Posted by Michael Wonder on 17 May 2017
Sangamo receives fast track designation from the FDA for SB-525 investigational haemophilia A gene therapy
16 May 2017 - Sangamo Therapeutics announced today that the U.S. FDA has granted fast track designation to SB-525, the Company's clinical stage cDNA gene therapy candidate for haemophilia A, which is being developed as part of an exclusive, global collaboration and license agreement with Pfizer.
SB-525 uses a recombinant adeno-associated virus to deliver a human Factor VIII cDNA construct and proprietary, synthetic 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 Factor VIII protein.
