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GTX 102

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Drug Profile

GTX 102

Alternative Names: GTX-102- GeneTx Biotherapeutics; GTX102

Latest Information Update: 12 Feb 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

  • 08 Jan 2025 Ultragenyx Pharmaceutical completes the phase-I/II KIK-AS trial in Angelman syndrome (In adolescents, In children) in the US, Canada, France, Australia, Germany, Israel, Spain and the UK (Intrathecal) (NCT04259281)
  • 31 Dec 2024 Ultragenyx Pharmaceutical has patent protection for GTX 102 in USA and other foreign countries, licensed from TAMU, before December 2024
  • 03 Dec 2024 Phase-III clinical trials in Angelman syndrome (In adolescents, In children) in USA, Spain, Poland, Netherlands, Japan, Germany, Canada, Australia (Intrathecal)

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