Experimental gene therapy aims to halt rare fatal brain disease in children
Disease control
Ongoing
This early-stage trial tests a one-time gene therapy called CLN-301 in 7 children aged 3 to 10 with CLN3 Batten disease, a rare genetic disorder that causes progressive loss of vision, movement, and thinking skills. The therapy delivers a working copy of the CLN3 gene directly in…
Phase: PHASE1, PHASE2 • Sponsor: Neela Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC