Sangamo Therapeutics, Inc., a clinical stage biopharmaceutical company, focused on the research, development, and commercialization of engineered DNA-binding proteins as novel therapeutic products for various monogenic and infectious diseases with unmet medical needs. The company was founded in 1995 and is based in Richmond, California.
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All five patients in an early-stage trial given the one-time treatment, giroctocogene fitelparvovec, did not have spontaneous bleeding episodes or require infusions of the blood-clotting protein they otherwise lack, the companies said. Pfizer and Sangamo's therapy is one of several treatments being developed for hemophilia A, as an alternative to the current practice that requires regular infusions to replace the missing protein, factor VIII. On Wednesday, a rival gene therapy by BioMarin Pharmaceutical Inc was found to reduce bleeding episodes by over 90% in patients treated four years ago.
Pfizer Inc. (NYSE:PFE) and Sangamo Therapeutics, Inc. (Nasdaq:SGMO), a genomic medicines company, today announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec (SB-525, or PF-07055480), an investigational gene therapy for patients with severe hemophilia A. All five patients with severe hemophilia A who received the 3e13 vg/kg dose showed sustained factor VIII (FVIII) activity levels, with a median of 64.2% via chromogenic assay (patient-level geometric means after week 9 post-infusion). No patients experienced bleeding events or required FVIII infusions. The factor VIII activity levels reflect measurements up to 61 weeks, the extent of follow-up for the longest-treated patient in the cohort. These data are being presented today as a late-breaking or...