Research programme: Leber congenital amaurosis type 1 gene therapy - Sanofi Genzyme/University of Florida

Drug Profile

Research programme: Leber congenital amaurosis type 1 gene therapy - Sanofi Genzyme/University of Florida

Latest Information Update: 16 Dec 2016

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At a glance

  • Originator University of Florida
  • 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 Leber congenital amaurosis

Most Recent Events

  • 24 Sep 2014 Early research in Leber congenital amaurosis in USA (Intraocular)
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