Gene therapy trial targets blindness in rare batten disease
Disease control
Recruiting now
This study tests a new gene therapy, TTX-381, for vision problems caused by CLN2 disease (a form of Batten disease). About 16 people who already receive brain enzyme infusions will get a single eye injection to see if it is safe and can slow vision loss. The goal is to protect th…
Phase: PHASE1, PHASE2 • Sponsor: Tern Therapeutics, LLC • Aim: Disease control
Last updated May 07, 2026 15:51 UTC