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Sangamo Receives Fast Track Designation From The FDA For SB-318 And SB-913 In Vivo Genome Editing Product Candidates For The Treatment Of MPS I And MPS II

RICHMOND, Calif., July 13, 2017 /PRNewswire/ -- Sangamo Therapeutics, Inc. (SGMO) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SB-318 and SB-913, the Company's clinical stage in vivo genome editing product candidates for the treatment of Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively. The FDA's Fast Track designation is designed to facilitate the development and expedite the review of drugs and biologics to treat serious conditions and fill an unmet medical need. Once a drug receives Fast Track designation, early and frequent communication with the FDA is encouraged throughout the development and review process. The frequency of communication is designed to ensure that questions and issues are resolved quickly, potentially leading to earlier drug approval and access by patients.

Sangamo Therapeutics, Inc. (PRNewsFoto/Sangamo BioSciences, Inc.) (PRNewsFoto/)

MPS I and MPS II are caused by mutations in the genes encoding alpha-L-iduronidase (IDUA) and iduronate 2-sulfatase (IDS) enzymes, respectively. Using Sangamo's zinc finger nuclease (ZFN) genome editing technology, SB-318 (for MPS I) and SB-913 (for MPS II) are designed as a single treatment strategy intended to provide stable, continuous production of the IDUA or IDS enzyme for the lifetime of the patient.

SB-318 and SB-913 have already received Orphan Drug and Rare Pediatric Disease designations from the FDA. The FDA has cleared an Investigational New Drug application for these programs, and Phase 1/2 clinical trials evaluating SB-318 and SB-913 in adults with MPS I and MPS II, respectively, are open and screening subjects for enrollment.

Sangamo's In Vivo Genome Editing...


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