FDA Approves Sangamo's Proposal for Rolling BLA Submission of Gene Therapy Candidate for Fabry Disease
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Nov 21 2025
0mins
Source: NASDAQ.COM
FDA Acceptance: Sangamo Therapeutics announced that the FDA has accepted its request for a rolling submission of the Biologics License Application for isaralgagene civaparvovec, a gene therapy for Fabry disease.
Study Results: The Phase 1/2 STAAR study indicated that isaralgagene civaparvovec could provide a one-time, durable treatment with significant multi-organ benefits, surpassing current care standards.
Regulatory Designations: The therapy has received multiple designations from regulatory agencies, including Orphan Drug and Fast Track status from the FDA, and similar recognitions from the European Medicines Agency and U.K. authorities.
Submission Timeline: Sangamo plans to initiate the rolling submission of the BLA under the accelerated approval pathway in the fourth quarter of 2025.
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Analyst Views on SGMO
Wall Street analysts forecast SGMO stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for SGMO is 3.75 USD with a low forecast of 1.00 USD and a high forecast of 10.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
4 Analyst Rating
1 Buy
3 Hold
0 Sell
Hold
Current: 0.364
Low
1.00
Averages
3.75
High
10.00
Current: 0.364
Low
1.00
Averages
3.75
High
10.00
About SGMO
Sangamo Therapeutics, Inc. is a genomic medicine company. The Company’s zinc finger (ZF) epigenetic regulators are ideally suited to potentially address neurological disorders and its capsid engineering platform has demonstrated the ability to expand delivery beyond available intrathecal delivery capsids, including in the central nervous system (CNS), in preclinical studies. Zinc finger technologies are derived from naturally occurring human proteins. They are composed of a DNA-binding domain, which binds with precision to any sequence of DNA, and various functional domains (such as nucleases and transcription factors) based on therapeutic needs. Its pipeline includes Chronic Neuropathic Pain - ST-503 and Isaralgagene civaparvovec (ST-920). ST-503 is an investigational epigenetic regulator for the treatment of intractable pain due to iSFN, a type of chronic neuropathic pain. ST-920 is a wholly owned gene therapy product candidate for the treatment of Fabry disease.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Sangamo Initiates BLA Submission for ST-920, Expected in Q2 2026
- Clinical Trial Success: Sangamo's STAAR study demonstrated a positive mean annualized eGFR slope at 52 weeks across all dosed patients, with the FDA agreeing to use this as an endpoint for accelerated approval, indicating ST-920's potential as an effective treatment for Fabry disease patients.
- Favorable Safety Profile: Isaralgagene civaparvovec exhibited a favorable safety and tolerability profile in clinical trials, suggesting its potential as a one-time gene therapy that can provide durable clinical benefits, surpassing current treatment standards for Fabry disease.
- Accelerated Approval Pathway: Sangamo expects to complete its Biological License Application (BLA) submission to the FDA in the second quarter of 2026 using a rolling submission approach, allowing for ongoing review of completed modules, thereby expediting the approval process to meet urgent patient needs.
- Regulatory Support: Isaralgagene civaparvovec has received Orphan Drug, Fast Track, and RMAT designations from the FDA, along with Orphan Medicinal Product designation from the European Medicines Agency, highlighting its potential value and regulatory recognition in global markets, further enhancing Sangamo's competitive position.
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Sangamo Initiates BLA Submission for ST-920, Expected Completion in Q2 2026
- Clinical Trial Results: Sangamo's STAAR study demonstrated a positive mean annualized eGFR slope at 52 weeks across all dosed patients, with the FDA agreeing to use this as an endpoint for accelerated approval, indicating ST-920's potential in treating Fabry disease.
- Safety Profile: Isaralgagene civaparvovec has shown a favorable safety and tolerability profile, expected to provide a safe and durable treatment option for Fabry patients, significantly improving multi-organ clinical benefits.
- Submission Timeline: Sangamo plans to complete its Biological License Application (BLA) submission in Q2 2026 using a rolling submission approach, allowing the FDA to review completed modules continuously, thus expediting the approval process.
- Regulatory Support: The therapy has received Orphan Drug, Fast Track, and RMAT designations from the FDA, reflecting regulatory recognition of its potential market value and enhancing Sangamo's competitive position in the gene therapy sector.
Continue Reading