Research programme: gene therapy - Spark Therapeutics IrelandAlternative Names: GT 038; RHO-adRP; RhoNova; SPK- LHON
Latest Information Update: 19 Sep 2016
At a glance
- Originator Genable Technologies
- Developer Spark Therapeutics Ireland
- Class Gene therapies
- Mechanism of Action Gene transference; RNA interference
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
- Preclinical Retinitis pigmentosa