The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here.
Sangamo Therapeutics Inc., a genomic medicine company, and Pfizer Inc. announced updated results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A.
The data showed that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) activity levels. The first two patients treated at the 3e13 vg/kg dose rapidly achieved normal levels of FVIII activity as measured using a chromogenic assay, with no reported bleeding events, and the response continues to be durable for as long as 24 weeks, the extent of follow-up. The two patients more recently treated at the 3e13 vg/kg dose level are demonstrating FVIII activity kinetics that appear consistent with the first two patients treated in this dose cohort at similar early time points. Data from 10 patients treated with SB-525 were presented during an oral presentation on July 6 at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH), in Melbourne, Australia. The SB-525 ISTH presentation slides, which include the full data set, are available on Sangamo’s website in the Investors and Media section under Events and Presentations.
“The initial results with SB-525 gene therapy for patients with severe hemophilia A continue to look very promising,” said Barbara Konkle, M.D., Bloodworks Northwest, Professor of Medicine at University of Washington and a Principal Investigator of the Alta study. “It is encouraging that patients in the 3e13 vg/kg cohort have attained normal Factor VIII levels within 5-7 weeks of treatment with SB-525 gene therapy and have sustained Factor VIII activity with no bleeding episodes. It will be important to continue to follow these patients to understand the potential long-term durability of this gene therapy.”
Read the press release in its entirety here.Â