Sangamo Therapeutics (NASDAQ: SGMO) is trading higher this morning on news the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s SB-318, a genome editing product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I), a rare lysosomal storage disorder. The FDA grants orphan drug designation to drugs and biologics intended to treat rare diseases, providing incentives to advance development of rare disease drugs as well as commercialization of drugs that progress to approval. Sangamo also noted its plans to “conduct the first ever in vivo genome editing clinical trials including Phase 1/2 studies for three lead programs.”
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About Sangamo
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients’ lives using the company’s platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The company’s proprietary zinc finger nuclease (ZFN) in vivo genome editing approach is being evaluated in phase 1/2 clinical trials to treat hemophilia B and lysosomal storage disorders MPS I and MPS II. Sangamo also plans this year to conduct a phase 1/2 clinical trial to evaluate its AAV cDNA human Factor 8 gene therapy approach, SB-525, to treat hemophilia A. Sangamo has a strategic collaboration with Biogen, Inc. for hemoglobinopathies, including sickle cell disease and beta-thalassemia, and with Shire plc to develop therapeutics for Huntington’s disease. In addition, Sangamo has phase 1/2 and phase 2 clinical programs in HIV/AIDS (SB-728). It has established strategic partnerships with companies in non-therapeutic applications of its technology, including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company’s website at www.sangamo.com.
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