines using the Fusogenix PLV delivery system. The Fusogenix platform enables redosable extra-hepatic delivery of DNA or RNA including gene editing cargoes with superior tolerability to LNP or AAV. The Entos team has extensive experience with formulation and delivery of gene editing cargoes, including whole gene insertion technologies. We have established an accredited clinical GMP manufacturing facility with full CMC and QA. | We have a next generation clinical stage genetic medicine delivery platform for RNA and/or DNA. We are seeking partners with validated innovative cargoes for rare disease, ideally for diseases manifesting in extra-hepatic cells and tissues. We have PLV formulations to target eye, lung, CNS and muscle. We are also seeking partners to support modules 2 and 3. | Module 1: Platform PGMs Design and Development |
| Brian Rash | Longeveron INC | brash@longeveron.com | Miami, FL | Longeveron Inc is a clinical stage regenerative medicine biotechnology company developing cellular therapies for life threatening birth defects and chronic aging-related conditions. Having completed five clinical trials in Alzheimer's disease, aging frailty, and hypoplastic left heart syndrome (HLHS), Longeveron has an advanced cell therapy pipeline and is pursuing an additional, transformative new cell therapy for pediatric dilated cardiomyopathy (PDCM). | To successfully develop a transformative therapy for PDCM, we hope to partner with a group that has well-characterized, GMP-qualified and HLA homozygous iPSC cell lines, with CRISPR capabilities, to engineer the final product. Selection and purification of the target differentiated cardiomyocyte precursor will be followed by a pre-IND animal study ideally in a swine model of myocardial infarction (for team partnering) to show sufficient efficacy and safety to begin clinical trials. | Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Timothy Ogden | Bardet Biedl Syndrome Foundation | timothy.ogden@bardetbiedl.org | Philadelphia, PA | A patient-led organization for BBS, focused on improving care and treatment for all people affected. We fund the Clinical Registry Investigating BBBS (CRIBBS) with 700+ enrolled patients with clinical data, and collaborate with BBS orgs in other countries with clinical registries. We support basic science and clinical trial development and execution via relationships with affected families. We support and collaborate with clinical centers to deliver excellent care for syndromic ciliopathies. | Researchers, clinicians, pharma and biotech companies dedicated to delivering solutions for, and working closely with, patients affected by syndromic ciliopathies. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Ramona Walls | Critical Path Institute | rwalls@c-path.org | Tucson, AZ | Critical Path Institute accelerates transformation of scientific innovation into real-world health impact by advancing regulatory science, data integration, and platform validation. Through global collaborations, C-Path builds trusted frameworks, model-informed tools, and federated data systems that enable faster, safer therapeutics. We bring proven expertise in regulatory innovation, platform evaluation, and development of interoperable, accessible data platforms to advance THRIVE’s mission. | C-Path seeks partners who are leading innovation in precision genetic medicine development, and scalable delivery models to co-create regulatory pathways, data networks and standards, and access frameworks. Ideal collaborators share our commitment to scalable, interoperable, and sustainable innovation in genetic cures. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Angelos Oikonomopoulos | Greenstone Biosciences | angelosoik@greenstonebio.com | Palo Alto, CA | Our proprietary hiPSC biobank comprises over 2,000 patient-derived lines spanning more than 100 rare diseases. We combine New Approach Methodologies (NAMs)—including organoids, microphysiological systems, and multi-omics readouts—with high-throughput cardiac electrophysiology and toxicity assays, as well as 3D cardiac and intestinal organoid workflows. This integrated platform generates predictive disease models, de-risks early programs, and guides candidate selection. | We’re seeking partners to integrate our AI/ML-driven discovery engine—powered by our extensive hiPSC biobank, CRISPR-enabled isogenic panels, and scalable 2D/3D organoid–microphys platforms (electrophysiology, contractility, toxicity), including patient-cohort “clinical-trial-in-a-dish” workflows—to accelerate preclinical, modality-agnostic evaluation of small molecules, biologics, and oligonucleotide therapies across diverse rare-disease models. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Stephen Ekker | University of Texas at Austin | stephen.ekker@austin.utexas.edu | Austin, TX | We have key expertise in gene editing especially in TALE base editors for mitochondrial DNA editing. While we use these in mitochondria, they and other technologies in my lab are great for nuclear editing too. | We are looking for key innovative partners for key preclinical testing. Also open to virus-free mRNA delivery platforms. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Christopher Ahern | University of Iowa | christopher-ahern@uiowa.edu | Iowa City, IA | The Ahern lab at the University of Iowa has developed a tRNA platform technology for the restoration of full-length proteins for diseases caused by premature termination codons (PTCs). This foundation tRNA library contains validated lead molecules for every known PTC that causes human disease. We and others have demonstrated high-fidelity encoding at the site of the PTC and broad tolerance in vivo. These data show that a single engineered tRNA can repair multiple disease causing PTCs. | We seek partners in the rare disease space where PTCs remain untreated. Our approach takes advantages of common PTCs, such as Arg-TGA, giving rise to a "rare as one" therapeutic strategy. To advance partnerships, we have animal models which contain PTC reporters that can be used to optimize delivery and tRNA engineering in vivo. We are highly collaborative and wish to work with academic and biotech partners to advance this technology to the clinic. | Module 2: Investigational Medicine |
| Denise Pinson | Medvacon Life Sciences LLC | denise.pinson@medvacon.com | Sparta, NJ | Medvacon brings deep expertise in regulated quality systems, compliance, validation, and audit readiness. Strengths include technology transfer, instrument/system qualification, CSV, data integrity and remediation support for cGMP environments. We can ensure a rigorous, audit-ready framework for downstream adoption, regulatory filings, and scale. | In a teaming relationship, Medvacon would provide the quality and regulatory framework that enables novel genetic delivery and engineering platforms to advance efficiently toward clinical and commercial readiness. We would lead validation planning, quality system integration, risk management, and compliance oversight to ensure all components of the THRIVE platform meet FDA and cGMP standards for safety, reliability, and scalability. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Monica Coenraads | Rett Syndrome Research Trust | monica@rsrt.org | Trumbull, CT | RSRT is the global leader in Rett research and committed to advancing genetic medicines. Rett is a neurodevelopmental disorder caused by mutations in the MECP2 gene. Remarkably, Rett-like symptoms are reversible in mouse models. RSRT has demonstrated in vivo genomic correction of pathogenic mutations via AAV and is actively pursuing RNP and nanoparticle-based delivery. RSRT has a biorepository, humanized mouse models, clinician networks, patient registry, biomarker and biosensor efforts. | RSRT is looking for partners with an interest in commercializing genetic medicines and experience in clinical design and operations. RSRT is also interested in partners with advanced CNS delivery and GMP production capacity. RSRT will contribute cost matching in clinical development. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Amit Benazraf | Mana.bio | amit@mana.bio | Tel Aviv | AI-driven drug delivery company focused on developing tissue-specific lipid nanoparticles (LNPs). Our proprietary machine learning platform designs LNPs that enable targeted delivery to specific organs and cell types. Mana built predictive AI models and achieved novel IP-protected in vivo results, including lung, T cell, and cartilage delivery of mRNA, CRISPR, ASOs, etc... | We seek partners across the precision genetic medicines ecosystem: therapeutic cargo innovators (RNA, DNA, modulators), rare disease clinical hubs and satellite clinics, patient advocacy groups, AI/data and regulatory experts, and commercialization partners. Mana.bio contributes an AI-driven LNP delivery platform to enable scalable, patient-centric solutions. | Module 1: Platform PGMs Design and Development |
| Feyisayo Eweje | Procure Bio | feweje@procurebiotech.com | Boston, MA | Procure Bio is developing recombinant, non-viral, protein nanoparticles for cell-targeted delivery of genetic medicines. Built from a human-derived protein, our platform offers biocompatibility, redosability, and genetic programmability of size, chemistry, and targeting moieties. Our focus: in vivo gene editing in multiple tissue types/disease states, including the retina (inherited retinopathies), lungs (CF), and hematopoietic stem cells (blood disorders). | We are looking to engage with partners across modules - specifically looking for entities with access to/ability to develop base and prime editors available for commercialization (Module 1); PAGs, clinical operations organizations, and regulatory experts to engage regarding trial design and execution in inherited blindness, CF, or blood disorders (Module 2); and entities with GMP-grade abilities for scaled recombinant protein manufacturing (Module 2 and 3). | Module 1: Platform PGMs Design and Development |
| Chelsea Trengrove | Neoclease | chelsea@neoclease.com | Boston, MA | Neo is pioneering AI-driven, gene-specific nucleases and mini prime editors that overcome the targeting, size, and off-target hurdles of Cas9. Our compact (<650 aa), PAM-independent editors enable tunable, single-gene precision editing in vivo. Using an AI-guided in silico design cycle that completes in one week and in vitro validation in six, we rapidly generate and optimize bespoke editors to advance curative programs for rare diseases. | Neo seeks partners to integrate our rapid-cycle gene editing platform into THRIVE: delivery innovators (LNP or peptide), clinical and hospital networks for first-in-human trials, and manufacturing collaborators to scale decentralized production. We also welcome data, regulatory, and rare-disease organizations aligned with democratizing precision genetic medicines. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Robert Getts | Code Biotherapeutics Inc | bgetts@codebiotx.com | SpringHouse, PA | Code Bio leverages its proprietary, novel, multivalent, synthetic DNA platform, 3DNA, which has been engineered to overcome many challenges inherent with delivery of genetic medicines, such as ability to re-dose, immunogenicity and gene size limitation to develop novel solutions to treat diseases. The platform is designed to support multivalent targeting using a variety of targeting molecules (antibodies, peptides, aptamers) to selectively and precisely target diseased cells and tissues. | We seek partners with genetic payloads designed to treat the high unmet needs of patients to combine with our 3DNA platform as a targeted delivery solution. Together we can develop a complete solution combining precision targeting with the right genetic payload on a unique DNA-based multivalent nanoparticle. | Module 1: Platform PGMs Design and Development |
