Sangamo Therapeutics Q4 Earnings Call Highlights
by Amy Steele · The Cerbat GemSangamo Therapeutics (NASDAQ:SGMO) executives used the company’s fourth-quarter and full-year 2025 earnings call to highlight progress across its Fabry disease and neurology pipelines, while repeatedly emphasizing that additional funding and a potential commercial partner remain central to the company’s ability to execute its plans.
Fabry gene therapy advances with rolling BLA submission
Chief Executive Officer Sandy Macrae said Sangamo reported “positive top-line results” in June from the registrational Phase 1/2 STAAR study of ST-920 (isaralgagene civaparvovec) in adults with Fabry disease. Macrae said the U.S. Food and Drug Administration (FDA) reiterated in October 2025 that the study’s 52-week mean annualized estimated glomerular filtration rate (eGFR) slope “may serve as the primary basis of approval under an accelerated approval pathway.”
Chief Development Officer Nathalie Dubois-Stringfellow said Sangamo initiated a rolling Biologics License Application (BLA) submission to the FDA in December and has now submitted both the non-clinical and clinical modules. She added that an antibody assay companion diagnostic designed to screen patient eligibility has been submitted to, and accepted by, the FDA’s Center for Devices and Radiological Health (CDRH) for pre-market approval.
Dubois-Stringfellow identified completion of the chemistry, manufacturing and controls (CMC) module as the next key milestone. She said Sangamo has completed manufacturing and testing of process validation lots “with acceptable results achieved,” has completed method validation, and has manufactured its “first commercial lot.” She added the company anticipates completing the full BLA submission “as early as this summer,” but qualified that timing as “subject to our ability to secure adequate additional funding.”
Clinical readouts spotlight kidney and cardiac observations
Dubois-Stringfellow pointed to data presented in February at the WORLDSymposium, including what she described as improved kidney function in the STAAR study. She said the company observed a “positive mean annualized eGFR slope at one year across all those patients in the study, and at two years for 19 patients,” which she characterized as a departure from “the historical renal decline characteristic of the disease.”
She also said the company saw “stabilization in cardiac function,” including stability of cardiac structure and cardiac biomarkers, calling that “especially encouraging” given cardiovascular disease is “the most common cause of death” in Fabry patients. Dubois-Stringfellow added that Sangamo presented additional pharmacology and immunogenicity findings and referenced a fertility and embryo-fetal development study as well as work related to AAV integration and germline transmission risk in mice.
Dubois-Stringfellow said the program has shown a “well-tolerated safety profile,” the ability for patients to withdraw from enzyme replacement therapy, and other clinical benefits, supporting Sangamo’s view that ST-920 could be a “one-time durable treatment option.”
CMC and cash management drive timing; partnership search continues
In the question-and-answer session, Macrae told analysts that CMC work has been “on the critical path” for the Fabry filing. He said that after receiving guidance that an accelerated approval filing could be supported by a single study, Sangamo “had to hustle to complete the CMC activity that would normally be performed over phase II and phase III.”
Macrae also described “helpful interactions” with FDA’s CMC group, including recent “clear, detailed pages of guidance” on what is needed to complete the submission and “maximize its chance of success.” He said the company is following that guidance closely, noting that CMC can be “the piece that is most challenging for cell and gene therapies.”
Macrae added that Sangamo is also “managing our cash very carefully” and moderating spending to preserve runway while it seeks a Fabry partnership. He said that combination—agency requirements and “wise, prudent spending”—has extended the CMC timeline compared with prior expectations.
On business development, Macrae reiterated that “securing a commercial partner for Fabry remains our number one focus.” He said Sangamo has been in discussions for “18 months” with various parties, but that “most of the ones we spoke to last year have now gone,” often due to “regulatory uncertainty.” He said Sangamo is now in discussions with “multiple partners” and described those conversations as constructive, though he noted the counterparties are “new to the discussions” and therefore require due diligence, management presentations, and negotiations.
Asked whether additional FDA engagement is planned around the eGFR endpoint and recent changes at the agency, Macrae said Sangamo last spoke to FDA in October and believes FDA reiterated that “the eGFR at one year could be used to file for accelerated approval.” He said the company has not returned to the agency since then and does not plan to, given the rolling submission is already underway. “If the agency changes or their way of dealing with gene therapy changes, we would of course address that,” Macrae said, adding that Sangamo believes it has sufficient guidance to proceed.
Neurology pipeline expands with Fast Track and site activations
Sangamo also emphasized its shift toward neurology. Macrae said the company “transitioned to become a clinical-stage neurology company” with six activated sites in the Phase 1/2 STAND study evaluating ST-503 in chronic neuropathic pain.
Dubois-Stringfellow said ST-503 received FDA Fast Track designation in December for patients with “intractable pain due to small fiber neuropathy (SFN).” She said the designation enables more frequent interaction with FDA and that the program “may also be eligible to apply for accelerated approval and priority review if relevant criteria are met.” She said Sangamo has activated four additional sites since the prior update to reach “a total of six active sites,” which are now identifying patients.
Dubois-Stringfellow also noted that a manuscript describing the preclinical safety and pharmacology of ST-503 in human neurons, mice, and non-human primates was published in Science Translational Medicine, calling it part of the preclinical foundation for the ongoing study.
For ST-506, the company’s epigenetic regulator program for prion disease delivered intravenously using its neurotropic STAC-BBB capsid, Dubois-Stringfellow said CTA-enabling work is ongoing. She said the GLP toxicology study has been completed and analysis is underway.
Capsid licensing momentum and funding raised since early 2025
Macrae said Sangamo has continued to position itself as a “collaborator of choice” for neurotropic capsids, citing the April announcement of a third neurology capsid license agreement with Eli Lilly for up to five central nervous system disease targets.
He also said the company has raised “over $130 million in funding since the start of 2025” through a combination of non-dilutive license fees and milestone payments, as well as equity financing.
In response to a question about why AAV9 is being used for the neuropathic pain program rather than the STAC-BBB capsid, Macrae said the pain program began “four or five years ago before the STAC-BBB capsid was known” and that AAV9 is effective when administered intrathecally to target the dorsal root ganglion, adding that AAV9 has been used and “seen to be safe and effective.” On STAC-BBB, he said Sangamo is “very pleased” to see large neurology companies choose the capsid and that Sangamo is working with each partner to help advance their programs.
Management concluded the call by underscoring that, despite pipeline progress, the company’s long-term runway remains unresolved and is closely tied to its efforts to secure additional capital and a Fabry commercialization partner.
About Sangamo Therapeutics (NASDAQ:SGMO)
Sangamo Therapeutics, Inc is a clinical-stage biotechnology company headquartered in Brisbane, California, that specializes in the development of genomic therapies based on its proprietary zinc finger nuclease (ZFN) technology. Founded in 1995, Sangamo pioneered ZFN-based genome editing to precisely alter DNA sequences for the treatment of serious genetic and rare diseases. The company’s platform encompasses in vivo genome editing, ex vivo cell therapy, and genome regulation approaches, with a focus on durable therapeutic effects through permanent genetic modification or sustained gene expression control.
Through its genome editing programs, Sangamo is advancing multiple product candidates into clinical trials for conditions such as hemophilia A and B, mucopolysaccharidosis types I and II, and lysosomal storage disorders.