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Sangamo Therapeutics Announces Global Epigenetic Regulation and Capsid Delivery License Agreement with Genentech to Develop Novel Genomic Medicines for Neurodegenerative Diseases

  • Agreement leverages Sangamo’s proprietary capsid delivery platform and epigenetic regulation capabilities to address certain neurodegenerative diseases
  • Sangamo expected to receive $50 million in near-term upfront license fees and milestone payments and is eligible to earn up to $1.9 billion in development and commercial milestone payments across multiple medicines, as well as tiered royalties on net sales

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced it has entered into a license agreement with Genentech, a member of the Roche Group, to develop intravenously administered genomic medicines to treat certain neurodegenerative diseases. Sangamo has granted Genentech an exclusive license to Sangamo’s proprietary zinc finger repressors that are directed to the tau gene, which is critically involved in Alzheimer’s disease and other tauopathies, as well as an undisclosed second neurology target. Sangamo has also agreed to exclusively license to Genentech, for tau and the second neurology target, Sangamo’s proprietary, neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has demonstrated potent blood-brain barrier penetration and brain transduction in nonhuman primates.

“Sangamo has been pioneering the field of genomic medicine for years to address devastating neurodegenerative diseases with limited current treatment options,” said Sandy Macrae, Chief Executive Officer of Sangamo. “We strongly believe in the power of our zinc finger technology to regulate the expression of key genes involved in disease. The recent discovery of our industry-leading intravenously delivered AAV capsid, STAC-BBB, has the potential to address longstanding challenges in delivering therapeutics to the central nervous system. We are excited to share this powerful combination with Genentech to advance potential treatment options for devastating neurodegenerative disorders, and we are hopeful this could be the first of multiple capsid collaborations to come with other partners.”

“We are uniquely positioned with our collective experience, expertise and resources in neurological research to explore transformative approaches, including gene therapy, that treat neurodegenerative diseases,” said Boris L. Zaïtra, Head of Roche Corporate Business Development. “Through groundbreaking research and partnerships with companies such as Sangamo, we are committed to pursuing important breakthroughs in both early diagnosis and treatment. Our relentless pursuit of scientific innovation is taking us into areas of enormous unmet medical need and progress in treating diseases of the brain and nervous system.”

Under the terms of the agreement, Sangamo is responsible for completing a technology transfer and certain preclinical activities, and Genentech is responsible for all clinical development, regulatory interactions, manufacturing and global commercialization. Genentech is expected to pay Sangamo $50 million in near-term upfront license fees and milestone payments. Sangamo is eligible to earn up to $1.9 billion in development and commercial milestones spread across multiple potential products under the agreement and tiered royalties on net sales of such products, subject to certain specified reductions.

Sangamo continues to engage in business development discussions with additional potential collaboration partners about the Sangamo STAC-BBB capsid delivery platform, its epigenetic regulation capabilities, and other assets, including Fabry disease.

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 Twitter/X.

Sangamo Forward Looking Statements

This press release contains forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the potential for Genentech to develop intravenously administered genomic medicines to treat neurodegenerative diseases by leveraging Sangamo’s epigenetic regulation and capsid delivery capabilities, the potential for STAC-BBB to address challenges in delivering therapeutics to the central nervous system, the potential of Sangamo’s capsids to deliver therapies treating neurological diseases, expectations concerning Sangamo’s completion of the requisite technology transfer and capsid optimization activities, the potential for Genentech to complete clinical development, regulatory interactions, manufacturing and global commercialization of any resulting products, expectations concerning Sangamo’s receipt of an upfront license fee and near-term milestone payments and the timing thereof, the potential for Sangamo to receive development and commercial milestone payments and royalties, plans and expectations concerning additional collaborations with respect to Sangamo’s STAC-BBB capsid delivery platform, epigenetic regulation capabilities, and other assets, 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, the research and development process, including the results of preclinical studies and clinical trials; the regulatory approval process for product candidates; the potential for technological developments that obviate technologies used by Sangamo and its partners; the potential for Genentech to breach or terminate its agreement with Sangamo; and the potential for Sangamo to fail to realize its expected benefits from the Genentech agreement, including but not limited to further validating the importance of the zinc finger platform to support the development of therapeutics for neurodegenerative diseases; Sangamo’s inability to secure additional collaboration partners; and Sangamo’s need for substantial additional funding to operate as a going concern. There can be no assurance that Sangamo will earn any milestone or royalty payments under the Genentech agreement or obtain regulatory approvals for product candidates arising from this agreement. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's and Genentech’s operations and businesses. These risks and uncertainties are described more fully in Sangamo’s Securities and Exchange Commission, or SEC, filings and reports, including in its Annual Report on Form 10-K for the year ended December 31, 2023, as supplemented by its Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, 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.

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