Research programme: Leber congenital amaurosis type 1 gene therapy - Sanofi Genzyme/University of Florida
Latest Information Update: 16 Dec 2016
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.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- 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)