| Aaron Leopold | NTxBio | aaron.leopold@ntxbio.com | Rio Rancho, NM | NTxBio produces the NTxscribe platform, an automated benchtop RNA manufacturing system that's designed to serve n-of-1 and rare disease patient populations. NTxscribe is fully-closed to enable instant changeover between patients, fits in any biosafety cabinet, and leverages continuous flow to deliver high-quality, high-purity RNA. | We are seeking upstream clinical partners who are looking for automated + distributable manufacturing solutions. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Pooja Tiwari | ARNAV Biotech | pooja.tiwari@arnavbiotech.com | Atlanta, GA | RNA and CRISPR based gene editors and modulators | ARNAV biotech can support in pre-clinical testing of these therapies including RUO RNA manufacturing, LNP discovery, assay development and tesing. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Emerson Villalta | Harmoni | emerson@harmonione.com | Minneapolis, MN | Harmoni builds consent-first, FHIR-native infrastructure to unify patient-authorized real-world data for rare and complex conditions. We integrate EHR, imaging, labs, wearables, and PROs into longitudinal, de-identified datasets that enable precision research, evidence generation, and coordinated care across decentralized settings. | Harmoni seeks collaborators advancing precision and longitudinal research who need secure, interoperable data foundations. We provide a consent-governed, FHIR-native environment that links clinical, genomic, and computational efforts—transforming real-world data into high-quality evidence for discovery and translation. | Module 3: Real-world Capabilities Pilots and Scaling |
| Walter Block | ImgGyd LLc | wfblock@wisc.edu | Madison, WI | The ImgGyd consortium is transforming the delivery of therapeutics to the brain through image-guided intraparenchymal delivery. Gene therapy companies have developed therapeutic agents with effective target engagement in small animal trials, but struggle to reach over 5% of the larger human brain. Fragmentary coverage slows the progression of fatal diseases, but can’t halt them. Our consortium will work with you to get much larger brain coverage with easier to define clinical outcomes. | We are looking for partners who have exciting approaches to lysosomal storage diseases, leukodystrophies, ataxias, Huntington's Disease and related brain diseases where broad brain and/or cerebellum is needed for effective treatment. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Frédéric ZAMPATTI | ALGENSCRIBE | contact@algenscribe.com | Nice | Algenscribe is a French biotool company developing a Gene Editing Platform to address some remaining challenges in the gene editing field using a nuclease (e.g. Crispr/Cas9). Our tools, at different stages of development, have potentially the ability to: • improve the efficiency of Gene Replacement through HDR, • reduce the level of indels linked to the cut made by the nuclease, • perform base editing through HDR, • avoid the use of AAV. | As a biotool company Algenscribe is seeking to team up with a leading Gene Editing company which: 1°) Lacks parts of technical tools to address module 1 2°) Could perform module 2 & 3. | Module 1: Platform PGMs Design and Development |
| Tay Shin | GT Biosciences | tay@gtbiosciences.com | Boston Cambridge, MA | We are building Deliveromics, an AI-driven in vivo screening platform that maps hundreds of lipid nanoparticle (LNP) delivery at single-cell resolution in a single animal. Deliveromics enables the rapid in vivo discovery and optimization of cell-type-specific LNPs, including those that cross the blood–brain barrier, to achieve precise delivery of genetic medicines for neurological and other rare diseases. | We seek partners with therapeutic payloads (mRNA, siRNA, ASO, gene editors, etc.) and disease expertise who can leverage our delivery platform for the treatment of rare diseases. We are also interested in collaborating with manufacturers, NHP facilities, and those with regulatory experience to advance LNPs into clinical development. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Oren Glanz | Lempo Therapeutics Ltd. | oreng@lempo-tx.com | Rehovot, Israel | Lempo develops MP001, an autologous CD34+ HSPC therapy using ex vivo non-viral CRISPR/Cas9 knockout of the MPO gene to treat Systemic Sclerosis-associated Pulmonary Arterial Hypertension (SSc-PAH). This approach aims for the durable suppression of NETosis and oxidative stress by producing myeloperoxidase (MPO)-deficient neutrophils. We are collaborating with UCLA on the First-in-Human clinical trial planned for next year. | We are actively seeking a delivery partner (i.e., LNP, VLP) capable of delivering RNP CRISPR/Cas9 cargo directly into bone marrow hematopoietic stem cells (HSCs) in vivo. We also seek partners to conduct clinical trials and manufacture our in vivo product. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| MARINA KOLOCHA | Lifecycle Integration for Treatments in Healthcare | mk.mplm@outlook.com | PITTSFIELD, MA | LITREATH biopharmaceutical expertise is spanning more than 20 years, including trusted advisory, executive leadership and integrated lifecycle asset discovery, development, approval, access, and use of over 290 assets for treatment seriously debilitating and life-threatening diseases (SDLTDs) (72% in pediatrics) in 45 therapeutic classes, resulting in approximately $4.8bn in capital committed to alliances with biotechnology and pharmaceutical partners of all sizes. | In our work we apply validated tools and algorithms designed for medical product discovery, development and research predictability, acceleration to regulatory review and approval, efficient safety monitoring, maximized patient’s access and use or exit scenario. These tools and algorithms support multi-step and multi-stakeholders’ processes with decisions, milestones for enough evidence, added value and inflection points | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling, Module 1: Platform PGMs Design and Development |
| Neal Amin | Sculpta, Inc. | neal@sculpta.bio | San Francisco, CA | We develop Protein Sculpting therapeutics that leverage the safety, specificity, and efficacy of antisense oligonucleotide (ASO) splice-switching PGMs for a wider array of targets and mechanistic effects. Driving our novel target discovery platform is the first ever, single cell-resolution atlas of RNA splicing events in the human body, across the aging spectrum from development into adulthood, in health and disease, and across all major organ systems. | We are seeking partners with 1) unique oligonucleotide delivery approaches, 2) platforms that can further utilize our corpus of single cell RNA splicing data, and 3) individuals who want to have serious discussions about out-of-the-box approaches that would be transformative for patients. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| SUBRATA BATABYAL | Nanoscope Technologies LLC | sbatabyal@nanoscopetech.com | Bedford, TX | Nanoscope has developed a range of biomedical technologies which include diagnostics and therapeutic devices and molecules. These have already generated interest from several clinicians, industries and leaders. We strive to translate the technology to market and bedside. | Nanoscope has developed nonviral laser-based precision gene delivery and molecule to treat disease like various neurological disorders. We seek to partner with academics and patient advocacy group to advance our technology in alignment with ARPA's THRIVE program. c | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Sang hoon Kim | Nanoscope Instruments inc | skim@nanoscopeinstruments.com | Bedford, TX | Nanoscope Instruments, Inc. (Bedford, TX) develops advanced OCT-guided imaging and intervention systems for ocular research. Our R&D focuses on multimodal diagnostics, compact optical designs, and real-time imaging. We also apply our tools and expertise with partners to treat hereditary rare diseases using our proprietary non-viral gene delivery platform. | Nanoscope Instruments, Inc. seeks partners who share our vision of advancing imaging and gene therapy for rare diseases. We look for collaborators in research, clinical translation, and product development with strengths in ophthalmology, neuroscience, and biotechnology. Ideal partners bring complementary expertise to help expand our non-viral gene delivery platform and accelerate patient-focused innovation. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Vincent Forster | Nerai Bioscience | vf@nerai.bio | Zurich, Switzerland. | Nerai leverages high-throughput protein engineering and AI to develop a new generation of highly specific and programmable genome editors. Our research is focused on creating curative, one-time genetic therapies for rare inherited metabolic diseases. The company's lead program is aimed at correcting the genetic basis of citrullinemia type 1, a urea cycle disorder affecting the liver, by translating a precisely targeted in vivo gene editing therapy. | We seek strategic partners to advance our LNP-mRNA genome editing therapy toward clinical translation. Specifically, we aim to collaborate with leading reference centers experienced in running pediatric urea cycle disorders trials, GMP-certified vendors for large-scale LNP-mRNA manufacturing, and expert preclinical CROs capable of conducting GLP-compliant IND-enabling studies in non-human primate. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Poorya Hosseini | Collage Bio | ph@collagebio.com | New York and Boston, NY | Collage Bio is pioneering a new class of programmable RNA therapeutics built on novel antisense architectures that go beyond the limitations of conventional ASOs and siRNAs. Our patented, structurally enhanced polynucleotides are designed and optimized through an AI-driven platform to enable precise, multi-target RNA modulation across diseases where durable, transcript-level control is essential for in vivo genetic medicine. | We seek partners developing advanced in vivo delivery solutions that enhance precision and efficacy across existing tissues or unlock access to novel cell types, as well as experts in target biology and translational models enabling clinical validation of next-gen precision genetic medicines. Our programmable antisense platform pairs seamlessly with such innovations to accelerate affordable, scalable cures. We also welcome collaborators for Modules 2 and 3. | Module 1: Platform PGMs Design and Development |
| Sherwood Yao | Atom Bioworks | s.yao@atombioworks.com | Cary, NC | UPenn spinout. Develop AI driven protein binder for LNP to achieve cell type specific delivery of RNA payloads. | Clinical PI or hospital-affiliated investigator (MD) in CNS disease, Regulatory development firm or biotech with prior platform IND experience | Module 1: Platform PGMs Design and Development |
| Wayne Poon | NeuCyte, Inc. | wayne.poon@neucyte.com | Mountain View, CA | Stem Cell-Based Models of N=1 Rare Diseases | We are looking for partners to validate their in vivo therapeutics in N=1 iPSC-derived models to establish | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Amanda Haupt | Revvity | amanda.haupt@revvity.com | Waltham, MA | Revvity offers the Pin-point™ base editing platform for precise, multiplex in vivo editing, enabling simultaneous ABE + CBE knock-in and knockout. We provide reagents, assays, automation, and analytics tools, plus diagnostics/omics expertise and a global network of clinical labs for genomic and biomarker analysis to support the development of safe, scalable precision genetic medicines. | Revvity seeks collaborators with complementary expertise in non-viral delivery technologies, translational or clinical development, and regulatory strategy. Ideal partners include academic centers, gene-therapy biotechs, and patient-focused organizations who can provide in vivo validation, patient access, and IND-enabling infrastructure to advance DSB-free precision medicine platforms together. | Module 1: Platform PGMs Design and Development |
| Heidi Bjornson-Pennell | CZI | heidi.bjornson@chanzuckerberg.com | Redwood City, CA | CZI’s Translational Impact & Engagement (TIE) program leads the Rare As One initiative, uplifting and supporting nearly 100 patient-led rare disease organizations that are driving groundbreaking research within their communities. By strengthening their research capacity and fostering deep partnerships between patients, scientists, and clinicians, we aim to accelerate translational breakthroughs and demonstrate how centering patients as key stakeholders leads to faster, more meaningful cures. | We are interested in supporting the development of teams with partners committed to building meaningful, equitable collaborations with patient-led organizations—engaging patients as integral contributors from project design through implementation, insight generation, and result interpretation, and leveraging their deep community connections and patient-developed resources to drive impactful, community-informed research. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development |
