Leber congenital amaurosis gene therapy - Applied Genetic Technologies Corporation

Drug Profile

Leber congenital amaurosis gene therapy - Applied Genetic Technologies Corporation

Alternative Names: AAV2-RPE65 Vector; rAAV2-CB-hRPE65

Latest Information Update: 27 Apr 2016

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At a glance

  • Originator Applied Genetic Technologies Corporation
  • Developer Applied Genetic Technologies Corporation; University of Massachusetts Medical School
  • 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.

    Yes - Leber congenital amaurosis
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Leber congenital amaurosis

Most Recent Events

  • 22 Apr 2016 Interim efficacy and adverse events data from a phase I/II trial in Leber congenital amaurosis released by Applied Genetic Technologies
  • 25 Jun 2015 Phase I/II development is ongoing in USA
  • 10 Sep 2014 This programme is still active
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