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Sangamo Therapeutics (SGMO) orphan drug designation to SB-318

January 11, 2017 8:29am

For the treatment of Mucopolysaccharidosis Type I (MPS 1)

WHY a BUY - news, the stock is UP +$0.20 in the pre-market

The U.S. FDA has granted orphan drug designation to SB-318, a genome editing product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I), a rare lysosomal storage disorder.

SGMO's zinc finger nuclease (ZFN) genome editing technology, SB-318 is designed as a single treatment strategy intended to provide stable, continuous production of the IDUA enzyme for the lifetime of the patient.

The Bottom Line: Orphan drug designations are granted to drugs and biologics intended to treat rare diseases. The designation provides incentives to advance development of rare disease drugs and for commercialization of those drugs that progress to approval.

MPS I is caused by mutations in the gene encoding the alpha-L-iduronidase (IDUA) enzyme.

SGMO closed DOWN -$0.05 to $3.65 and is UP in the pre-market $0.20 or +5.48%

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