Gene therapy trial offers hope for babies with rare, deadly metabolic disease
Disease control
Recruiting now
This study tests a one-time gene therapy called ECUR-506 in baby boys under 9 months old with a severe form of OTC deficiency, a genetic disorder that prevents the body from breaking down ammonia. The goal is to see if the treatment is safe and can reduce dangerous ammonia levels…
Phase: PHASE1, PHASE2 • Sponsor: iECURE, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC