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Pfizer: hemophilia gene therapy shows promise in study

(CercleFinance.com) - A gene therapy for severe haemophilia A developed by Pfizer and Sangamo Therapeutics showed "promising" initial results, the companies said on Monday.


The Phase 1/2 data from 10 patients showed that the investigational SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) activity levels, Pfizer and Sangamo said.

The first two patients 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, they noted.

The two patients more recently treated are demonstrating FVIII activity that appears consistent with the first two patients, the drugmakers said.

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