Sangamo: ST-920 gene therapy shows sustained efficacy; defers Phase 3 pending partnership

SANGAMO THERAPEUTICS, INC

As of Sep 19, 2023 cutoff, 24 patients dosed; all 12 withdrawn from ERT remain off ERT with sustained supraphysiological α-Gal A activity.
In dose escalation phase, all 9 patients had sustained elevated α-Gal A activity for nearly 3 years; 5 ERT patients completed withdrawal.
At highest dose (2.63×10^13 vg/kg), ERT-naïve patients showed supraphysiological α-Gal A up to ~500 days with lyso-Gb3 reduction/stabilization.
Mean annualized eGFR slope -0.915 mL/min/1.73m²/yr; 69% improved total FOS-MSSI; statistically significant SF-36 and GSRS improvements.
Company defers further investment in registrational trial planning until collaboration partnership or financing is secured.

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