Sangamo Receives FDA Fast Track Designation for Hemophilia A Gene Therapy
The FDA granted Sangamo a Fast Track designation for its SB-525 cDNA gene therapy for hemophilia A, which is being developed with Pfizer.
SB-525 uses a recombinant adeno-associated virus to deliver a human factor VIII cDNA construct and a liver-specific promoter to the nucleus of liver cells with a single infusion.
The therapy is designed as a single treatment strategy intended to provide continuous, therapeutic expression of the factor VIII blood-clotting protein. SB-525 has already received an orphan drug designation from the FDA.
The FDA has cleared an IND application for the program, and data from a Phase I/II clinical trial evaluating SB-525 in adults with hemophilia A is expected in late 2017 or early 2018.