Sangamo Therapeutics Advances Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease
MWN-AI** Summary
Sangamo Therapeutics, Inc. has advanced its rolling submission of a Biologics License Application (BLA) to the U.S. FDA seeking accelerated approval for isaralgagene civaparvovec (ST-920), an investigational gene therapy for adult patients with Fabry disease. The rolling submission, which commenced in December 2025, allows Sangamo to submit completed portions of the BLA incrementally for FDA review. Recently, the company submitted both preclinical and clinical modules, demonstrating their commitment to expediting the approval process.
The FDA has agreed to use data from the STAAR study, which showcased a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients, as an endpoint for accelerated approval. This finding suggests that ST-920 offers a potentially transformative treatment option for Fabry disease, which is characterized by serious multi-organ complications resulting from the buildup of globotriaosylceramide due to enzyme deficiency.
Isaralgagene civaparvovec is particularly notable for being a one-time infusion therapy that does not require preconditioning, and it has already received several designations from regulatory agencies, including Orphan Drug and Fast Track from the FDA. The STAAR study is a global Phase 1/2 trial designed to evaluate the safety and efficacy of this novel therapy in treating Fabry disease.
Sangamo believes that the accumulated data from the STAAR trial indicate a well-tolerated and durable therapeutic option that could significantly alter the current treatment landscape for Fabry disease. As they move forward, the company anticipates that this innovative gene therapy could provide meaningful benefits across multiple organ systems in affected patients.
MWN-AI** Analysis
Sangamo Therapeutics (Nasdaq: SGMO) presents an intriguing investment opportunity following the advancement of its rolling Biologics License Application (BLA) submission for isaralgagene civaparvovec (ST-920), a promising gene therapy for Fabry disease. The strategic maneuver of initiating a rolling submission allows for the ongoing review of completed modules by the FDA, potentially accelerating the trajectory toward approval. This approach optimally positions Sangamo amid evolving regulatory landscapes, especially as the FDA has acknowledged the mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks as a valid endpoint supporting the application.
The positive outcomes from the STAAR study bolster Sangamo's value proposition. The ability of isaralgagene civaparvovec to provide a one-time, well-tolerated treatment option with multi-organ clinical benefits could indeed revolutionize the predominant Fabry treatment paradigm. Investors should monitor the forthcoming clinical data, particularly the anticipated results of the 104-week follow-up, as these will be critical in substantiating the treatment's durability and efficacy.
Additionally, with established designations such as Fast Track and Orphan Drug by the FDA, Sangamo is positioned favorably to tap into niche markets. However, investors must remain cognizant of the inherent risks. The landscape for biotech investments can be volatile, characterized by dependencies on clinical trial outcomes and regulatory approvals that can fluctuate significantly. Sangamo's highlighted need for further capital and potential partnerships could impact its operational stability and the pace at which it can move forward.
In summary, while Sangamo's pipeline offers considerable upside, potential investors should conduct a thorough risk assessment and stay informed on clinical progress and regulatory responses. Monitoring key milestones will be essential in assessing whether Sangamo can realize its transformative vision for Fabry disease treatment.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
Data support potential of isaralgagene civaparvovec as a one-time, well tolerated and durable Fabry disease gene therapy to provide meaningful, multi-organ clinical benefits that could fundamentally shift Fabry treatment paradigm
STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) has agreed will serve as an endpoint to support accelerated approval pathway
RICHMOND, Calif., March 09, 2026 (GLOBE NEWSWIRE) -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced advancement of the rolling submission of a BLA to the FDA seeking accelerated approval of isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease.
Following initiation of the rolling submission in December 2025, Sangamo has now submitted the preclinical and clinical modules to the FDA for review. Rolling submission allows for completed modules of the BLA to be submitted and reviewed by the FDA on an ongoing basis rather than waiting for the entire BLA to be submitted at once. In addition, the antibody assay companion diagnostic, which is designed to screen patients for eligibility with isaralgagene civaparvovec, has been submitted to, and accepted by, the FDA’s Center for Devices and Radiological Health (CDRH) seeking Premarket Approval (PMA).
