Research programme: adeno-associated virus based gene therapies - Homology Medicines/Novartis

Drug Profile

Research programme: adeno-associated virus based gene therapies - Homology Medicines/Novartis

Latest Information Update: 20 Mar 2018

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At a glance

  • Originator Homology Medicines
  • Developer Homology Medicines; Novartis
  • 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 Eye-Disorders; Haemoglobinopathies

Most Recent Events

  • 15 Mar 2018 Homology Medicines has patent protection for human haematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) in USA
  • 13 Nov 2017 Research programme: adeno-associated virus based gene therapies licensed to Novartis worldwide (excluding USA)
  • 13 Nov 2017 Early research in Eye disorders in Switzerland (Parenteral)
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