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Research programme: AAV-based gene therapies - Passage Bio/GEMMA Biotherapeutics

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Research programme: AAV-based gene therapies - Passage Bio/GEMMA Biotherapeutics

Alternative Names: AAV based therapeutics - Passage Bio/University of Pennsylvania; CNS disorder gene therapies - Passage Bio; CNS disorder gene therapies - Passage Bio/GEMMA Biotherapeutics

Latest Information Update: 07 Aug 2024

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At a glance

  • Originator University of Pennsylvania
  • Developer GEMMA Biotherapeutics; Passage Bio; University of Pennsylvania
  • 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

  • Research CNS disorders; Huntington's disease
  • Discontinued Canavan disease; Charcot-Marie-Tooth disease

Most Recent Events

  • 07 Aug 2024 Passage Bio enters licensing agreement with GEMMA Biotherapeutics for AAV-based gene therapies
  • 01 Aug 2024 Early research in CNS disorders is ongoing USA (Parenteral)
  • 31 Jul 2024 Passage Bio terminates its collaboration for CNS gene therapy programs with University of Pennsylvania

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