ARPA-H awards $160 M to accelerate bespoke gene‑editing therapies for rare diseases

ARPA-H announced a $160 million, five‑year investment through its THRIVE program to speed the development of personalized gene‑editing treatments for rare genetic conditions.

The awardees include the Children’s Hospital of Philadelphia, UC Berkeley’s Innovative Genomics Institute and gene‑editing biotech GemmaBio. Recipients are expected to begin first‑in‑human trials by the third year that can evaluate multiple individualized products for a single disease, and to publish blueprints to guide other rare‑disease researchers.

In related updates, Biohaven disclosed a change in its scientific leadership, a Botox rival reported success against migraines, and two separate deals were announced, though specifics were not detailed in the roundup.