Experimental gene therapy hopes to slow rare brain disease in toddlers
Disease control
Recruiting now
This study tests a gene therapy called BBP-812 in children up to 30 months old with Canavan disease, a rare genetic brain disorder. The treatment uses a harmless virus to deliver a working copy of the ASPA gene, aiming to reduce harmful brain chemicals and improve motor and think…
Phase: PHASE1, PHASE2 • Sponsor: Aspa Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:09 UTC