Research programme: AAV-based gene therapies - Homology Medicines

Drug Profile

Research programme: AAV-based gene therapies - Homology Medicines

Alternative Names: AAVHSC 15; AAVHSC 17; AAVHSC vectors; AMEnDR™; HMI-102

Latest Information Update: 24 May 2018

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At a glance

  • Originator Beckman Research Institute
  • Developer 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 No

Highest Development Phases

  • Preclinical Phenylketonuria
  • Research CNS disorders; Cystic fibrosis; Duchenne muscular dystrophy; Lysosomal-storage diseases

Most Recent Events

  • 18 May 2018 Pharmacodynamics data from preclinical studies in Phenylketonuria released by Homology Medicines
  • 18 May 2018 Homology Medicines plans a phase I/II trial for Phenylketonuria for HMI 102 in 2019
  • 15 Mar 2018 Homology Medicines has patent protection for human haematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) in USA
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