(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.
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