Neuronal Ceroid Lipofuscinosis Type 2 research hub

A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

This study is testing a new gene therapy, TTX-381, for children with CLN2 disease, also known as Batten disease. Researchers want to see if it's safe and helps improve eye problems caused by the condition. It's the first time this treatment is being used in people.