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Research programme: hepatolenticular degeneration gene therapies - Prime Medicine

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Research programme: hepatolenticular degeneration gene therapies - Prime Medicine

Latest Information Update: 18 Nov 2024

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At a glance

  • Originator Prime Medicine
  • Class Gene therapies
  • Mechanism of Action Gene 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 Hepatolenticular degeneration

Most Recent Events

  • 12 Nov 2024 Prime medicine announces intention to submit an IND and/or clinical trial application (CTA) for Hepatolenticular degeneration in the first half of 2026
  • 24 Oct 2024 Pharmacodynamics data from a preclinical trial in Hepatolenticular degeneration released by Prime Medicine
  • 30 Sep 2024 Prime Medicine plans an IND-enabling activities for Hepatolenticular degeneration in the fourth quarter of 2024

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