New gene therapy hemophilia hope sends Sangamo shares upwards

Genomic medicine company Sangamo Therapeutics (Nasdaq: SGMO) and its pharma partner Pfizer (NYSE: PFE) have announced updated follow-up results from the Phase I/II Alta study on the SB-525 gene therapy in patients with severe hemophilia A.

The data, presented at ASH, showed that SB-525 was generally well-tolerated and demonstrated sustained increased Factor VIII (FVIII) levels following treatment with SB-525 through to 44 weeks, the extent of follow-up for the longest treated patient in the 3e13vg/kg dose cohort.

Barbara Konkle, Bloodworks Northwest, professor of Medicine at University of Washington and a principal investigator of the Alta study, said: “I am pleased that all five patients in the high-dose (3e13 vg/kg) cohort rapidly achieved normal levels of Factor VIII, and that Factor VIII levels have been stable and durable in the normal range for the first two patients up to 44 and 37 weeks following treatment respectively, with no bleeding events or factor usage up to a follow-up of 44 weeks in the longest treated patient.