RPE65 gene therapyAlternative Names: REP 65 gene therapy
Latest Information Update: 22 Jan 2003
At a glance
- Originator Nonindustrial source
- Mechanism of Action
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.
Highest Development Phases
- No development reported Blindness
Most Recent Events
- 07 May 2001 Preclinical development for Leber congenital amaurosis in USA (Intraocular)