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scAAV9 U7 ACCA

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Drug Profile

scAAV9 U7 ACCA

Alternative Names: scAAV9.U7.ACCA

Latest Information Update: 08 Apr 2021

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At a glance

  • Originator Audentes Therapeutics
  • Developer Astellas Gene Therapies; Nationwide Children's Hospital
  • Class Gene therapies; Small interfering RNA
  • Mechanism of Action Dystrophin expression stimulants; RNA interference
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Duchenne muscular dystrophy

Most Recent Events

  • 01 Apr 2021 Audentes Therapeutics is now called Astellas Gene Therapies
  • 07 Feb 2020 Phase-I/II clinical trials in Duchenne muscular dystrophy (In infants, In children) in USA (Parenteral) (NCT04240314)
  • 07 Feb 2020 Preclinical data for Duchenne muscular dystrophy released by Audentes Therapeutics (NCT04240314)

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