Sangamo Therapeutics (NASDAQ:SGMO) said it continued to advance its regulatory filing for its Fabry disease gene therapy candidate while navigating a transition of its common stock to the OTCQB Venture Market following a Nasdaq delisting determination.
On the company’s first quarter 2026 conference call, Chief Executive Officer Sandy Macrae said Sangamo has made progress on the rolling biologics license application, or BLA, for ST-920, also known as isaralgagene civaparvovec, its investigational gene therapy for adults with Fabry disease. Chief Development Officer Nathalie Dubois-Stringfellow said the first two modules of the BLA have been submitted to the U.S. Food and Drug Administration.
FDA Pathway for ST-920 Remains Central Focus
Dubois-Stringfellow said ST-920 has a pathway to accelerated approval that has been agreed with the FDA, using mean annualized eGFR slope at 52 weeks across all 32 dosed patients in the company’s Phase I/II STAAR study.
She also said the FDA recently affirmed that an additional confirmatory study will not be required and that Sangamo may submit 104-week data across all dosed patients when seeking traditional approval.
“We continue to believe that the data support the potential of ST-920 as a one-time, well-tolerated, and durable treatment to provide meaningful multi-organ clinical benefit that could fundamentally shift the Fabry disease treatment paradigm,” Dubois-Stringfellow said.
The company presented detailed clinical data on ST-920 through four platform and poster presentations at the 22nd Annual WORLDSymposium in February, she added.
During the question-and-answer portion of the call, Macrae said the clinical module containing one-year data has already been submitted. He said the company is undertaking data cleaning processes to gather the full two-year dataset and will determine the appropriate venue and timing for presenting or submitting those data.
Macrae said Sangamo is now seeing patients with as much as five years of experience, describing the findings as “very stable.” Dubois-Stringfellow added that four patients are now beyond five years and have “supraphysiological” levels of alpha-Gal, while product safety “remains remarkable.” The company also said 17 patients are off enzyme replacement therapy, with some off ERT for more than three years.
Stock Moves to OTCQB as Company Plans Nasdaq Appeal
Macrae said Sangamo’s common stock moved to the OTCQB Venture Market after the company received a delisting determination from the Nasdaq Capital Market due to non-compliance with Nasdaq’s minimum bid requirements. The stock began trading on the OTCQB earlier this month under the same ticker symbol, SGMO.
The company intends to appeal the Nasdaq determination at a hearing in June, Macrae said.
“While we are disappointed by the Nasdaq delisting determination, the transition to the OTCQB market will allow Sangamo to maintain access to the public markets while we seek to advance our promising development pipeline,” Macrae said.
Macrae said Sangamo remains focused on raising additional capital and evaluating strategic options to maximize the value of its assets. He said the company continues to be supported by a global investment bank and remains engaged in business development discussions for a potential Fabry commercialization agreement.
In response to an analyst question about whether the FDA’s affirmation on the two-year eGFR data could affect business development discussions, Macrae declined to discuss specific conversations. However, he said parties evaluating the asset are likely “pleased by the recent affirmation from the agency.”
Neurology Pipeline Advances
Sangamo also provided updates on its neurology programs. Dubois-Stringfellow said six clinical sites have been activated for the Phase I/II STAND study evaluating ST-503, the company’s investigational epigenetic regulator for patients with intractable pain due to small fiber neuropathy. She said those sites are working to identify patients.
The company also highlighted a manuscript published in Science Translational Medicine detailing preclinical safety and pharmacology of ST-503 in human neurons, mice and non-human primates.
For ST-506, Sangamo’s epigenetic regulator for prion disease, Dubois-Stringfellow said the company continues to advance clinical trial application-enabling activities. She said the good laboratory practice toxicology study has been completed and analysis is ongoing.
Dubois-Stringfellow also said Sangamo held another productive interaction with the U.K.’s Medicines and Healthcare Products Regulatory Agency, including alignment on diagnostic testing, analytical validation and non-clinical safety matters.
ASGCT Presentations Highlight Platform Work
Macrae said Sangamo had three abstracts accepted to the American Society of Gene and Cell Therapy annual meeting in Boston. He said the presentations highlighted advances in zinc finger epigenetic regulation for Nav1.7 and prion disease, as well as a platform presentation on the company’s Modular Integrase technology.
Macrae said Sangamo has achieved “impressive levels of targeted integration” in multiple cell types, which he said moves the company closer to its goal of large-scale genome engineering to integrate large DNA sequences into the genome.
In closing remarks, Macrae said the period remains important for Sangamo as the company advances its pipeline while seeking strategic opportunities to strengthen its financial position.
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.
