The U.S Food and Drug Administration (FDA) granted fast-track designation to SB-525 for the treatment of hemophilia A.
SB-525 is an investigational cDNA gene therapy that uses a recombinant adeno-associated virus to deliver a human factor VIII cDNA construct to the nuclei of liver cells with a single infusion, according to the drug’s manufacturers. The drug is intended for use as single, continuous therapy.
The FDA previously granted orphan drug designation to SB-525. The therapy will be evaluated in a phase I/II trial of adults with hemophilia A, which is expected to open and begin enrolling participants by fall 2017.
Source: Sangamo Therapeutics press release, May 16, 2017.