Research programme: haemophilia A gene therapy - Spark TherapeuticsAlternative Names: SPK FVIII program
Latest Information Update: 12 Aug 2015
At a glance
- Originator 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 Haemophilia A
Most Recent Events
- 05 Aug 2015 Preclinical trials in Haemophilia A in USA (Parenteral)