Sangamo Files BLA After 1.965 mL/min eGFR Gain and Reports $37.4M Q4 Loss
Sangamo reported a 1.965 mL/min/1.73m2 mean annualized eGFR slope at 52 weeks in its 32-patient STAAR Fabry study and has initiated rolling BLA submission for isaralgagene civaparvovec under the FDA Accelerated Approval pathway. It posted a Q4 net loss of $37.4M on $14.2M revenues and secured over $130M since early 2025.
1. Fabry Disease Trial and BLA Submission
In its registrational STAAR study for Fabry disease, Sangamo achieved a positive mean annualized eGFR slope of 1.965 mL/min/1.73m2 at 52 weeks across 32 dosed patients, with durable α-Gal A activity maintained up to 4.5 years and stable cardiac function. The company has begun rolling submission of a BLA for isaralgagene civaparvovec under the FDA Accelerated Approval pathway, advancing its CMC module ahead of a full filing expected by summer 2026, subject to securing additional funding.
2. Neurology Pipeline Expansion
Sangamo has transitioned into a clinical-stage neurology company by activating six sites in the Phase 1/2 STAND study of ST-503 for small fiber neuropathy, which holds FDA Fast Track designation. The prion disease candidate ST-506 has completed GLP toxicology studies as CTA-enabling activities proceed, and the company secured its third STAC-BBB capsid license agreement with Eli Lilly to deliver genomic medicines against up to five CNS targets.
3. Financial Performance and Funding
For Q4 2025, Sangamo posted a net loss of $37.4 million, or $0.11 per share, on revenues of $14.2 million, driven by a $6.0 million Pfizer license option, Astellas collaboration fees and capsid licensing revenue. Full-year 2025 net loss was $122.9 million on $39.6 million revenues, down from $57.8 million in 2024, and the company raised over $130 million since early 2025, including $25 million from an underwritten equity offering, to fund its pipeline.