First-Ever gene therapy targets brain cells in devastating childhood disease
Disease control
ENROLLING_BY_INVITATION
This study is testing a new, one-time gene therapy for children with Canavan disease, a severe and fatal genetic brain disorder. The therapy is delivered directly into the brain during a surgical procedure, aiming to replace the faulty gene in the specific brain cells that cause …
Phase: PHASE1, PHASE2 • Sponsor: Myrtelle Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC