SHANK3 HAPLOINSUFFICIENCY

Clinical trials for SHANK3 HAPLOINSUFFICIENCY explained in plain language.

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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…

Matched conditions: SHANK3 HAPLOINSUFFICIENCY

Phase: PHASE1, PHASE2 • Sponsor: Jaguar Gene Therapy, LLC • Aim: Disease control

Last updated Apr 23, 2026 11:59 UTC

SHANK3 HAPLOINSUFFICIENCY

Get alerted when new SHANK3 HAPLOINSUFFICIENCY trials appear

First human trial tests One-Shot gene therapy for rare genetic disorder