Sangamo believes that the totality of data from the registrational STAAR study demonstrates the potential of isaralgagene civaparvovec as a one-time, well-tolerated and durable gene therapy treatment option for Fabry disease to provide meaningful, multi-organ clinical benefits that could fundamentally shift the Fabry treatment paradigm. Furthermore, the STAAR study demonstrated a positive mean annualized eGFR slope at 52-weeks across all dosed patients in the study, which the FDA has agreed will serve as endpoint to support accelerated approval. These data were presented via four platform presentations and in poster presentations at the recent 22nd Annual WORLDSymposium™. These data are available on Sangamo’s website on the Presentations page.
About the STAAR Study
The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning. Isaralgagene civaparvovec has been granted Orphan Drug, Fast Track and RMAT designations from the FDA, Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (?-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities.
About Sangamo Therapeutics
Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and that its capsid discovery platform can expand delivery beyond currently available intrathecal delivery capsids, including in the central nervous system. Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with us on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements regarding Sangamo’s current expectations. These forward-looking statements include, without limitation, statements relating to: the safety and efficacy and therapeutic potential of isaralgagene civaparvovec, including the potential for it to be a one-time, durable treatment option for Fabry disease to provide meaningful, multi-organ clinical benefits that could fundamentally shift Fabry treatment paradigm; the potential for isaralgagene civaparvovec to qualify for and receive approval under the FDA’s accelerated approval program, including the adequacy of data generated in the Phase 1/2 STAAR study to support any such approval; expectations concerning the availability of additional data to support a potential BLA submission for isaralgagene civaparvovec; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to Sangamo’s lack of capital resources to obtain regulatory approval for and commercialize its product candidates in a timely manner or at all, including the ability to secure a commercialization partner for ST-920; the uncertain timing and unpredictable nature of clinical trial results, including the risk that preliminary or topline data is not indicative of final results, that the therapeutic effects observed in the latest clinical data from the Phase 1/2 STAAR study will not be durable in patients and that final clinical trial data from the study will not validate the safety and efficacy of isaralgagene civaparvovec, including that the 52-week data from the Phase 1/2 STAAR study will not support a BLA submission and/or that the 104-week data from such study will not verify the clinical benefit of isaralgagene civaparvovec or support FDA approval, and that the patients withdrawn from ERT will remain off ERT; Sangamo’s need for substantial additional funding to execute its operating plan and to continue to operate as a going concern; the effects of macroeconomic factors or financial challenges on the global business environment, healthcare systems and Sangamo’s business and operations; the research and development process; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; the potential for technological developments that obviate technologies used by Sangamo; Sangamo’s reliance on collaborators and the potential inability to secure additional collaborations; and Sangamo’s ability to achieve expected future financial performance.
All forward-looking statements about Sangamo’s future plans and expectations, including Sangamo’s development plans for its product candidates, are subject to Sangamo’s ability to secure adequate additional funding. There can be no assurance that Sangamo and its current or potential future partners will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and its collaborators. These risks and uncertainties are described more fully in Sangamo’s Securities and Exchange Commission, or SEC, filings and reports, including in Sangamo’s Annual Report on Form 10-K for the year ended December 31, 2024, as supplemented by its Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, each filed with the SEC, and future filings and reports that Sangamo makes from time to time with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.
Contacts
Investor Relations and Media Inquiries
Louise Wilkie
ir@sangamo.com
media@sangamo.com
FAQ**
How does Sangamo Therapeutics Inc. (SGMO) plan to utilize the positive eGFR slope data from the STAAR study to support the regulatory approval of isaralgagene civaparvovec in treating Fabry disease?
In what ways does Sangamo Therapeutics Inc. (SGMO) envision isaralgagene civaparvovec fundamentally shifting the treatment paradigm for Fabry disease beyond just the current standard of care?
What specific measures is Sangamo Therapeutics Inc. (SGMO) taking to ensure the durability and long-term safety of isaralgagene civaparvovec post-approval, especially regarding the clinical benefits observed in the STAAR study?
How does Sangamo Therapeutics Inc. (SGMO) plan to address potential risks related to funding and regulatory uncertainties as they progress toward commercializing isaralgagene civaparvovec for Fabry disease treatment?
**MWN-AI FAQ is based on asking OpenAI questions about Sangamo Therapeutics Inc. (NASDAQ: SGMO).
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