One-Time gene therapy could free babies from lifelong medication for rare liver disease
Disease control
Recruiting now
This study tests a single-dose gene therapy called ECUR-506 in baby boys under 9 months old with a severe form of OTC deficiency, a genetic liver disorder that causes dangerous ammonia buildup. The goal is to see if the treatment is safe and can reduce or eliminate the need for d…
Phase: PHASE1, PHASE2 • Sponsor: iECURE, Inc. • Aim: Disease control
Last updated May 07, 2026 18:40 UTC