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Research programme: gene-editing therapies - Aceragen/Scriptr Global

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Drug Profile

Research programme: gene-editing therapies - Aceragen/Scriptr Global

Latest Information Update: 20 Jan 2023

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At a glance

  • Originator Idera Pharmaceuticals; Scriptr Global
  • Developer Aceragen
  • Class Gene therapies
  • Mechanism of Action Gene modulators
  • 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; Myotonic dystrophy

Most Recent Events

  • 17 Jan 2023 Idera Pharmaceuticals is now called Aceragen
  • 01 Feb 2021 Idera Pharmaceuticals enters into a collaboration and option agreement with Scriptr Global to develop gene-editing therapeutics, for the treatment of Myotonic dystrophy 1 and Friedrich’s ataxia
  • 01 Feb 2021 Early research in Friedreich's ataxia in USA (Parenteral)

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