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
Diagnosis of CLN6
At least 4 months old
Presence of another inherited neurologic disease
Prior stem cell transplantation
Prior gene transfer, gene editing, or viral vector therapy
The Charlotte and Gwenyth Gray Foundation
Lead sponsor
University of California, San Diego
Collaborator