Brain-Delivered gene therapy offers hope for devastating childhood disorder
Disease control
Recruiting now
This is the first human trial of a gene therapy for children with CTNNB1 syndrome, a severe genetic disorder that affects brain development. Researchers will test if a single dose of a therapy called Urbagen, delivered directly to the brain, is safe and can improve motor function…
Phase: PHASE1, PHASE2 • Sponsor: CTNNB1 Foundation • Aim: Disease control
Last updated Mar 19, 2026 14:56 UTC