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GTX 102

Drug Profile

GTX 102

Alternative Names: GTX-102- GeneTx Biotherapeutics

Latest Information Update: 25 May 2020

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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 UBE3A protein expression modulators
  • 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 I/II Angelman syndrome

Most Recent Events

  • 06 May 2020 Ultragenyx maintain its option to acquire GeneTx
  • 04 May 2020 GTX 102 receives Fast Track designation for Angelman syndrome [Intrathecal,Injection] (In adolescents, In children) in USA
  • 18 Feb 2020 Phase-I/II clinical trials in Angelman syndrome (In children, In adolescents) in USA (Intrathecal) (NCT04259281)
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