Shire has submitted a request to the US Food and Drug Administration to test its investigational factor VIII (FVIII) gene therapy SHP654 in patients with the bleeding disorder haemophilia A.
SHP654 (also known as BAX 888) aims to protect haemophilia A patients against bleeds through the delivery of a long-term, constant level of factor expression, the firm noted.
Shire’s gene therapy programme for haemophilia A uses a recombinant adeno-associated virus serotype 8 (rAAV8) vector that selectively targets the liver.
It involves the delivery of a functional copy of FVIII to the body’s liver to enable its own production of FVIII, rather than relying on a factor-based treatment.
The Investigational New Drug submission is based on the results of preclinical and phase I studies showing the potential utility of its candidate, the firm noted.
If Shire gets a green light for human testing it intends to study SHP654 in a global multi-center trial evaluating safety and examining the doses required to boost factor VIII activity levels and affect haemophilic bleeding.