Research programme: AAV-based gene therapies - Homology Medicines

Drug Profile

Research programme: AAV-based gene therapies - Homology Medicines

Alternative Names: 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.

    No
  • 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)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top