ARPA-H is awarding up to $160 million under its THRIVE program to multiple research teams developing custom, in vivo gene-editing therapies for rare genetic diseases, with the goal of starting clinical trials within three years and expanding umbrella trials with multiple products and diseases by year five.
The awardees include major institutions such as Children’s Hospital of Philadelphia, UC Berkeley/Innovative Genomics Institute, St. Jude Children’s Research Hospital, the Broad Institute, Massachusetts General Hospital, Stanford University, and biotech companies like GemmaBio and Profluent Bio, each targeting different rare disease groups.
The program emphasizes building integrated platform technologies, standardized toxicology and biodistribution profiles, and novel clinical trial designs to enable personalized genetic medicines that can be developed quickly for individual patients, supported by new FDA regulatory pathways and U.S. manufacturing infrastructure such as the GIVE program.
Sources:
ARPA-H launches $160 million effort to develop custom gene editing drugs - The Boston Globe
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