Sangamo/Pfizer Alta study: 4-year data shows mean ABR 1.4, sustained FVIII activity

SANGAMO THERAPEUTICS, INC

Cohort 4 (3e13 vg/kg) mean ABR 1.4, median 0.0; 3 of 5 participants had zero bleeds post-infusion.
FVIII activity maintained in mild-to-normal range in 4 of 5 participants over 4-year follow-up; 1 participant had FVIII BLOQ.
No new safety signals; no thrombotic, neoplastic events, or FVIII inhibitors; LFT elevations resolved with steroids.
Pfizer expects pivotal Phase 3 AFFINE readout mid-2024.

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