Sangamo Therapeutics (NASDAQ:SGMO) is up 14% premarket on light volume in response to updated results from the Phase 1/2 ALTA study evaluating gene therapy SB-525 in patients with severe hemophilia A. The data were presented at the International Society on Thrombosis and Hemostasis Congress in Melbourne.
The first two patients treated at the 3e13 vg/kg dose level rapidly achieved normal sustained factor VIII levels with no reported bleeding events and no factor usage during the 24 weeks of follow-up. Two additional patients recently treated showed comparable results at similar early time points.
SB-525 was generally well-tolerated and demonstrated dose-dependent increases in factor VIII activity levels.
Collaboration partner Pfizer (NYSE:PFE) is unchanged in premarket action.
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