Experimental gene therapy offers hope for rare brain disease in toddlers
Disease control
Recruiting now
This study tests a new gene therapy called BBP-812 for children with Canavan disease, a rare genetic disorder that damages the brain. The treatment uses a harmless virus to deliver a working copy of the missing gene. The trial aims to see if it is safe and can improve motor and t…
Phase: PHASE1, PHASE2 • Sponsor: Aspa Therapeutics • Aim: Disease control
Last updated Apr 26, 2026 20:02 UTC