other material
confidence high
sentiment neutral
materiality 0.80
Sangamo reports positive eGFR slopes in Phase 1/2 STAAR gene therapy for Fabry; BLA planned Q1 2026
SANGAMO THERAPEUTICS, INC
- Mean annualized eGFR slope of +1.965 mL/min/1.73m2/yr (95% CI: -0.153, 4.083) at 52 weeks across 32 patients.
- All 18 patients on ERT at baseline safely withdrew; one later resumed per physician's decision.
- 69% of patients improved FOS-MSSI severity score at 12 months; SF-36 QoL scores showed statistically significant gains.
- BLA submission for isaralgagene civaparvovec targeted Q1 2026 under Accelerated Approval pathway, subject to funding.
- Company notes need for substantial additional capital to continue as going concern and advance Fabry program.