Research programme: gene therapies - Audentes Therapeutics

Drug Profile

Research programme: gene therapies - Audentes Therapeutics

Alternative Names: AAV-CASQ2 gene therapy - Audentes; AT 307; AT-003 - Audentes/REGENXBIO

Latest Information Update: 05 Oct 2015

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At a glance

  • Originator Audentes Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Ventricular tachycardia

Most Recent Events

  • 14 Nov 2017 Audentes Therapeutics plans to file an IND application for AT 307 for Ventricular tachycardia (CASQ2-related catecholaminergic polymorphic ventricular tachycardia) in the first quarter of 2018
  • 05 Oct 2015 Preclinical trials in Ventricular tachycardia in USA (Parenteral)
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