SB- Fix receives orphan drug designation for Hemophilia B
FDA announces orphan drug designation status to SB-FIX .
Release Date: 09-Oct-2016
FDA announces orphan drug designation status to SB-FIX , zinc finger nuclease (ZFN)-mediated genome editing product for the treatment of hemophilia from Sangamo Biosciences. The company has aces in therapeutic genome editing.
SB-FIX is designed as a single treatment strategy intended to provide stable, continuous production of Factor IX clotting protein (FIX) for the lifetime of the patient. Sangamo's ZFN-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic "safe-harbor site," that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein product.
Hemophilia B is a rare bleeding disorder caused by mutations in genes that encode factors which help the blood clot and stop bleeding when blood vessels are injured. The patients of Hemophilia B experience continuous bleeding after injuries which leads to joint diseases such as arthritis.
FDA grants orphan drug designation for certain rare and life threatening diseases which affects less than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and provides several benefits to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.
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