Research programme: AAV-based gene therapies - Homology Medicines

Drug Profile

Research programme: AAV-based gene therapies - Homology Medicines

Alternative Names: AAVHSC 17; AAVHSC vectors; AMEnDR™

Latest Information Update: 20 Sep 2016

Price : $50

At a glance

  • Originator Beckman Research Institute
  • Developer California Institute of Technology/CalTech; Homology Medicines
  • 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.

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical CNS disorders

Most Recent Events

  • 15 Sep 2016 Homology Medicines and the California Institute of Technology collaborates for in-vivo adeno-associated virus technology
  • 15 Sep 2016 Preclinical trials in CNS disorders in USA (unspecified route)
  • 02 May 2016 Early research in Undefined indication in USA (Parenteral)
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