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RICHMOND, Calif. – Sangamo Therapeutics, Inc. (NASDAQ:SGMO), a genomic medicine company, today presented preclinical data indicating significant advancements in central nervous system (CNS) delivery technology. The company’s novel adeno-associated virus (AAV) capsid, STAC-BBB, has shown promising results in non-human primates (NHPs), achieving robust penetration of the blood-brain barrier (BBB) and widespread transgene expression in the brain following intravenous administration.
The STAC-BBB capsid has demonstrated industry-leading brain tropism, with transgene expression levels that are 700 times higher than those observed with the benchmark capsid AAV9. This capsid variant was engineered using Sangamo’s SIFTER capsid platform and is part of the company’s efforts to develop genomic medicines for a variety of neurological diseases.
In addition to broad brain coverage, the preclinical studies highlighted potent repression of target genes associated with prion disease and tauopathies, suggesting potential for modifying disease progression. The capsid’s biodistribution profile also indicated desired de-targeting from the liver and other peripheral tissues, which is optimal for AAV-based treatments of neurological diseases.
Sangamo’s CEO, Sandy Macrae, expressed optimism about the STAC-BBB capsid’s capabilities, which could enable the company to resume several neurology epigenetic regulation programs that were previously on hold. These programs include those that were part of former collaborations with Biogen (NASDAQ:) and Novartis (SIX:).
The company has outlined plans to pursue Investigational New Drug (IND) submissions and/or Clinical Trial Applications (CTA) for up to three neurology programs by the end of 2025. This includes a CTA for a prion disease program expected in the fourth quarter of 2025 and an IND submission for a tau program potentially as early as the same period.
Sangamo’s management team will further discuss these results in a conference call scheduled for today at 4:30pm Eastern Time. The call will provide an opportunity for the investment community to gain insights into the significance of these findings and their implications for the company’s future.
This news is based on a press release statement and represents a significant step for Sangamo in its mission to address serious neurological conditions with limited or no treatment options. The company continues to explore avenues for advancing its programs, both internally and with potential collaborators, subject to securing adequate funding.
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