Sangamo reports positive Phase 1/2 data for Fabry gene therapy ST-920; BLA plan Q1 2026

SANGAMO THERAPEUTICS, INC

Mean annualized eGFR slope +1.965 at 52 weeks across 32 patients; +1.747 at 104 weeks for 19 patients.
All 18 patients on ERT at baseline withdrawn from ERT and remain off; α-Gal A activity sustained up to 4.5 years.
Reported eGFR slope compares favorably to Replagal (-2.2), Fabrazyme (-1.5), Galafold (-0.4) in published studies.
Significant SF-36 quality-of-life improvements: role-physical +14.8, vitality +9.6, bodily pain +9.0 at week 52.
No preconditioning required; most adverse events grade 1-2; no safety-related discontinuations.

item 8.01

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