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Research programme: glycogen storage disease type I therapeutics - Prime Medicine

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Drug Profile

Research programme: glycogen storage disease type I therapeutics - Prime Medicine

Latest Information Update: 28 Oct 2024

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At a glance

  • Originator Prime Medicine
  • Class Gene therapies
  • Mechanism of Action SLC37A4 protein expression 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

  • Preclinical Glycogen storage disease type I

Most Recent Events

  • 24 Oct 2024 Pharmacodynamics data from a preclinical trial in Glycogen storage disease 1b released by Pime Medicine
  • 08 Aug 2024 Preclinical trials in Glycogen storage disease type I in USA (IV) before August 2024

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