Research programme: gene therapies - Spark Therapeutics/University of Massachusetts Medical School
Latest Information Update: 11 Nov 2016
At a glance
- Originator Spark Therapeutics
- Developer Spark Therapeutics; University of Massachusetts Medical School
- 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 Liver disorders; Neurological disorders; Retinal disorders