ARPA-H launches groundbreaking program to develop affordable precision genetic medicines for all Americans 

Program aims to prevent rare and common diseases with individualized targeted therapies 

The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services (HHS), today announced a new research and development opportunity through its Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) program. THRIVE intends to develop integrated platform technologies to accelerate precision genetic medicines (PGMs) and provide single-intervention precision treatments to slow, reverse, or prevent diseases at the genetic level. The program is designed to optimize affordability, scalability, and sustainability of lifesaving PGMs for patients through existing regional treatment centers and virtual clinics. This will allow patients to be seen and treated where they live. 

Chronic genetic conditions often progress rapidly, cause severe disability, and result in poor quality or significant loss of life. Most urgent are rare diseases, which collectively affect over 30 million Americans, mostly newborns, infants, and children. Despite their high unmet needs, roughly 95% of rare diseases have no approved treatments. Lifetime medical and other costs of rare diseases can be substantial and create financial hardship for families. 

“Revolutionary genetic engineering tools are poised to transform the outlook for patients with genetic diseases and shift the medical paradigm,” said Jason Roos, Ph.D., ARPA-H Acting Director. “By transforming precision genetic medicines into universal, widely available cures, THRIVE exemplifies the unique health care approach needed to slow, reverse, or prevent genetic diseases for all Americans, solidifying the U.S. as the leader in realizing advanced medical treatments.”  

THRIVE seeks to accelerate technical, translational, clinical, and associated regulation of integrated component genetic tools, including but not limited to therapeutic gene corrective tools, and preferential, cell-targeted delivery of those tools. If successful, THRIVE will provide options for early, one-time interventions with PGMs to address diseases at the underlying genetic level.  

"At ARPA-H, our vision is to empower every individual not just to survive, but to thrive. By developing pioneering interventions to halt, reverse, or prevent chronic diseases at the genetic level we aim to transform the possibilities for better health outcomes and improved life expectancies,” said Mimi Lee, M.D., Ph.D., THRIVE Program Manager. “THRIVE is designed for these precision genetic medicines to be distributed through existing regional treatment centers and virtual clinics for an affordable and seamless patient experience.”  

Through an Innovative Solutions Opening (ISO) solicitation, THRIVE invites proposals across three modules: design and development of platforms to enable rapid iteration of affordable PGMs, investigational medicine, and real-world viability pilots and scaling. Proposals are expected to use innovative approaches to develop solutions that accelerate affordable precision genetic medicines and demonstrate their capabilities in real-world settings. The program will not consider proposals that include gene supplementation therapy or ex vivo approaches. 

ARPA-H anticipates that teaming will be necessary to achieve the goals of THRIVE. Prospective proposers are encouraged to form teams with varied technical and operational expertise from geographically dispersed private and public entities to submit a research proposal. Other Transactions Agreements (OTAs) (not procurement contracts, grants, or cooperative agreements) under this ISO are anticipated. Investments will depend on the quality of the proposals received.  

Learn more about THRIVE on its program page, including information about the solicitation and Proposers’ Day.