Sangamo Therapeutics Advances Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease
Rhea-AI Summary
Sangamo Therapeutics (NASDAQ: SGMO) advanced a rolling Biologics License Application (BLA) submission to the U.S. FDA for isaralgagene civaparvovec (ST-920) for adults with Fabry disease on March 9, 2026. Sangamo has submitted preclinical and clinical modules and previously initiated the rolling submission in December 2025.
The company also submitted an antibody assay companion diagnostic to FDA CDRH seeking PMA. The STAAR registrational study showed a positive mean annualized eGFR slope at 52 weeks, which FDA agreed may serve as an endpoint to support accelerated approval.
Positive
- FDA agreed the STAAR study’s 52-week mean annualized eGFR slope may support accelerated approval
- Company submitted preclinical and clinical BLA modules, advancing the rolling submission begun in Dec 2025
- Companion antibody assay submitted to FDA CDRH seeking PMA, aligning diagnostic and therapeutic regulatory paths
Negative
- BLA is incomplete; rolling submission remains ongoing and final approval is not granted
- Companion diagnostic is submitted for PMA but not yet approved, leaving patient screening dependent on regulatory outcome
News Market Reaction – SGMO
In the Mar 9 session, SGMO gained 8.66%, reflecting a notable positive market reaction. Argus tracked a peak move of +4.8% during that session. Our momentum scanner triggered 16 alerts that day, indicating notable trading interest and price volatility.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Historical Context
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Feb 03 | Fabry data update | Positive | -7.6% | Detailed STAAR Phase 1/2 Fabry data and accelerated approval path for ST-920. |
| Feb 03 | Equity offering | Negative | -31.3% | Underwritten equity and warrant offering to raise about $25.0M in gross proceeds. |
| Dec 18 | Rolling BLA start | Positive | -1.9% | Initiation of rolling BLA submission for ST-920 using eGFR slope endpoint. |
| Dec 02 | Fast Track status | Positive | +3.5% | FDA Fast Track designation for ST-503 in small fiber neuropathy STAND study. |
| Nov 21 | BLA rolling accepted | Positive | +10.0% | FDA acceptance of rolling BLA for ST-920 and reaffirmed accelerated approval path. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Regulatory and clinical Fabry updates have often been positive yet sometimes met with negative or muted price reactions, while financing news aligned with a sharp selloff.
Over the last several months, Sangamo has focused on regulatory and clinical progress for ST-920 in Fabry disease. On Nov 21, 2025, the FDA accepted a rolling BLA request with eGFR slope supporting an accelerated pathway, followed by initiation of the rolling submission on Dec 18, 2025. Detailed STAAR data and a clear approval path were highlighted again on Feb 3, 2026. However, a $25.0M underwritten offering on the same date led to a large negative price reaction, underscoring financing overhang alongside continued clinical and regulatory momentum.
Key Terms
biologics license application regulatory
gene therapy medical
fabry disease medical
companion diagnostic medical
premarket approval regulatory
pma regulatory
cdrh regulatory
AI-generated analysis. How Rhea-AI works. Not financial advice.
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 (α-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
