Research programme: gene therapies - Spark Therapeutics
Latest Information Update: 15 May 2015
At a glance
- Originator The Childrens Hospital of Philadelphia
- Developer Spark Therapeutics
- 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
- Preclinical Haematological disorders; Neurodegenerative disorders; Retinal disorders