Factor VIII gene therapy - Targeted Genetics
Latest Information Update: 16 May 2007
At a glance
- Originator University of North Carolina
- Class Blood coagulation factors; Coagulants
- Mechanism of Action Blood coagulation factor replacements; Factor X stimulants; 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.
Highest Development Phases
- Discontinued Haemophilia A