One-Time gene injection holds promise for rare childhood brain disease
Disease control
Ongoing
This early-stage trial tests a single injection of gene therapy (AAV9/CLN7) into the spinal fluid of children with CLN7 Batten disease, a rare and severe genetic disorder that causes progressive loss of motor and mental abilities. The main goal is to check safety, but researchers…
Phase: PHASE1 • Sponsor: Benjamin Greenberg • Aim: Disease control
Last updated Apr 26, 2026 20:01 UTC