Novel Hemophilia A Gene Therapy Gets Fast Track Status

The Food and Drug Administration (FDA) has granted Fast Track designation to SB-525 (Sangamo Therapeutics) for the treatment of hemophilia A.

SB-525 is a cDNA gene therapy which utilizes a recombinant adeno-associated virus (rAAV) to deliver a human Factor VIII cDNA construct and proprietary, synthetic liver-specific promoter to the nucleus of liver cells. The therapy is designed as a single infusion to provide a continuous, therapeutic expression of Factor VIII protein. SB-525 is being developed in collaboration with Pfizer.

The Phase 1/2 clinical trial for SB-525 in adult patients with hemophilia A is expected to begin enrollment by the end of the second quarter 2017. Study data are expected in late 2017 or early 2018.

For more information visit Sangamo.com.