Positive STAAR registrational results (Fabry disease)
Top-line STAAR study results showed a positive mean annualized eGFR slope at 52 weeks across all study patients and sustained positive eGFR slope at 2 years for 19 patients, indicating improved/stabilized kidney function versus historical decline in Fabry disease. No percentage changes were disclosed.
Rolling BLA submission initiated for ST-920
Company initiated and is progressing a rolling Biologics License Application (BLA) to the U.S. FDA for ST-920 under an accelerated approval pathway; nonclinical and clinical modules have been submitted and the company anticipates completing the BLA submission as early as this summer, subject to securing additional funding.
Companion diagnostic accepted by FDA CDRH
The antibody assay companion diagnostic to screen patients for ST-920 eligibility has been submitted to and accepted by the FDA Center for Devices and Radiological Health (CDRH) seeking premarket approval, supporting the regulatory package for potential approval and commercialization.
CMC and manufacturing progress, first commercial lot produced
Completed manufacturing and testing of process validation lots with acceptable results, completed method validation, and manufactured the first commercial lot; the CMC module remains the primary filing gating item but substantial CMC milestones have been achieved.
Raised meaningful non-dilutive and equity funding
Since the start of 2025 Sangamo has raised over $130 million through nondilutive license fees, milestone payments and equity financings, providing additional runway and validation for asset value.
Transition to clinical-stage neurology company and STAND activation
Became a clinical-stage neurology company with activation of 6 clinical sites in the Phase I/II STAND study (4 sites activated since December) evaluating ST-503 for chronic neuropathic pain and ongoing patient identification.
Regulatory and scientific validations for neurology programs
ST-503 received FDA Fast Track designation for intractable pain due to small fiber neuropathy (SFN); preclinical safety and pharmacology manuscript for ST-503 published in Science Translational Medicine; GLP toxicology study for ST-506 completed with analysis ongoing.
Third STAC-BBB capsid license agreement with major partner
Signed a third neurology capsid license agreement (with Eli Lilly) to deliver genomic medicines for up to five CNS targets, reinforcing Sangamo's positioning as a collaborator of choice for neurotropic capsids and validating capsid technology externally.