| Ivan Martinez Duncker | CDG CARE | duncker@cdgcare.org | Colorado Springs, CO | CDG CARE drives translational research on 200+ Congenital Disorders of Glycosylation—systemic diseases ideal for developing modular, multi-tissue genetic therapies. Our focus spans biomarker discovery, natural-history data (RDCRN), FDA-aligned clinical trials, and AI-driven analytics. By linking glycosylation biology to cancer, immune, and metabolic pathways, we enable scalable precision-medicine innovation. CDG CARE is connected with multiple clinical and research sites in the US and abroad. | We are a global patient advocacy organization seeking partners across all stages of precision genetic medicine development to co-develop scalable, affordable in vivo therapies. We aim to link our CDG community and Clinical Research Network with biopharma and academia, integrating glycosylation biology and precision genetics across rare and common diseases through clinical, manufacturing, and data innovation. | Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Jing Nie | Riboway Therapeutics | jing.nie@riboway.com | Cambridge, MA | Riboway Therapeutics leverages AI to decode and target RNA for precise control of protein expression and function. Our smart ASO technology enables us to upregulate, downregulate, activate, or inhibit target proteins, depending on therapeutic needs. So far, we have successfully validated 5 out of 5 targets attempted. Our lead program, targeting the root cause of a rare genetic ataxia disease, demonstrated superiority in potency, safety, dosing frequency, and clinical trial design. | Riboway Therapeutics’ AI-powered RNA targeting platform is making every target protein druggable, in any direction (upregulation, downregulation, activation, or inhibition). We can offer novel solutions for any THRIVE project. We would be the perfect partner if you are looking for a new solution or cargo for your delivery system. | Module 1: Platform PGMs Design and Development |
| Daniel Getts | CREATE Medicines | baman@createmedicines.com | Cambridge, MA | CREATE Medicines is developing an all-RNA gene-editing platform enabling precise, site-specific genomic integration without DNA templates or double-strand breaks. We engineer LINE-1 retrotransposon-based gene writers, delivered via targeted LNPs and non-integrating lentivirus, to create a scalable platform for in vivo correction of hereditary and rare diseases—built on our experience advancing RNA therapeutics into human trials. We can deliver fully on Module 1. | Seeking teaming partners for Modules 2 & 3, including: • Clinicians and investigators interested in in vivo gene editing and RNA-based therapeutics • Academic medical centers specializing in hereditary and rare diseases • ARPA-H pilot network participants or regional gene-therapy hubs able to support multi-site clinical readiness • Patient advocacy partners committed to ensuring patient perspectives guide program design and clinical translation | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Rona Lee | cure NDD | curendd@gmail.com | San Francisco, CA | Cure NDD focuses on accelerating research and therapy development for X-linked neurodevelopmental disorders (NDDs). Our priorities include advancing genetic and RNA-based treatments, improving diagnostic pathways, and fostering data sharing among clinicians, researchers, and patient families to better understand disease mechanisms, biomarkers, and therapeutic outcomes across rare NDDs. | Cure NDD seeks partners with expertise in chemical biology, RNA therapeutics, and translational research to accelerate drug discovery for X-linked neurodevelopmental disorders. We also welcome collaborations with clinical trial developers, CROs, and academic networks experienced in rare disease trials, biomarker development, and patient registry integration to bring novel therapies from bench to bedside. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Andrew Varley | RNA & Formulation Core; University of British Columbia | rfc@msl.ubc.ca | Vancouver, British Columbia, Canada | Our organization advances RNA and lipid nanoparticle (LNP) manufacturing to enable precision genetic medicines for rare diseases. We develop rapid, high-quality synthesis and impurity control methods for mRNA, saRNA, and circRNA. Our research integrates scalable RNA manufacturing, advanced analytics, and translational collaborations to deliver safe, effective in vivo therapies for hereditary disorders. | Our RNA and LNP manufacturing expertise enables partners to validate ideas and explore sequence designs, including UTRs, terminal additions, and poly(A) tail designs. We provide modular systems for efficient production of mRNA, saRNA, and circRNA, allowing rapid comparison of constructs and delivery outcomes to accelerate development of precision genetic medicines. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Kira Mann | MitoAction | kira@mitoaction.org | Boston, MA | MitoAction is developing a groundbreaking pre-damage intervention platform to transform mitochondrial disease care. By identifying patients before irreversible damage occurs, the platform enables earlier diagnosis, connects them to four breakthrough therapies in development, and creates a scalable system to overcome rare disease research bottlenecks, advancing precision medicine and improving patient outcomes worldwide. | MitoAction seeks innovative partners who share our vision for early diagnosis and precision intervention in mitochondrial disease. Ideal collaborators bring expertise in data science, biomarker discovery, clinical research, or digital health infrastructure to help validate and scale our pre-damage intervention platform, accelerating therapeutic development and improving outcomes for rare disease patients. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Ariel Vitenzon | MuriPhys LLC | avitenzon@muriphys.com | Durham, NC | MuriPhys, LLC, is an AAALAC Accredited rodent focused preclinical CRO with extensive in vitro and in vivo translational. Our comprehensive infrastructure supports the full spectrum of research, including behavioral phenotyping, electrophysiology, surgical manipulation, in vivo imaging and ophthalmology capabilities. | As a preclinical CRO we are looking to team with potential drug developers and support their IND enabling projects. Our mission is to bridge the gap between discovery and clinical readiness by providing scientifically rigorous, customizable preclinical research services tailored to the unique needs of emerging therapeutic platforms, particularly in the areas of gene therapy, regenerative medicine, and neuroscience. | Module 1: Platform PGMs Design and Development |
| Lauren Chaby | Project 8p | info@project8p.org | New York, NY | We are a patient-driven research foundation accelerating solutions and therapies for chromosome 8p disorders as a model for the 16 million individuals affected by chromosomal rearrangements. Through our global multi-omics Natural History Study, biobank, and multidisciplinary clinics, we enable rapid patient recruitment, biospecimen access, and integrated data generation—leveraging high-tech tools to accelerate discovery and clinical trial readiness across rare diseases. | We seek partners developing innovative therapeutic platforms, delivery systems, and technological tools that embrace patient-centered collaboration. Ideal teammates include technology developers committed to co-designing studies with our community, sharing data openly, and engaging our shared clinician networks through scalable, patient-friendly innovative approaches to accelerate equitable access and real-world impact. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling, Module 1: Platform PGMs Design and Development |
| Rohini Kalvakuntla | Helex | rohini@helex.bio | Boston, MA | Helex is developing novel genetic medicines for kidney diseases with a high unmet need. Helex's proprietary Lipid nanoparticle technology platform has optimized the delivery of various therapeutic cargo to the kidney cell types to address a plethora of diseases. Our lead program is a first-in-class LNP-gene editing based therapeutic for Autosomal Dominant Polycystic Kidney Disease. We are also seeking partnerships to use our LNP technology for targets in the kidney, liver, lung and spleen. | We are looking to partner with therapeutic companies that have proprietary technologies in the space of gene therapy, gene editing, siRNA, mRNA and are looking for non-viral, LNP delivery partners. We are a very experienced LNP group and have worked with multiple routes of administration like IV, renal artery, retrograde ureter amongst others. We have significant experience with LNP formulations and scale up with various therapeutic cargoes bringing a strong and versatile delivery platform. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Lauren Beach | Northwestern University Feinberg School of Medicine | lauren.beach@northwestern.edu | Chicago, IL | The Advancing Opportunities for Clinical Science Equity (ADVOCATE Center) at Northwestern University's Feinberg School of Medicine features a talented interdisciplinary team including expertise across the clinical and translational spectrum in medicine, genetics, clinical research, informatics, public health, law, bioethics, scientific writing, patient reported outcomes, and patient and community outreach and engagement. We provide clinical research solutions across diverse therapeutic areas. | ADVOCATE is seeking partners with expertise in drug development for rare neurodevelopmental diseases to complement our strong skills in genetics, clinical informatics, participant recruitment, regulatory solutions, implementation science, bioethics, and scientific communications to support success THRIVE teams for this ARPA-H opportunity. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Yang(Claire) Zeng | DoriNano, Inc. | claire@dorinano.com | Cambridge, MA | DoriNano is developing a programmable DNA origami nanoparticle platform for precision genetic medicines, enabling delivery of siRNA, mRNA, and DNA gene insertion cargos with tunable structure, repeat dosing, and tissue targeting. We have demonstrated efficacy and safety in cancer and infectious disease models (Nature Nano, NBE) and established cargo quantifiable methods, with NHP tox data further supporting our platform. | We can deliver diverse cargos with precision, homogeneity, safety, and tissue targeting. We seek partners with in vivo disease models, genetic cargo/editing technologies, and clinical or translational capabilities. Ideal collaborators offer complementary strengths in PGM payloads, rare disease biology, or CMC/IND support to co-develop multi-cargo, multi-indication programs aligned with THRIVE goals. | Module 1: Platform PGMs Design and Development |
