other material
confidence high
sentiment positive
materiality 0.85
Sangamo reports updated Phase 1/2 STAAR data for ST-920; eGFR slope +3.061; FDA Accelerated Approval path confirmed
SANGAMO THERAPEUTICS, INC
- 33 patients treated; gene therapy ST-920 generally well-tolerated; no deaths or discontinuations.
- ERT-naïve/pseudo-naïve patients showed sustained α-Gal A activity up to 42 months; 17/18 ERT patients remain off ERT.
- 23 patients with ≥12 months follow-up: mean annualized eGFR slope +3.061 (95% CI 0.863-5.258), indicating potential kidney improvement.
- FDA agreed to Accelerated Approval using 52-week eGFR slope; BLA submission expected H2 2025.
- Significant QoL (SF-36 General Health +10.6, p=0.0020) and GI symptom improvements; anti-α-Gal A antibodies reduced/eliminated in 9/10 patients.