Gene therapy aims to free patients from daily steroids for rare adrenal disorder
Disease control
Ongoing
This early-phase trial tests a gene therapy called BBP-631 for classic congenital adrenal hyperplasia (CAH), a genetic condition that forces patients to take daily steroids. The therapy uses a harmless virus to deliver a working copy of the faulty gene. Only 8 adults are enrolled…
Phase: PHASE1, PHASE2 • Sponsor: Adrenas Therapeutics Inc • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC