First human trial tests One-Shot gene therapy for rare genetic disorder
Disease control
Recruiting now
This early-stage study is testing a single dose of an experimental gene therapy called JAG201 in children aged 2-9 with SHANK3 deficiency, which causes Phelan-McDermid Syndrome. The main goal is to check if the treatment is safe and tolerable when injected into the fluid around t…
Phase: PHASE1, PHASE2 • Sponsor: Jaguar Gene Therapy, LLC • Aim: Disease control
Last updated Apr 23, 2026 11:44 UTC