Posted by Michael Wonder on 14 Jul 2017
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
13 July 2017 - Sangamo Therapeutics announced today that the U.S. 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.
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 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 Paediatric 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 enrolment.
