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GTX 102

Drug Profile

GTX 102

Alternative Names: GTX-102- GeneTx Biotherapeutics; GTX102

Latest Information Update: 05 Oct 2021

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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 RNA interference; 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 I/II Angelman syndrome

Most Recent Events

  • 29 Sep 2021 US FDA removes clinical hold on phase I/II clinical study of GTX 102 for the treatment of Angelman syndrome in USA
  • 16 Sep 2021 Biomarkers information updated
  • 16 Sep 2021 GeneTx Biotherapeutics and Ultragenyx Pharmaceutical re-initiates a phase I/II KIK-AS trial in Angelman syndrome in USA (NCT04259281)
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