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Research programme: Friedreichs Ataxia therapeutics - Prime Medicine

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Drug Profile

Research programme: Friedreichs Ataxia therapeutics - Prime Medicine

Latest Information Update: 09 Sep 2024

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At a glance

  • Originator Prime Medicine
  • Class Gene therapies
  • Mechanism of Action Frataxin expression modulators; 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

  • Research Friedreich's ataxia

Most Recent Events

  • 17 Aug 2024 Early research in Friedreich's ataxia in USA (Parenteral) prior to August 2024 (Prime Medicine pipeline, August 2024)

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