GTX 101 - GeneTx Biotherapeutics

Drug Profile

GTX 101 - GeneTx Biotherapeutics

Latest Information Update: 29 Mar 2018

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At a glance

  • Originator Texas A&M AgriLife Research; Texas A&M University
  • Developer GeneTx Biotherapeutics
  • Class Antisense oligonucleotides; Neuroprotectants
  • Mechanism of Action Genetic transcription inhibitors
  • 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

  • Preclinical Angelman syndrome

Most Recent Events

  • 27 Mar 2018 GTX 101 receives Orphan Drug status for Angelman syndrome in USA
  • 22 Feb 2018 GTX 101 licensed to GeneTx Biotherapeutics in USA
  • 22 Feb 2018 Preclinical trials in Angelman syndrome in USA (unspecified route), prior to February 2018
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