Research programme: eye disorder gene therapies - Applied Genetic Technologies Corporation/BiogenAlternative Names: XLRP gene therapy
Latest Information Update: 10 Jun 2016
At a glance
- Originator Applied Genetic Technologies Corporation
- 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 Yes
Highest Development Phases
- Preclinical Age-related macular degeneration; Retinitis pigmentosa
Most Recent Events
- 07 Jun 2016 Research programme: eye disorder gene therapies - Applied Genetic Technologies Corporation/Biogen receives Orphan Drug status for Retinitis pigmentosa in European Union
- 02 May 2016 Pharmacodynamics data from preclinical studies in Retinitis pigmentosa released by by Applied Genetic Technologies Corporation