Research programme: gene therapies - Audentes Therapeutics

Drug Profile

Research programme: gene therapies - Audentes Therapeutics

Alternative Names: AAV-CASQ2 gene therapy - Audentes; AAV-MTM1 (AT001); AT 132; AT 307; AT 342; AT 982; AT-001 - Audentes/REGENXBIO; AT-002 - Audentes/REGENXBIO; AT-003 - Audentes/REGENXBIO; XLMTM gene therapy (AT001) - Audentes/Genethon

Latest Information Update: 06 Feb 2017

Price : $50

At a glance

  • Originator Audentes Therapeutics; Genethon
  • Developer Audentes Therapeutics; Genethon; University of Pennsylvania
  • Class Gene therapies
  • Mechanism of Action Alpha glucosidase expression stimulants; Gene transference; Myotubularin expression stimulants
  • Orphan Drug Status

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

    Yes - Musculoskeletal disorders
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Glycogen storage disease type II; Hyperbilirubinaemia; Musculoskeletal disorders; Ventricular tachycardia

Most Recent Events

  • 06 Feb 2017 US FDA approves IND application for AT 342 in Hyperbilirubinaemia
  • 01 Feb 2017 Audentes plans a phase I/II trial for Hyperbilirubinaemia
  • 12 Nov 2016 Audented Therapeutics plans to file an IND application with the US FDA for Glycogen storage disease type II in the fourth quarter of 2016
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