Sangamo reports updated Phase 1/2 Alta data for hemophilia A gene therapy; FVIII maintained through ~4 years

SANGAMO THERAPEUTICS, INC

Single infusion of giroctocogene fitelparvovec (3e13 vg/kg) maintains FVIII activity in mild-to-normal range through up to 208 weeks.
Mean annualized bleeding rate in high-dose cohort was 0 in year 1 and 1.2 overall; no participants resumed prophylaxis.
No confirmed FVIII inhibitor development, no thrombotic events, and liver safety resolved without sequelae.
Pfizer expects pivotal Phase 3 AFFINE trial readout in mid-2024.
Updated data to be presented at ASH Annual Meeting on December 11, 2023.

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