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JAG 201

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Drug Profile

JAG 201

Alternative Names: JAG-201

Latest Information Update: 01 Mar 2026

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At a glance

  • Originator Broad Institute
  • Developer Jaguar Gene Therapy
  • Class Behavioural disorder therapies; Gene therapies
  • Mechanism of Action Gene transference; SHANK3 protein replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Telomeric 22q13 Monosomy Syndrome
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Autism spectrum disorder; Telomeric 22q13 Monosomy Syndrome

Most Recent Events

  • 24 Feb 2026 JAG 201 receives Orphan Drug status for Telomeric 22q13 Monosomy Syndrome in USA
  • 28 Mar 2025 No recent reports of development identified for preclinical development in Autism spectrum disorder in USA (Intraventricular, Injection)
  • 28 Oct 2024 Jaguar Gene Therapy plans a phase I/II trial for Telomeric-22q13-Monosomy-Syndrome (In Children) in USA (Intraventricular) (NCT06662188)

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