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Pharmaceuticals / Submissions and Approvals

Sangamo Receives FDA Fast Track Designation for Hemophilia A Gene Therapy

May 19, 2017

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.

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