Experimental gene therapy offers hope for babies with rare brain disease
Disease control
Terminated
This study tests a gene therapy called PBKR03 for children under 9 months with early infantile Krabbe disease, a severe genetic disorder affecting the brain and nerves. The therapy aims to deliver a working copy of the GALC gene to help stop or slow the disease. Researchers will …
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated May 14, 2026 12:06 UTC