One-Shot gene therapy trial aims to fix rare hormone disorder
Disease control
Ongoing
This is the first-ever study in people to test a gene therapy for congenital adrenal hyperplasia (CAH), a genetic disorder where the body can't make enough cortisol. Eight adults with classic CAH will receive a single intravenous dose of the experimental therapy, called BBP-631. …
Phase: PHASE1, PHASE2 • Sponsor: Adrenas Therapeutics Inc • Aim: Disease control
Last updated Mar 10, 2026 12:52 UTC