AT 132 - Audentes Therapeutics

Drug Profile

AT 132 - Audentes Therapeutics

Alternative Names: AT-001 - Audentes Therapeutics/Genethon; AT-132

Latest Information Update: 05 Oct 2017

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At a glance

  • Originator Audentes Therapeutics; Genethon
  • Developer Audentes Therapeutics
  • Class Gene therapies
  • Mechanism of Action 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 - Congenital structural myopathies
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Congenital structural myopathies

Most Recent Events

  • 03 Oct 2017 Medicines and Healthcare Products Regulatory Agency approves CTA for AT 132 in Congenital structural myopathies (X-Linked Myotubular Myopathy) in United Kingdom
  • 27 Sep 2017 AT 132 receives Fast Track designation for Congenital structural myopathies [IV] (In children, In infants) in USA
  • 27 Sep 2017 AT 132 receives Rare Pediatric Disease designation from the FDA for Congenital structural myopathies
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