One-Shot gene therapy tested to halt fatal childhood brain disease
Disease control
Completed
This study tested a one-time gene therapy injection for children with a rare, fatal genetic brain disorder called CLN6 Batten disease. The therapy aimed to deliver a working copy of the faulty CLN6 gene directly to the nervous system to slow or stop disease progression. Researche…
Phase: PHASE1, PHASE2 • Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC