Sangamo Therapeutics Inc (NASDAQ: SGMO) made its way up the charts Friday morning, on revealing that it has received Fast Track designation from the FDA for SB-318 and SB-913.
The U.S. Food and Drug Administration’s (FDA) 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.
A release issued Friday morning by the drug maker, based in Richmond, California, said 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.
Sangamo is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company has open Phase 1/2 clinical trials in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II.
Sangamo stock sang Friday morning, gaining 55 cents, or 5.8%, to $10.05.
