Gene therapy shot aims to save sight in batten disease kids
Disease control
Recruiting now
This early study tests a one-time gene therapy injection under the retina for children with CLN2 Batten disease who already receive enzyme therapy. The main goal is to check safety in 16 participants, with a secondary look at whether it can preserve vision. It is a first-in-human…
Phase: PHASE1, PHASE2 • Sponsor: Tern Therapeutics, LLC • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC