Description
The Advanced Research Projects Agency for Health (ARPA-H) is seeking proposals for the Treating Hereditary Rare Diseases With In Vivo Precision Genetic Medicines (THRIVE) program. The program aims to develop groundbreaking new ways to tackle health-related challenges through high-potential, high-impact biomedical and health research. The program will focus on developing precision genetic medicines (PGMs) that can slow, reverse, or cure disease. The program consists of three modules: Module 1 (Design and Development), Module 2 (Investigational Medicine), and Module 3 (Real-world Viability Pilots and Scaling). Proposers are expected to submit a solution summary and a full proposal. The program will provide funding for up to 5 years.
Key aspects of the program include:
* Developing PGMs that can address multiple variants within a gene, clusters of mutations, or clusters of diseases with common nodal genetic pathways
* Developing solutions that address mutations leading to severe conditions in neonatal or pediatric populations
* Establishing a publicly accessible data platform to enable AI-assisted iteration and growth of a robust PGM industry
* Piloting comprehensive RD patient solutions, from identification, diagnosis, treatment, and clinical long-term follow-up at satellite clinics virtually networked to partnered expert centers
* Demonstrating ability to administer novel PGMs to patients where they reside