| Robert Kruse | HydroGene | robert@hydrogenetx.com | Cambridge, MA | HydroGene is developing a delivery platform based on local precision administration into specific organs via a physical delivery method. We are payload agnostic and can accommodate DNA, RNA, or protein with no size limitations. We have validated our approach in porcine and non-human primate models with advanced data in liver showing redosing and durability, along with early delivery data in kidney and pancreas. | HydroGene seeks partners with gene insertion and gene editing platforms to complement our delivery approach, especially ones who would benefit from large DNA template delivery, as well as technologies around better gene regulation. We would also be interested in partners who support clinical translation in Module 2 and 3 in collaboration with physicians on our team. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Melissa Chaikof | Usher 1F Collaborative | mkchaikof@usher1f.org | Newton, MA | A patient-led organization focusing on gene and drug therapies to save the vision of those with Usher syndrome type 1F. We support translational research, currently with two very promising gene therapies, a drug screen to identify existing drugs for repurposing, and a 4-year natural history study in partnership with Foundation Fighting Blindness, currently in year 4 of 4. | Researchers, clinicians, and biopharma companies dedicated to helping us advance our gene therapies to clinical trial and test our identified drugs for efficacy for halting or slowing progression of vision loss in patients with Usher 1F. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Yagiz Aksoy | EosGene Therapeutics | info@eosgene.com | Sydney | EosGene Therapeutics develops spatiotemporally activated, non‑viral liposomal nanoparticle (LNP) platforms for in vivo precision genetic medicine. Our research focuses on light‑ and radiation‑triggered base and prime editing systems that correct disease‑causing mutations in rare pediatric and oncology indications. We integrate nanotechnology, CRISPR biology, and scalable, low‑cost manufacturing to create curative, accessible therapies. | EosGene seeks partners with complementary expertise in clinical translation, GMP manufacturing, regulatory strategy, and patient engagement for rare and pediatric genetic diseases. Ideal teaming partners include hospitals, CDMOs, advocacy groups, and data science organizations that share our mission to deliver safe, affordable (<$250 K/dose), in vivo gene‑editing therapies through spatiotemporally controlled LNP platforms. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Ajay Verma | Formation Venture Engineering Foundry, Inc. | ajay@formationve.com | Beverly, MA | FVE Foundry is an innovation-focused biotech venture studio. We aim to accelerate the development and derisking of next-generation therapies for brain disorders. Core strengths include integrated PK/PD, delivery technology engineering (Fc and protein conjugates) and biomarker strategies. We specialize in connecting discovery science with clinical application through scalable study design and a collaborative translational network. | We seek collaborators with de-risked drug assets (novel or clinical stage gene therapy, RNA or precision biologic candidates) for rare CNS & metabolic disease indications. Ideal partners already possess preclinical or IND/CTA-ready therapeutics that can benefit from PK optimization, multi-platform delivery testing, and in vivo validation in disease-relevant models. We value expertise in eactor manufacturing, regulatory stratgey, advanced biomarker platforms, and patient-centered rare CNS trials | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Deb Gouveia | EVERYONE MEDICINES INC | dgouveia@eomeds.com | Boston, MA | EveryONE Medicines (EOM) is the first company focused solely on individualized medicines, establishing a sustainable model for delivering customized therapeutics to patients at scale. EOM is modality agnostic, currently developing ASOs for children with CNS conditions under a master-protocol crossing diseases and mutations, catalyzing a shift to process approval and reimbursement. We believe individualized medicines should be accessible to everyone amenable so they never know life with disease. | EOM is looking for partners in building out a toolbox of different modalities to target different types of disease, as well as AI and technology partners to help make individualized medicines faster, better and cheaper. We are interested in teaming up with hospitals to help create a network of interventional genetics centers, and establish a new collaborative model for delivering precision therapeutics at scale. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development |
| Eszter Hars | Shwachman-Diamond Syndrome Alliance Inc | ehars@sdsalliance.org | Woburn, MA | We are a research-focused patient advocacy organization dedicated to enabling and accelerating the development of therapies for Shwachman-Diamond Syndrome (SDS). Our main focus is gene therapy to address the underlying cause of SDS, a recessive single-gene disorder caused by mutations in the SBDS gene, particularly by reducing or eliminating the risk of leukemia that originates in the bone marrow and HSCs. | We are looking for partners well-positioned for translational research and therapy development, so that we can support them with patient insights, regulatory engagement, other infrastructure needs, and disease models. | Module 3: Real-world Capabilities Pilots and Scaling, Module 1: Platform PGMs Design and Development |
| Patrick Thiaville | 4basebio | patrick.thiaville@4basebio.com | Cambridge, UK | 4basebio is biotechnology company pioneer the use of synthetic DNA to enable next-generation therapetuics and vaccines. Through its proprietary enzymatic DNA synthesis platform, 4basebio produces RUO, High Quality, and GMP grade sythentic DNA and mRNA with speed, purity, and scalability, overcoming the limitations of plasmid-based platforms. The company offers application specific DNA constructs tailored to the diverse needs of the gene therapies, genome editing, mRNA and DNA therapies. | 4basebio can partner with prospective teams on the development, optimization and manufacture of synthetic DNA. Additional services can be provided for the manufacture of mRNA as well as formulation services. | Module 1: Platform PGMs Design and Development |
| Keith Alkek | Scriptr Global | keith.alkek@scriptrglobal.com | Houston, Texas and Rochester, NY, TX | We are exploring two monogenic liver targeted, LNP delivered circular RNA and one rare cancer application for in vivo CAR-T. | Scriptr's technology is a novel backbone payload platform. Our RNA technology is a linear RNA that does not require a cap and tail. It self-circularizes in situ and is highly programmable. We have pre-clinical experience, and could benefit from novel targets, regulatory and clinical expertise. | Module 1: Platform PGMs Design and Development |
| Adarsha Koirala | AAVINUE INC. | adarsha@aavinue.com | Worcester, MA | AAVINUE Inc. is a gene therapy company developing non-viral gene therapy technologies (Delivery and Cargo) for rare diseases such as Stargardt’s disease, Usher Syndrome, Duchenne muscular dystrophy, ALS etc. Our Mini-nucleosome platform (protein-based vector system) can deliver nucleic acids (DNA, RNA and Oligos) to several cell types in the brain, retina, muscle and other organs. | Our company is looking for partners that have capabilities -in gene/base editing, -RNA modalities, -small animal surgeries to access hematopoietic stem cells (HSC), or other stem cells in animal models and are seeking in vivo gene modification to treat rare diseases partners,- ligand discovery platforms that can allow to us to expand the repertoire of our targeted gene delivery system to other tissue/organs affected in rare diseases | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Kasie Bailey | Truveta | kasieb@truveta.com | Seattle, WA | Truveta is the leading health data and analytics company built by 30 U.S. health systems spanning across the U.S representing 1 in 3 Americans through our platform housing over 120 million patients records. This platform is updated daily with new data including clinical notes, imaging and genomics from 900 hospitals and 20,000 clinics—Truveta delivers unmatched depth and quality for research, regulation, and innovation. | Any partner who needs real time RWE to define cohorts, train AI models, and generate real-world evidence faster and with full transparency. | Module 3: Real-world Capabilities Pilots and Scaling, Module 1: Platform PGMs Design and Development |
| Peter Seidler | Cognigenics | peter.seidler@cognigenics.io | San Francisco, CA | Cognigenics develops RNA-based therapeutics for neuropsychiatric, neurodegenerative, and rare CNS disorders through precise modulation of neural receptor and gene expression. Our iRNAi platform combines siRNA, shRNA, and CRISPR payloads with CNS-targeted AAV, LNP, and peptide-conjugate delivery via a non-invasive intranasal device, enabling localized, reversible gene modulation validated across 30+ preclinical studies. | Cognigenics seeks collaborators in RNA chemistry, nanoparticle formulation, regulatory science, and digital health to advance precision genetic medicines for the CNS. We welcome partners with expertise in CNS-targeted LNPs, conjugate and device optimization, IND-enabling modeling and safety, clinical translation in neurogenetics or rare disease, and digital biomarker tools for real-world outcome monitoring. | Module 1: Platform PGMs Design and Development |
| Uri Manor | University of California, San Diego | u1manor@ucsd.edu | La Jolla, CA | Our multidisciplinary team of scientists, patient- advocates, and industry partners is developing precision medicines for hereditary neuropathies. We are using CMT2A as our POC. We use patient-derived iPSC and rat models of CMT2A and deep learning-based imaging assays for phenotypic rescue of organelle dynamics and axonal health. We also provide expertise in siRNA/ASO (Yeo Lab), and CRISPR base-editor design (Komor Lab) for in vivo mutation correction, and SOTA mass spec and cryoEM cores. | Our team, led by UC San Diego, is building a comprehensive therapeutic "playbook" for hereditary neuropathies, starting with Charcot-Marie-Tooth type 2A (CMT2A). We have assembled a world-class group of experts and developed robust pre-clinical models (TA1 and TA3) to systematically de-risk and compare multiple therapeutic modalities in parallel. We are specifically looking for partners in: Advanced Delivery Systems Clinical Translation & Regulatory Expertise GMP Manufacturing and Scale-Up | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Sina Khorsandi | MusiQ Bio | sina@musiqbio.com | Dallas, TX | MusiQ Bio is developing MsQ, an ultrasound-triggered microbubble platform for in vivo delivery of genetic medicines to non-hepatic tissues. Our current focus is on precise, noninvasive delivery of gene therapies across the blood–brain barrier for treating hereditary neurodegenerative diseases, including Huntington’s disease. By combining cell-specific targeting with ultrasound activation, MsQ enables localized delivery while minimizing systemic exposure and off-target effects. | We are seeking partners with expertise in genetic medicine development, including gene therapy design and rare disease models, as well as partnerships with academic, clinical, or industry groups advancing in vivo delivery for hereditary diseases | Module 1: Platform PGMs Design and Development |
| Walter Acosta | SylamoreBio | wacosta@sylamorebio.com | Jonesboro, AR | SylamoreBio breakthrough receptor-independent delivery platform enables highly efficient transport of therapeutic modalities across biological barriers such as CNS. Our SyLEC delivery system is based on a natural plant lectin with high affinity to the human cell surface glycans driving tissue penetration and cell uptake. Proof-of-concept data has focused on rare lysosomal diseases demonstrating completely normalization of CNS pathologies in multiple mouse models, | Our platform technology can be applied to treat multiple rare disease conditions cause by defective soluble proteins. By expressing the lectin-fused product in the liver, we can enable delivery of the product to hard-to treat organs such as the brain. We are fully agnostic to the liver transduction method and can propose developing this platform using novel gene transfer modalities. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Marcelo Mendes | Anemocyte Srl - Gerenzano | mmendes@anemocyte.com | Insubrias BioPark, Via Roberto Lepetit, 34, 21040 Gerenzano VA, Italy | Anemocyte is an Italian Biotech Manufacturing Organization with a strong focus on the research, development, manufacturing, and analytical services and products to address needs in the field of: · pDNA – Plasmids (R&D, Hiqh-Quality, and GMP) for Viral Vector and mRNA; · mRNA – mRNA (R&D) for vaccines and therapeutic treatments. Our experience: 25 years of research, development and manufacturing experience (from R&D to GMP) in the field of Cell and Gene Therapies and Nucleic Acids. | Anemocyte is searching for team players in nucleic acid therapeutics as for example pDNA-based therapeutic developers who are approaching clinical phases using technologies such as viral vector technologies for cell & gene therapy. Moreover, Anemocyte is able to collaborate with companies and/or academic institutions that currently perform research and development activities in the areas of cell and gene therapy and genomic medicine using mRNA and/or pDNA. | Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Pleasantine Mill | LifeArc Centre for Rare Respiratory Diseases | pleasantine.mill@ed.ac.uk | Edinburgh | The LifeArc Centre for Rare Respiratory Diseases (est. 09/24) is a UK platform accelerating treatments for rare lung conditions. Co-created with patient groups, we're building accesible disease models and screening tools to fast track translational pipelies for much needed therapies and creating trial ready patient cohorts. Our initial focus : three high morbidity diseases affecting all ages- rare cystic lung disease, rare interstitial lung diseases and primary ciliary dyskinesia (PCD). | We are looking for individuals interested in novel delivery approaches to airway cells and interests in PCD or diseases of mucociliary clearance, as it is the exemplar we are targetting first with genetic therapies. