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Research programme: muscular dystrophy gene therapies - Columbia University/Sarepta Therapeutics

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Drug Profile

Research programme: muscular dystrophy gene therapies - Columbia University/Sarepta Therapeutics

Alternative Names: AAV gene therapy - Columbia University/ Sarepta Therapeutics

Latest Information Update: 28 Jun 2024

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At a glance

  • Originator Sarepta 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

  • No development reported Muscular dystrophies

Most Recent Events

  • 28 Jun 2024 No recent reports of development identified for research development in Muscular-dystrophies in USA (Parenteral)
  • 04 May 2020 Early research in Muscular dystrophies in USA (Parenteral), prior to May 2020 (Sarepta Therapeutics pipeline, May 2020)

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