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Apazunersen - GeneTx Biotherapeutics

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Drug Profile

Apazunersen - GeneTx Biotherapeutics

Alternative Names: GTX 102; GTX-102 - GeneTx Biotherapeutics

Latest Information Update: 11 Sep 2025

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At a glance

  • Originator Texas A&M AgriLife Research; Texas A&M University
  • Developer GeneTx Biotherapeutics; Ultragenyx Pharmaceutical
  • Class Antisense oligonucleotides; Neuroprotectants
  • Mechanism of Action Gene silencing; UBE3A protein expression stimulants
  • Orphan Drug Status

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

    Yes - Angelman syndrome
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Angelman syndrome

Most Recent Events

  • 05 Sep 2025 Ultragenyx Pharmaceutical plans phase II Aurora trial in Angelman Syndrome (In infants, In children, In adolescents and In adults) in November 2025 (NCT07157254)
  • 31 Jul 2025 Ultragenyx Pharmaceutical completes enrollment in a phase III clinical trials in Angelman syndrome (In adolescents, In children) in USA, Spain, Poland, Netherlands, Japan, Germany, Canada, Australia (Intrathecal) (NCT06617429)
  • 27 Jun 2025 Apazunersen - GeneTx Biotherapeutics receives Breakthrough Therapy status for Angelman syndrome in USA

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