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Tyona Pike | F&I Foresight Initiatives | Pikesforesightinitiatives@gmail.com | Cookeville, TN | Our research focuses on patient-centered care and access barriers for genetic and rare diseases, with expertise in longitudinal data collection, patient experience, and real-world outcomes. We also study care coordination, navigating complex genetic medicine pathways, and implementing distributed care models. | F&I Foresight Initiatives seeks partners developing genetic medicine platforms (Module 1), enabling early clinical access (Module 2), and implementing decentralized care (Module 3). We need collaborators with clinical operations expertise and real-world scalability know-how. We want partners committed to patient-centric design, equity, and affordability in developing and deploying new genetic therapies. | Module 3: Real-world Capabilities Pilots and Scaling |
| Eeshit Dhaval Vaishnav | Sequome | edv@sequome.com | South San Francisco, CA | Sequome designs biological sequences. | We would love to have the opportunity to learn from partners with clinical and regulatory expertise. | Module 1: Platform PGMs Design and Development |
| Jason Colquitt | Across Healthcare | jason@acrosshealthcare.com | Carrollon, GA | Matrix is a regulatory-grade data and coordination platform for rare disease. Current work: consented longitudinal capture; ePRO/eCOA; FHIR/EHR integration; imaging/device ingestion; safety/outcomes; decentralized hub/satellite workflows. We activate sites, curate RWD, and support 15-year follow-up—already partnering with 130+ rare patient advocacy groups on longitudinal natural history studies and industry-sponsored trials. | Seeking partners that need a proven innovative software technology to enable their decentralized workflows ready for interventional PGM care; hub/satellite; PIs/Coordinators; Patients/Caregivers; PAGs; etc. We provide the tech stack for site activation, collaboration, remote monitoring, EHR native data ingest, long-term surveillance, and follow-up—built to easily connect THRIVE’s data to the other ARPA-H programs. | Module 3: Real-world Capabilities Pilots and Scaling, Module 3: Real-world Capabilities Pilots and Scaling |
| Matthew Begemann | Confluence Genetics, LLC | mbegemann@confluence.ag | St. Louis, MO | Our team has developed a CRISPR-based platform (Cas-CLEAR) with a novel mode of action for cell elimination. The mode of action involves sequence specific targeting following by rapid non-specific DNA & RNA damage resulting in cytotoxicity. This platform is being developed for oncology applications. Our team has identified and validated (on-target/off-target) targets for multiple oncology indications using an in vitro assay. We are scaling into human cells lines with several delivery mechanisms. | We are experts in novel CRISPR technology development for health and industrial applications. We are looking for partners teams with expertise in delivery of gene therapy and oncology applications. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Joshua Welch | University of Michigan | welchjd@umich.edu | Ann Arbor, MI | Developing cell type specific enhancers using neural network models and single-cell data. High-throughput single-cell screening with human organoid models. Predicting effects of gene therapies using neural networks and single-cell data. | Looking for industry partners with expertise in delivering DNA-based gene therapies that may benefit from targeted enhancers for cell type selectivity. | Module 1: Platform PGMs Design and Development |
| Purvi Kunwar | Precision NeuroMed | pkunwar@precisionneuromed.com | Redwood City, CA | Precision NeuroMed is developing the Brain-Only Convection-Enhanced Delivery (CED) Platform, an integrated solution comprising a microcatheter infusion system and our “GPS for the brain” software for treatment planning using patient-specific MRI and catheter-based modeling. The Platform is designed to effectively deliver therapeutic drugs at high concentrations loco-regionally for CNS diseases in a reproducible, commercially viable system. | We are looking for partners developing innovative drug treatments in need of a drug delivery platform. The Brain-Only CED Platform bypasses the blood-brain barrier and will enable precision delivery of drugs (e.g., liposomes, nanoparticles, proteins). The Platform is designed to decrease time in the OR, enabling infusions in awake patients, mitigates issues inherent in currently available CED catheters (e.g., backflow, loss to CSF), and our software enables automated delivery planning. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| David Nelles | Tacit Therapeutics, Inc. | dnelles@tacittx.com | San Francisco, CA | Tacit Therapeutics has developed an RNA trans-splicing platform that rewrites large segments of RNA by harnessing the native spliceosome, enabling RNA repair without foreign nucleases. By sidestepping CRISPR/Cas-related immunogenicity and designing products that generalize across mutational spectra, we address economic and safety barriers to enable broad application of genetic medicines to both rare and common diseases. | we are looking for delivery technologies that can deliver RNA or DNA to the cellular nucleus | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Kan Cao | University of Maryland | kcao@umd.edu | College Park, MD | We focus on rare LMNA-related diseases (“laminopathies”). In progeria, we demonstrated that ABE gene correction via AAV delivery rescued animal lifespan, collaborating with partners at Harvard, NIH, and Brown. We are now expanding into LMNA-related congenital muscular dystrophy (LCMD), leveraging gene editing in stem cells/iPSCs, and have generated mutant-specific antibodies and related tools. We seek partners to advance these therapies. | We seek medical partners to advance our programs from preclinical (Module 1) to translational and clinical stages (Modules 2–3). We are looking for collaborators to assess off-target effects of gene editing tools, and for partners with FDA regulatory expertise to help guide therapeutic development and approval pathways. | Module 1: Platform PGMs Design and Development |
| Kevin Ess | University of Colorado/Children's Hospital Colorado | kevin.ess@childrenscolorado.org | Aurora, CO | The University of Colorado Anschutz Medical Campus and Colorado Children’s Hospital are well experienced in diagnosing and treating rare genetic disorders. Our group in particular is very focused on pediatric neurogenetic disorders. Our expertise and experience include brain malformations that cause pediatric epilepsy. We are also interested in the treatment of leukodystrophies that feature postnatal abnormalities in the development and function of white matter. | We are looking for potential partners who have expertise in the delivery of genetic therapies to the brain. Potential collaborators should be interested in partnering with a major children’s hospital to achieve the goal of developing and delivering neurogenetic therapies. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Amutha Boominathan | Lifespan Research Institute | amutha.boominathan@lifespanresearchinstitute.org | Mountain View, CA | Lifespan Research Institute’s (LRI) mission is to accelerate research and development of therapies that target the root causes of aging and age-associated diseases. The MitoREPAIR program at LRI focuses on developing gene therapies for mitochondrial DNA (mtDNA) disorders through "allotopic expression", the nuclear recoding and expression of mtDNA-encoded genes. Our goal is to enable therapeutic intervention for >250 rare pediatric and adult diseases linked to pathogenic variants in the mtDNA. | We are looking collaborators for the second module, Real-world capabilities Pilots and scaling to develop patient specific mtDNA mutation iPSC or organoid models to test the efficacy of our gene therapies. This is a crucial tool to test the success of these personalized therapies and an unmet need in the field. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Derese Getnet | Simmbion | dgetnet@simmbion.org | Baltimore, MD | Simmbion is pioneering a trypanosome-based "living medicine" platform designed to transform the delivery of biologics through a single annual injection that enables continuous, in vivo production of therapeutic proteins, dramatically improving adherence, durability, reversibility, cold chain free of treatment, and cost-effectiveness. with support from NSF and FDA Interact, Simmibion has concurrences on two-study path to BLA. | Simmbion is advancing a first-in-class living medicine platform (Simmbion) and is actively seeking partnerships with innovators in the cell-free protein expression and delivery technologies. comparative studies will enable us to benchmark performance, durability, safety, and scalability to highlight the unique advantages of Simmbions. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Harsha K Rajasimha, MS, Ph.D. | JEEVA CLINICAL TRIALS INC | harsha@jeevatrials.com | MANASSAS, VA | Jeeva Clinical Trials is reimagining the infrastructure to enable end-to-end clinical trial process automation with Agentic AI. By bringing traditionally siloed activities such as patient recruitment, data management, trial scheduling, and digital patient engagement, all in a seamless, user-friendly platform built for efficiency for orphan drug development, patient registries at regulatory grade quality process 200% faster, 300% less manual effort, and transparent per patient per month pricing. | Clinical researchers or patient organizations conducting clinical research, natural history studies, patient registries, or therapy development for rare diseases. Planning a small population or large population study Observational or interventional clinical study Industry sponsored or investigator initiated trials Regulated Service providers seeking efficiency of clinical research with AI Agents Those seeking to innovate patient-centric clinical trial execution faster, cheaper, better. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Peter Weinstein | Circurna, Inc. | peter@circurna.com | Houston, TX | Circurna is developing circular RNA therapeutics to treat different diseases, including rare diseases. Circurna's circular RNA is a platform technology that allows for its use to express different proteins or act for CRISPR to treat different diseases. It is low cost and allows for quick development of therapeutics to get them into human clinical trials quickly. | Circurna is looking for teaming partners with specific expertise with regard to a specific rare disease genetic mutation disorder. Circurna can design and create the circular RNA with CRISPR technology to treat the disorder but assistance in identifying the specific genetic mutation would be helpful. