Sangamo advances gene therapy for Fabry disease

RICHMOND, Calif. - Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company with a market capitalization of $162 million, has reached a significant milestone in the development of its gene therapy product candidate for Fabry disease, isaralgagene civaparvovec, also known as ST-920. According to InvestingPro data, the company faces financial challenges with negative EBITDA of $93 million in the last twelve months, reflecting the capital-intensive nature of biotech development. The company announced today that all patients in the Phase 1/2 STAAR study have completed a minimum of 52 weeks of follow-up, a requirement for the U.S. Food and Drug Administration’s (FDA) Accelerated Approval pathway. With the stock trading 77% below its 52-week high of $3.18, InvestingPro analysis indicates the company is currently undervalued, though investors should note its weak financial health score and negative profit margins.

Preliminary data suggest a positive mean estimated glomerular filtration rate (eGFR) slope at 52 weeks, indicating potential renal benefits of the therapy. This data builds on previous reports presented in February 2025. ST-920 has been well tolerated by patients, and a pivotal data readout is anticipated by the end of the second quarter of 2025.

A recent Type B meeting with the FDA in April 2025 has provided Sangamo with a clear Chemistry, Manufacturing and Controls (CMC) pathway for a planned Biologics License Application (BLA) submission scheduled for the first quarter of 2026. Approval and commercial launch could follow as early as the second half of 2026, contingent upon regulatory processes.

Nathalie Dubois-Stringfellow, Ph.D., Chief Development Officer at Sangamo, expressed optimism about the one-year mean eGFR slope data and the clarity gained from the FDA meeting, which together outline a regulatory path toward potential approval for ST-920.

The STAAR study is a global, open-label, single-dose, dose-ranging trial designed to assess the safety and tolerability of ST-920 in patients with Fabry disease, a lysosomal storage disorder characterized by enzyme deficiency leading to organ damage.

The FDA has granted ST-920 Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations. It has also received Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency (EMA) and Innovative Licensing and Access Pathway designation from the U.K. Medicines and Healthcare products Regulatory Agency.

While discussions with the EMA are ongoing, Sangamo is also engaged in business development negotiations for potential commercialization agreements.

This news is based on a press release statement and reflects the current status of Sangamo Therapeutics’ efforts to bring new treatments to patients with serious neurological diseases.

In other recent news, Sangamo Therapeutics has entered into a significant licensing agreement with Eli Lilly, granting Lilly exclusive rights to use Sangamo’s STAC-BBB capsid technology. This deal includes an $18 million upfront payment and the potential for Sangamo to earn up to $1.4 billion in additional fees and milestone payments for five potential neurology disease targets. Analysts from H.C. Wainwright have maintained a Buy rating on Sangamo with a $10 price target, underscoring confidence in the company’s strategic partnerships and pipeline. Meanwhile, Jefferies has adjusted its price target for Sangamo to $2, while still maintaining a Buy rating, reflecting a cautious view of the company’s financial outlook. Truist Securities also maintains a Buy rating, highlighting Sangamo’s progress in developing treatments for chronic neuropathic pain and prion disease, with results expected in late 2026. Sangamo is reportedly in advanced talks for another potential partnership for its STAC-BBB program, which could extend its financial runway beyond 2026. The company is also on track to present one-year eGFR data by mid-2025 and plans to file for accelerated approval for its Fabry disease program later that year. Sangamo’s current financial position includes $42 million in cash, expected to support operations until mid-2025.

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