Gene Therapy Trial for CLN6 Batten Disease

The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal ceroid lipofuscinosis). The main questions it aims to answer are if he gene therapy safe and well tolerated, and if the gene therapy help slow disease progression or improve symptoms. Participants will: Receive a single dose of the gene therapy through an injection into the fluid around the spinal cord (intrathecal administration) Have regular study visits over 2 years for safety checks and assessments of disease progression Be followed for an additional 3 years in a long-term follow-up study

NCT Number	NCT07582484
Lead Sponsor	The Charlotte and Gwenyth Gray Foundation Collaborators: University of California, San Diego
Conditions	CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis
Enrollment	12 participants
Start Date	2026-08
Primary Completion	2027-08 (estimated)
Study Completion	2028-08 (estimated)
Updated on ClinicalTrials.gov	2026-05-14