drugset
Drugs / JAG201
last change Feb 2026 re-read 3 minutes ago

JAG201

also known as AAV2/9-miniSHANK3

Gene therapy (AAV / viral vector)

Developed for
Phelan-McDermid syndrome
Investigated by
Jaguar Gene Therapy, LLC

Trials 1

today NCT06662188 · Ph 1/2
20242025202620272028
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT06662188 Jan 2024 → Jun 2028 expected Phelan-McDermid syndrome Jaguar Gene Therapy, LLC Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 3

DateIssuerRelease
2026-02-24 Jaguar Gene Therapy, LLC Results Jaguar Gene Therapy Announces Successful Completion of Dosing of First Patient Cohort in Clinical Trial Evaluating JAG201 for the Treatment of a Leading Monogenic Cause of Autism Spectrum Disorder Known as Phelan-McDermid Syndrome jaguargenetherapy.com ↗
Early indications of clinical benefit have been observed across neurodevelopmental domains, including communicative, motor, cognitive and social.
2024-07-15 Jaguar Gene Therapy, LLC Regulatory Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome jaguargenetherapy.com ↗
Specifically, the company received the FDA’s agreement to administer JAG201 to both pediatric and adult patients.
2024-01-31 Jaguar Gene Therapy, LLC Regulatory Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome jaguargenetherapy.com ↗

All press releases naming this drug 3 releases

DateIssuerRelease

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as JAG201 ClinicalTrials.gov intervention name — accepted as the source's own label NCT06662188 ↗
Known as AAV2/9-miniSHANK3 “Adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene (AAV2/9-miniSHANK3)” NCT06662188 ↗
Modality Gene therapy (AAV / viral vector) “Adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene” NCT06662188 ↗
Route Other “administered via intracerebroventricular (ICV) injection” NCT06662188 ↗