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Jay Schneider | Mayo Clinic Florida | schneider.jay1@mayo.edu | Jacksonville, FL | We've created a gene-edited pig recapitulating mutation in the RNA binding protein RBM20, which cause rare but lethal form of inherited cardiomyopathy, and now would like to develop CRISPR based strategy to repair this mutation in vivo directly in the heart. | We will combine a number of specialist investigators within Mayo Clinic and perhaps collaborate with UT Southwestern (Dr. Eric Olson) and with the company that generated and maintains the gene edited pig (Recombinetics). | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| David Raiser | STRM.BIO | dmr@strm.bio | Cambridge, MA | STRM.BIO has developed a novel non-viral modality for selective delivery of genetic cargo to the bone marrow. The company is focused on and in vivo engineering of bone marrow resident cell populations and therapeutics development for rare genetic blood diseases. | STRM.BIO is seeking teaming partners with (1) editing technologies, especially those that are not amenable to viral delivery; (2) other proprietary payloads for which targeted delivery to the bone marrow would be advantageous; and (3) clinical trial design expertise. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Miguel Sena-Esteves | UMass Chan Medical School | heather.grayedwards@umassmed.edu | Worcester, MA | 1. AAV Gene Therapy Development: Maintaining a robust pipeline of 50 programs including CRISPR based approaches. 2. Comprehensive regulatory support. 3. Integrated manufacturing pipeline: from our mid-stage facility, which produces affordable phase I/II compatible AAV lots (costing $30,000 per dose), all the way to a GMP facility positioned for commercialization of FDA approved drugs. 4. Internal toxicology unit. 5. Real time immune response monitoring informing and guiding clinical decisions. | 1) Manufacturing network development to establish partners who can deploy our standardized manufacturing process across the U.S., especially rural areas. 2)Partnerships with new platforms that are ready for clinical translation. 3)Biomarker discovery 4)Ultra rare clinical trial design 5)Artificial Intelligence /Machine Learning for remove clinical trail follow up, especially MRI. 6) Remote clinical trial infrastructure using wearables and remote clinical testing for tracking patient outcomes. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Lukas Lange | Probably Genetic | lukas@probablygenetic.com | San Francisco, CA | Probably Genetic's platform can power module 3: we identify rare disease patients by combining AI phenotyping, at-home genetic testing, and a built-in nationwide network of physicians and genetic counselors. Our platform enables rapid rollouts of clinical trials and treatments at national scale for the >230k patients using our service. | We are looking to partner with teams servicing modules 1 and 2. | Module 3: Real-world Capabilities Pilots and Scaling |
| Fyodor Urnov | UC Berkeley | urnov@berkeley.edu | Berkeley, CA | Developing and clinically deploying innovative platform-based approaches for CRISPR-on-demand therapies for rare pediatric and adult disease. | The IGI welcomes any and all suggestions for collaboration in this space. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Gabe Meister | Battelle | meisterg@battelle.org | Columbus, OH | Battelle is the world's largest independent non-profit R&D organization. Our mission is to translate scientific discoveries into societal impact. Battelle has invested in the development of an innovative polymeric nanoparticle technology to enable delivery of genetic medicines. Our delivery technology is produced via a cell free process, is highly tunable, and has demonstrated delivery of all types of nucleic acid therapeutic modalities, proteins, and small molecules. | Battelle provides delivery technologies to support the ARPA-H THRIVE program. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Arkady Hagopian | Syntropy Technologies, LLC | arkady.hagopian@emdgroup.com | Cambridge, MA | Syntropy Technologies, a joint venture between EMD Digital and Palantir Technologies, focuses on deploying secure healthcare data platforms that integrate, harmonize, and enable real-time collaboration across diverse biomedical datasets. Our research includes advancing privacy-preserving AI/ML analytics, supporting distributed rare disease research, and building infrastructure for scalable, regulatory-grade data sharing in precision medicine. | Syntropy Technologies is seeking teaming partners with complementary expertise spanning precision genetic medicine development, clinical research, rare disease patient advocacy, and regulatory strategy who are committed to open data sharing, multidisciplinary problem-solving, and building scalable models that align with THRIVE’s goals. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Rebecca Ihrie | University of Colorado | rebecca.ihrie@cuanschutz.edu | Aurora, CO | Single cell studies of patient iPSC-derived models of brain development and intraoperative brain tissue specimens | We are looking for partners with potential editing delivery mechanisms for neurodevelopmental disorders who are interested in partnering with a major children's hospital and research center. | Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development |
| Tracey Sikora | NORD | tsikora@rarediseases.org | Washington, DC | NORD is the leading national rare disease patient advocacy organization with over 40 years of trusted leadership. In addition to serving as the voice of the rare disease community, we bring strong rare disease research expertise and a network of leading rare disease clinician-investigators for rapid patient identification and/or treatment. Our strengths in patient engagement, governance, recruitment, and data integrity ensure research that is both rigorous and deeply patient-informed. | We seek partners with strong precision genetic medicine platforms and commitment to multi-disease strategies. Ideal partners work transparently to integrate patient perspectives into trial design, endpoints, and scaling models. We value partners committed to building sustainable clinical trial networks, cost-effective manufacturing, and long-term access for rare disease patients. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| James Wilson | GEMMABio | jim.wilson@gemmabiotx.com | Philadelphia, PA | GEMMABio is a clinical stage rare disease genetic medicines company based on the Wilson lab at Penn. Our focus is the development and commercialization of ultra-orphan gene therapy/editing products. We can support all Modules of THRIVE although our deep experience with rare diseases and our track record of standing up successful first in human studies well positions us to partner via Module 2. Our partnerships in LATAM and the UAE can serve as hub and spoke models for Module 3 activities. | We are looking for a partner who is developing innovative editing technologies for treatment of loss of function monogenic disorders that are mutation specific or mutation agnostic (e.g., site specific integration). We envision a collaboration where the editing group takes the lead for Module 1 and GEMMABio leads Modules 2 and 3. We are also looking for a partner with access to innovative delivery technologies although GEMMABio can oversee work with a CDMO on scale-up and GMP production. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Amanda Powell | SRI International | amanda.powell@sri.com | Harrisonburg, VA | We are a highly-targeted therapeutic delivery company capable of identifying molecular guidance systems (MGS) capable of delivering any cargo intracellularly to any cell/tissue type of interest. Due to the highly specific and internalizing nature of our MGSs, off-target effects are minimized. | We are looking for partners capable of spearheading modules 2 and 3 to identify and establish long-term clinical care and support. | Module 1: Platform PGMs Design and Development |
| Juan Harrison | Harrington Discovery Institute | juan.harrison@harringtondiscovery.org | Cleveland, OH | Harrington is a drug discovery institute that supports academic institutions across the US, UK and Canada. It provides grantees financial grant and industry-level translational, development, commercial leadership. The Oxford-Harrington Center is dedicated to rare disease drug discovery (up to clinical trial testing) but also seeks to find systemic solutions to the economic, policy and procedural challenges faced by rare disease drug discovery. | A mission-driven enterprise that bridges academic discovery to commercial development and patient access, we partner with academia to source projects, industry to access resources and expertise, service providers to support projects, venture finance to start companies, philanthropy to fund projects. Our strategy is partnership and we have successfully operated as a translational hub since 2012. Seeking partners across the spectrum of discovery to achieve our mission as effectively as possible. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Roger Hajjar | Massachusetts General Brigham | rhajjar@mgb.org | Cambridge, MA | The mission of the GCTI is to bring together the community of MGB investigators who can utilize gene and cell therapy products and platforms to achieve their scientific and clinical goals to improve the health of patients. The Institute will differentiate itself by harnessing the exceptional strengths of clinician scientists, the large and unique patient population, and major discoveries throughout the hospital system to propel MGB as the leader in gene and cell therapies | GCTI supports preclinical-stage research to launch new clinical studies of these treatments, drawing on the unsurpassed expertise in clinical trials design and execution and the exceptional clinical experience and breadth that make Mass General Brigham a national leader in this domain. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Taleen Afeyan | Alltrna | tafeyan@alltrna.com | Cambridge, MA | Alltrna has developed an engineered tRNA platform uniquely positioned to treat multiple rare diseases sharing the same nonsense mutation with a single therapeutic. Our clinic-ready lead program AP003 uses LNP delivery to target a basket of inborn errors of metabolism, restoring full-length functional proteins. Current capabilities include GMP manufacturing, established disease clinical and patient networks, and the ability to rapidly expand to additional diseases, mutations or tissues. | We seek partners with clinically validated PGM platforms, particularly ASOs or other established oligonucleotide therapeutics, to complement our engineered tRNA approach. Additionally seeking: clinical trial organizations with satellite site networks for distributed care; embedded patient advocacy groups with genetic disease expertise; regulatory specialists interested in platform approvals; and next-generation delivery technologies for expansion beyond liver. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Dusan Bogunovic | Columbia University | db3700@cumc.columbia.edu | New York, NY | We design new PGMs and have population to test it in. | Help with scaling. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| C. Anthony Altar, PhD | Splice Therapeutics | tony@splice.one | Germantown`, MD | Splice Therapeutics current research includes optimizing lead RTMs that correct the Huntington's disease gene by replacing mutant exon 1 with a wild-type exon 1. We have a high-throughput discovery platform that is yielding RTMs that correct most mutations in two neurodevelopmental genes, in collaboration with a pharmaceutical partner. We also are creating RTMs for transient expression to kill cancer or viral infected cells, and for molecular imaging. | 1) Bioinformatics support to develop in silico tools to enhance our algorithms for predicting optimal RNA targets for RTM engagement. This would be evaluated by our high-throughput RTM screen for two or more ongoing projects. 2) Partners with DNA or RNA delivery technologies. RTMs need only ~200 nucleotides for activity, provide additional specificity beyond that provided by the delivery and promoter, and can use almost every gene delivery method. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Ali Madani | Profluent | ali@profluent.bio | Emeryville, CA | Born out of an AI research lab, Profluent develops foundation models at the frontier of AI and protein design. Our primary focus is the design of bespoke gene editors with optimal properties. We have demonstrated the first usage of AI to generate novel CRISPR-Cas complexes, such as OpenCRISPR, in addition to deaminases, RTs, and other effectors for large insertion. We invest heavily in both the AI research and wet lab techniques to enable a scalable path for on-demand tailored genomic medicines. | Our team includes top AI researchers from tech (Amazon, Salesforce, Meta) and leading scientists from biotech (Caribou, Editas, GSK). Collectively, we have deep expertise in AI-driven protein design, gene editing, and high throughput screening. We seek partners who share our vision and add strengths in scalable non-viral delivery, CMC, regulatory affairs, clinical translation and rare disease. | Module 1: Platform PGMs Design and Development |
