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EMU 005

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Drug Profile

EMU 005

Alternative Names: EMU005

Latest Information Update: 22 Sep 2025

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At a glance

  • Originator Emugen Therapeutics
  • Class Gene therapies; Neuroprotectants
  • Mechanism of Action RNA splicing 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 Neurodegenerative disorders; Neurodevelopmental disorders

Most Recent Events

  • 13 Aug 2025 Emugen Therapeutics in-licenses TfR1 CapX capsid technology from Apertura Gene Therapy
  • 13 Aug 2025 Early research in Neurodegenerative disorders in USA (Parenteral), prior to August 2025
  • 13 Aug 2025 Early research in Neurodevelopmental disorders in USA (Parenteral), prior to August 2025

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