Experimental gene therapy offers hope for children with rare fatal brain disease
Disease control
Not yet recruiting
This trial tests a one-time gene therapy for children with CLN6 Batten disease, a rare and fatal brain disorder. The treatment delivers a working copy of the CLN6 gene via a spinal injection. Researchers will check if it is safe and can slow the disease's progression. The study i…
Phase: PHASE1, PHASE2 • Sponsor: The Charlotte and Gwenyth Gray Foundation • Aim: Disease control
Last updated May 15, 2026 11:54 UTC