| Julie Douville | n-Lorem Foundation | julie.douville@nlorem.org | Carlsbad, CA | n-Lorem's regulatory team has achieved regulatory authorizations for 35 INDs for >40 patients to be treated with individualized ASOs. In line with the FDA Guidance for Individualized ASO Treatments, we have successfully navigated 4 divisions of the FDA and enabled acceleration of patient treatment. A critical component of our regulatory expertise is underpinned by nonclinical & chemistry experience with ASOs ensuring patients are only exposed to prudent risk when treated with an optimal ASO. | n-Lorem is looking to be a part of a team committed to finding solutions for patients who are left behind. A team that leverages the cutting-edge science and technology tools we have today to push the envelope of what was once considered impossible. As a non-profit, n-Lorem is focused on and dedicated to producing only the best possible ASOs as patient safety underpins our work. Our team would share these values, be committed to working rapidly, all with patient safety as top priority. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Timothy Day | Particella | timday@particella.bio | San Francisco, CA | Particella is therapeutics company that has developed a proprietary oral lipid nanoparticle technology, enabling the delivery of mRNA to gastrointestinal epithelial cells. This innovation provides access to the intestine for RNA medicines. Particella is focused on gut-targeted protein therapeutics that offer an oral product profile and demonstrate enhanced safely and efficacy. The mechanisms enabled by Particella allows for strong advantages to treat rare and fatal pediatric diarrheal diseases. | We are looking for partners with innovative gene insertion and gene editing technologies to complement our advanced oral LNP delivery platform for targeting gastrointestinal cells. Ideal collaborators bring programmable editors, insertion tools, or modulators that expand therapeutic scope to target and treat rare and fatal pediatric diarrheal diseases. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Shalin Naik | WEHI | naik.s@wehi.edu.au | WEHI, Melbourne, Australia. 3052 | CELECT-HSC: A universal “correct and select” gene therapy platform to cure all monogenic blood and immune diseases. This includes 1) a multi-lineage haematopoiesis in vitro patient avatar to test personalised pegRNAs for mutation correction, genotoxicity, and functional correction, 2) addition of a proprietary edit that adds a selective resistance to commonly used drugs, enabling in vivo competitive advantage of the corrected gene edited hematopoietic stem cell. | Seeking partners who are specialised in rapid turaround of pegRNA and PE encoding LNPs, and HSC targeting technologies. Clinical expertise in implementing therapies for bone marrow failure, auto-inflammatory diseases, and immune deficiencies. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Pengfei Liu | Baylor College of Medicine | pengfeil@bcm.edu | Houston, TX | We operate a CAP-CLIA–approved clinical diagnostic lab providing RNA sequencing for the Undiagnosed Diseases Network and other partners. Our research focuses on advancing clinical implementation of emerging technologies to improve patient care. We have conducted pre-clinical studies developing antisense oligonucleotide therapies. Our mission is to integrate a therapy recommendation system in the current diagnostic workflow, connecting precision diagnostics with personalized therapeutics. | We seek partners with pharmaceutical expertise and regulatory insight to co-develop and validate our therapeutic recommendation and screening system. Such collaborations will ensure our pipeline addresses real-world needs, advances effectively through development, and is well positioned for clinical adoption. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Han Xiao | RICE UNIVERSITY | han.xiao@rice.edu | Houston, TX | Our lab focuses on developing tissue-specific delivery platforms for small molecules, proteins, and cell-based therapeutics. By integrating chemical biology, protein engineering, and nanotechnology, we create strategies to enhance drug stability, targeting, and efficacy while reducing off-target effects. Our goal is to enable precise, safe, and effective therapies that advance precision medicine and improve patient outcomes. | We are seeking collaborators who can provide models of rare genetic diseases to advance our work in tissue-specific delivery of small molecules, proteins, and cell-based therapies. By combining our delivery platforms with disease-relevant models, we aim to accelerate the development of precision medicines and broaden therapeutic options for patients with unmet medical needs. | Module 1: Platform PGMs Design and Development |
| Joseph Ruiz | Epigenos Biosciences | jruiz@epigenos.com | Chapel Hill, NC | While AAV-based vectors have shown promise as platforms for rare disease therapy, gene expression is non-reversible and inflexible, which creates risk in sensitive tissues and limits therapeutic design. Epigenos has developed CEMTrol, an epigenetic-based control system that builds tunability into the vector itself, which enables dose-dependent control after gene delivery using a small molecule with favorable pharmacokinetics that can be incorporated into any gene therapy vector of choice. | Epigenos’ CEMTrol system is versatile and vector agnostic. We seek teaming partners who need to solve any of the following limitations inherent with current gene therapy modalities that negatively impact their rare disease gene therapeutic programs: (a) lack of precision control; (b) gene silencing over time; (c) possibility of reducing AAV viral load required for treatment (if AAV-based therapies are used); and (d) regulatory appeal due to improved safety and reduced risk of adverse effects. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Erik Aznauryan | HarborSite | eaznauryan@harborsite.com | Cambridge, MA | HarborSite develops engineered tyrosine site-specific recombinases for the insertion of multi-gene cassettes into novel genomic safe harbors with high efficiency and specificity. We identified key amino acid positions controlling recombinase DNA recognition and showed that precise residue changes predictably alter recombinase specificity. Our platform has demonstrated full-gene insertion in both cell lines and primary human cells, and we are currently conducting in vivo validation in mice. | Our engineered recombinases preferentially operate on double-stranded circular DNA templates. We seek collaborators developing novel non-viral tools for targeted and efficient in vivo delivery of multi-kb DNA molecules to hepatocytes, lung and gut epithelium, and/or other tissues, while overcoming key barriers of immunogenicity and nuclear entry. Additionally, exploring partnerships with physicians, clinics, and patient advocacy groups working with patients with inborn errors of metabolism. | Module 1: Platform PGMs Design and Development |
| Saumya Jamuar | KK Women's and Children's Hospital | gmssaum@nus.edu.sg | 100 Bukit Timah Road, Singapore 229899 SINGAPORE | At KK Women's and Children's Hospital, we are committed to advancing medical knowledge, providing exceptional patient care, and supporting innovative research for people living with rare diseases. As the Lead Hospital of the Singapore Childhood Undiagnosed Disease Programme, we have spearheaded ground-breaking studies that combine deep phenotyping, genomic sequencing, and advanced bioinformatic analysis to uncover the genetic causes of rare and undiagnosed diseases. | We are looking for partners with background on preclinical and early clinical development of therapeutics. | Module 3: Real-world Capabilities Pilots and Scaling, Module 1: Platform PGMs Design and Development |
| Kieren Patel | B-Logic Therapeutics | kieren.j.patel@gmail.com | St. Loius, MO | B-Logic is pioneering in-vivo genetic medicines using VISTA™, an advanced adenovirus platform with unmatched payload capacity (up to 35 kb) for complex gene editing, replacement, and multi-gene circuits—capabilities no other system can achieve. Our programs span in-vivo CAR-T and autoimmune therapies, rare disease gene correction, HIV-neutralizing antibodies, and engineered B-cells producing complex biologics. | B-Logic is seeking partners with advanced gene editing tools (CRISPR, base/prime editors), tissue-specific targeting ligands, and validated therapeutic constructs that can be integrated onto our high-capacity adenoviral VISTA™ platform. We aim to collaborate with experts in synthetic biology, disease model validation, and translational biology to accelerate novel in-vivo therapies where payload size or complexity has been limited with other delivery systems. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Brent Stead | Specific Biologics Inc. | bstead@specificbiologics.com | Toronto, Canada | We are a venture-backed preclinical genome editing company developing our Dualase genome editors for seamless site-specific sequence removal and repair of both small and large sequences in vivo. | We seek potential partners for novel delivery technologies and clinical therapeutic area expertise in several disease areas. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Paul Kruszka | University of Virginia | pk3e@UVAHealth.org | Charlottesville, VA | UVA offers: (1) a high-volume clinical genetics program with access to diverse rare disease populations; (2) extensive experience conducting and hosting industry gene therapy trials; (3) strong translational infrastructure including IRB/IND expertise, biobanking, and regulatory support; and (4) a growing biotechnology institute with GMP and vector development capabilities. | Gene editing partners to create in vivo base editing and prime editing for rare pediatric genetic diseases. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Andrew Longenecker | PBD Project | research@pbdproject.org | Bethesda, MD | PBD Project is a nonprofit patient advocacy group and biotech incubator dedicated to unlocking the power of the peroxisome. We are focused on a broad set of diseases caused by peroxisome dysfunction, including Peroxisome Biogenesis Disorders, have built a strong network of patients and researchers, and have developed many assets to facilitate research (e.g., cell lines, animal models, patient data and network). | PBD Project would like to partner with a Program Team Lead (PTL) and provide resources to the team including research assets (e.g., patient cell lines, animal models), a strong network of disease experts and clinicians, and active patient community. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Ruby Tsai | Applied Stemcell Inc. | ruby.tsai@appliedstemcell.com | Milpitas, CA | We have developed integrase-based genome engineering technology named S-Select that enables site-specific, efficient, large cargo insertion into a safe harbor locus. S-Select provides a platform technology that corrects all mutations in a singe or multiple genes with negligible off-target risks. This aligns with the goals of THRIVE. | Novel delivery tools/technology to delivery large plasmids / RNP. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Khaled Younis | MedAiConsult | kh.younis@gmail.com | Cleveland, OH | MedAiConsult is a Small Enterprise (SME) advancing AI/ML in healthcare across imaging, genomics, and clinical decision support. Current work focuses on multimodal data integration, federated learning, and standards development through leadership of AI, Research, or Tools committees at SIIM, ISO, HL7, and IHE. We evaluate, fine-tune, and publish on multimodal foundation models, bridging technical innovation with standards to accelerate safe AI translation into practice. linkedin.com/in/dryounis | We seek complementary partners with expertise in wet-lab biology, clinical trial operations, and health data platforms to pair with our AI/ML and standards strengths. Ideal partners bring domain depth in genomics, molecular biology, or health systems integration to co-develop THRIVE modules. We value groups with translational capacity, regulatory readiness, and ability to scale solutions across diverse healthcare settings. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |
