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ACTX 401

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Drug Profile

ACTX 401

Alternative Names: ACTX-401

Latest Information Update: 09 Nov 2022

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At a glance

  • Originator Alcyone Lifesciences
  • Developer Alcyone 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Spinal muscular atrophy
  • Preclinical Charcot-Marie-Tooth disease

Most Recent Events

  • 29 Sep 2022 Preclinical trials in Charcot-Marie-Tooth disease in USA (Parenteral) (Alcyone Therapeutics pipeline, September 2022)
  • 18 May 2022 US FDA approves IND application for ACTX 401 in Spinal muscular atrophy
  • 18 May 2022 Pharmacodynamics and adverse event data from a preclinical trials in Spinal muscular atrophy released by Alcyone Therapeutics

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