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Jaguar Gene Therapy, Llc

Clinical trials sponsored by Jaguar Gene Therapy, Llc, explained in plain language.

Trials to join now! 1

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 1/2 • Sponsor: Jaguar Gene Therapy, LLC • Aim: Disease control

Last updated Sep 05, 2026 00:00 UTC