| Tim Yu | N=1 Collaborative | info@n1collaborative.org | Boston, MA | N1C unites stakeholders across academia, biopharma, and patient groups to advance precision genetic medicines for ultra-rare diseases. As the coordinating hub for development and delivery of such treatments, we centralize expertise and resources, and translate them into standards, tools, and frameworks to transform proof-of-concept efforts into repeatable care pathways. In doing so, we enable broader access, foster shared learning, and build the capacity needed to drive progress at scale. | We seek teams developing precision genetic medicine platforms, delivery tools, or enabling services who can leverage our shared translational backbone. Partners will benefit from access to expert support in N=1 protocol design and to preclinical and clinical data-sharing platforms built with collaborators including The Jackson Laboratory and C-Path, managed with community governance. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Madeleine Braun | The Jackson Laboratory | madeleine.braun@jax.org | Bar Harbor, ME | The Rare Disease Translational Center at JAX works with patient foundations, researchers and industry partners to bring therapeutics to the clinic. We provide a full pre-clinical pipeline, both in vitro and in vivo, for assessing target engagement, efficacy & safety, using mouse models, engineered & patient iPSCs, and other cellular systems while integrating ML to cut costs and animal use. We also design databases for open-access, community-generated preclinical datasets. | The Jax Rare Disease Translational Center is seeking partners who are developing therapeutics and delivery systems who can benefit from our preclinical pipeline that includes both in vivo and in vitro analysis. Our Center is 75+ individuals dedicated to rare, with quality systems and skills to support IND enabling studies. As members of the N1C, the Somatic Cell Genome Editing consortium and large NIH programs we leverage our capabilities at JAX to support and co-develop therapeutics. | Module 1: Platform PGMs Design and Development |
| Scot Wolfe | UMass Chan Medical School | scot.wolfe@umassmed.edu | Worcester, MA | We have expertise in gene editing platform development through both effector and guide engineering. We are developing a strategy called prime assembly (PA) that can correct or insert multiple kilobases without DSBs, recombinases, or HDR. A single PA-based intervention could correct large numbers of distinct pathogenic mutations. In addition, we are enhancing the activity of base and prime editing platforms through guide/template engineering to achieve enhanced in vivo activity and precision. | We are looking for teaming partners with expertise in nonviral delivery platform development, especially to the CNS, as well as implementation in specific diseases, and the design and execution of clinical trials. We also need partners to participate in (or help us develop a strategy to meet) the cost-sharing requirements of the THRIVE program. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Laurence Mignon | n-Lorem Foundation | laury.mignon@nlorem.org | Carlsbad, CA | n-Lorem is pioneering individualized cross-over trial designs that provide potentially cost- and time-effective framework for evaluating individualized ASO treatment in a trial of 1 to few. This innovative design enables robust data collection and comparing baseline to on-treatment for patients, a plus for conditions with limited natural history. We have been successful in this approach and have many ongoing clinical studies with >30 active patients on treatment with more to start soon. | n-Lorem is looking to be a part of a team committed to finding solutions for patients who are left behind. A team that leverages the cutting-edge science and technology tools we have today to push the envelope of what was once considered impossible. As a non-profit, n-Lorem is focused on and dedicated to producing only the best possible ASOs as patient safety underpins our work. Our team would share these values, be committed to working rapidly, all with patient safety as top priority. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Vrad Levering | Triple Ring Technologies | vlevering@tripleringtech.com | Newark, CA | Triple Ring Technologies is a leading partner in developing science-driven products across medtech and life sciences. Our interdisciplinary team, including many PhDs, excels in advancing technologies to FDA approval while collaborating with academic researchers. We have engaged with ARPA-H, both as subcontractors and primary awardees. We offer services for development of devices such as surgical tools to aid in cell delivery, assay development, and implantable sensors. ISO 13485 certified. | We partner with innovators to solve tough problems and create new businesses. From concept to FDA submission and commercialization, we handle technology development and redesign, as well as complex system integration. We are looking for teaming partners that could use our collaborative assistance to design enabling devices and technologies while navigating the FDA regulatory pathway. We have acted as primary, subcontractor, or vendor on previous submissions. | Module 3: Real-world Capabilities Pilots and Scaling |
| Pietro Genovese | Boston Children's Hospital | pietro.genovese@childrens.harvard.edu | Boston, MA | Boston Children’s Hospital leads translational research in gene correction for rare blood and immune disorders, including immunodeficiencies and bone marrow failure. Our team applies base and prime editing in T cells and HSCs, targeting diseases with low therapeutic thresholds. With proven success in first-in-human gene therapy trials, we aim to deliver safe, one-time treatments for lasting impact in pediatric and young adult patients. | We are seeking partners with expertise in manufacturing GMP-grade gene editing reagents, including mRNA, guide RNA (gRNA), and lipid nanoparticles (LNPs). Ideal collaborators will support clinical translation of base and prime editing technologies through high-quality, scalable production aligned with regulatory standards for first-in-human trials in rare immune and hematologic disorders. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Natsumi Takahashi-Vitiello | Foundation Fighting Blindness | ntakahashi-vitiello@fightingblindness.org | Columbia, MD | The Foundation Fighting Blindness (FFB) powers rare disease research and patient advocacy with unmatched expertise in inherited retinal diseases. Serving a multi-site team hub, FFB provides program management and strategic oversight, aligning milestones, managing risks, and ensuring regulatory and data integrity. Also, with expert ARPA-H program experience and strong stakeholder engagement, we drive scientific rigor and accelerate solutions toward commercialization and patient impact. | The Foundation Fighting Blindness (FFB) seeks strategic partners for specialized expertise in pre-clinical sciences, therapeutic engineering, bioinformatics, regulatory harmonization, and commercialization. These collaborations ensure access to cutting-edge capabilities while FFB drives patient engagement and advocacy, alignment, rigor, and impact across the programming. | Module 3: Real-world Capabilities Pilots and Scaling, Module 2: Investigational Medicine |
| Nick Hughes | Acrobat Genomics | nick.hughes@acrobatgenomics.com | San Francisco Bay Area, CA | Development of optimized gene editing systems for efficient in vivo delivery and editing tunability. | Complementary delivery technology, manufacturing capabilities, and connection to patient advocacy groups. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Alessandro Padova | Holostem srl | a.padova@holostem.com | Modena, Italy | Holostem srl is an italian biotech focused on developing cell and gene therapeutics for epithelial-related diseases. Holostem has developed Holoclar, an ex vivo autologous stem cell based therapy for patients affected by limbal stem cell deficiency. Current clinical pipeline is dedicated to development of stem cell based gene therapies for patients affected by junctional or dystrophic Epidermolysis Bullosa, a rare inherited disease. Holostem runs a fully approved GMP facility. | Interested in partnering with teaming partners with expertise in in-vivo delivery, tissue engineering and automation. | Module 2: Investigational Medicine, Module 2: Investigational Medicine |
| Thomas Page | KOMO Biosciences | thomas.page@komobiosciences.com | College Station, TX | Our research centers on hyperactive integrases, enzymes that deliver large DNA payloads into specific genomic sites at record-breaking efficiencies. Unlike nuclease-based methods, our approach avoids double-strand breaks, enabling safe and precise insertion of therapeutic genes. Our reagents are non-viral, are DNA-based, and affordable. By overcoming efficiency and payload size limits, we aim to enable cures across diverse genetic disorders and set a new standard for next-generation therapies. | We seek partners across complementary domains: innovators in non-viral DNA/RNA delivery (LNPs, polymer/protein nanoparticles); disease-area leaders to define endpoints and run fit-for-purpose assays; machine learning-driven design, automation, and QC; regulatory/clinical operations; patient engagement; scalable cell-free/synthetic biomanufacturing to cut COGS and enable platform INDs; off-target standards/assay developers; hub-and-spoke trial sites. | Module 1: Platform PGMs Design and Development, Module 2: Investigational Medicine |
| Douglas Martin | Auburn University | martidr@auburn.edu | Auburn, AL | Molecular therapy of genetic and infectious diseases. We are experienced in genetic disease modeling and initial stages of therapy development. The lab has focused primarily on lysosomal storage disorders but is interested in other neurodegenerative diseases that can be treated with molecular and/or cellular approaches. | We are especially interested in partnering with those experienced in -omics analysis, interactions with regulatory bodies such as FDA, and/or clinical trials. | Module 2: Investigational Medicine, Module 3: Real-world Capabilities Pilots and Scaling |
| Daniel Bauer | Boston Children's Hospital | daniel.bauer@childrens.harvard.edu | Boston, MA | We develop strategies for therapeutic gene editing of hematopoietic stem cells to treat blood and immune disorders. We focus on severe inherited diseases affecting children and young adults where edited cells would outcompete unedited cells. We conduct studies ranging from preclinical proof-of-concept to IND-enabling to first-in-human trials. Our scientific team focuses on both technology and biology, includes disease expert physicians, and works closely with patient advocacy groups. | Non-viral in vivo delivery platforms that target HSCs. | Module 2: Investigational Medicine, Module 1: Platform PGMs Design and Development |
| Wayne Doyle | Eclipsebio | wayne.doyle@eclipsebio.com | San Diego, CA | Eclipsebio develops AI-powered support for RNA therapeutic design, enabling faster and more effective optimization of drug candidates. These insights are validated with next-generation sequencing assays that deliver high-resolution QC and characterization, replacing outdated methods. Together, our AI and sequencing solutions accelerate RNA-based medicine development from discovery to clinical translation. | Eclipsebio seeks partners who share our vision of accelerating RNA medicine through integrated innovation. We provide AI-driven design and sequencing-based validation, ensuring therapies are optimized and rigorously characterized. We look for collaborators in clinical translation, delivery, manufacturing, or regulatory areas to complement these strengths, enabling end-to-end solutions that shorten timelines and improve reliability. | Module 1: Platform PGMs Design and Development, Module 3: Real-world Capabilities Pilots and Scaling |