One-Shot gene therapy trial offers hope for devastating infant disease
Disease control
Terminated
This study is testing a one-time gene therapy called PBKR03 for babies with early infantile Krabbe disease, a rare and severe neurological disorder. Researchers will give the therapy directly into the fluid around the brain to deliver a working copy of a missing gene. The main go…
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC