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Pfizer, Sangamo Report Encouraging Findings From Phase 1/2 Study Of Giroctocogene Fitelparvovec

(RTTNews) - Pfizer Inc. (PFE) and Sangamo Therapeutics, Inc. (SGMO) announced updated follow-up data from the phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with severe hemophilia A. All five patients in the high dose 3 x 1013 vg/kg cohort have had at least one year of follow-up and showed sustained factor VIII (FVIII) activity levels. Steady-state FVIII activity was achieved for all patients in the 3 x 1013 vg/kg cohort within 9 weeks of treatment. Giroctocogene fitelparvovec was generally well tolerated.

Seng Cheng, Chief Scientific Officer of Pfizer's Rare Disease Research Unit, stated: "With the first patient dosed in the Phase 3 AFFINE study in October 2020, we are on track for a readout from this pivotal Phase 3 trial in 2022, which will allow us to better assess the potential of our gene therapy across a larger sample size."

The companies plan to present further follow-up data from the Alta study when all five patients in the 3 x 1013 vg/kg dose cohort have been followed for at least two years.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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