Gene therapy trial hopes to tackle rare Autism-Linked condition
Disease control
Recruiting now
This early-stage trial tests a single dose of JAG201 gene therapy, given directly into the brain fluid, for people with SHANK3 haploinsufficiency (Phelan-McDermid syndrome), a rare genetic condition causing developmental delays and autism-like symptoms. The study includes childre…
Phase: PHASE1, PHASE2 • Sponsor: Jaguar Gene Therapy, LLC • Aim: Disease control
Last updated Jun 27, 2026 09:03 UTC