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

Rhea-AI Summary

Sangamo Therapeutics (Nasdaq: SGMO) has entered a global license agreement with Genentech to develop intravenous genomic medicines for neurodegenerative diseases. The deal focuses on Sangamo's zinc finger repressors targeting the tau gene, important in Alzheimer's and tauopathies, and another undisclosed neurology target. Sangamo is also licensing its proprietary AAV capsid, STAC-BBB, which has shown strong blood-brain barrier penetration.

Key financial terms include:

• $50 million in near-term upfront fees and milestone payments
• Up to $1.9 billion in potential development and commercial milestones
• Tiered royalties on net sales

Sangamo will handle technology transfer and preclinical activities, while Genentech will lead clinical development, regulatory interactions, manufacturing, and global commercialization.

Positive

• Sangamo to receive $50 million in near-term upfront license fees and milestone payments
• Potential to earn up to $1.9 billion in development and commercial milestone payments
• Eligible for tiered royalties on net sales of products developed under the agreement
• Partnership with Genentech, a member of the Roche Group, a major player in the pharmaceutical industry
• Leverages Sangamo's proprietary zinc finger technology and AAV capsid delivery platform
• Expands Sangamo's reach into neurodegenerative disease treatments

Negative

• Sangamo gives up exclusive rights to its zinc finger repressors for tau and another neurology target
• Sangamo loses control over clinical development, regulatory interactions, manufacturing, and commercialization for the licensed products

Insights

Biotech Industry Analyst

This partnership between Sangamo and Genentech is a significant development in the genomic medicine space, particularly for neurodegenerative diseases. The deal's structure, with $50 million in near-term payments and potential for up to $1.9 billion in milestones, reflects the high value placed on Sangamo's technology.

Key points to consider:

• Sangamo's zinc finger repressors targeting the tau gene could be a game-changer for Alzheimer's and other tauopathies
• The STAC-BBB capsid's ability to penetrate the blood-brain barrier addresses a major challenge in CNS drug delivery
• This deal validates Sangamo's technology and could lead to more partnerships, potentially boosting the company's value

While promising, investors should note that neurodegenerative disease treatments have historically faced challenges in clinical trials. The long-term value will depend on the success of these therapies in human studies.

Neuroscience Research Expert

This collaboration targets a critical area of unmet need in neurodegenerative diseases. The focus on tau, a protein implicated in Alzheimer's and other tauopathies, is particularly noteworthy. Key scientific aspects to consider:

• Zinc finger repressors offer precise gene regulation, potentially allowing for fine-tuned control of tau expression
• The STAC-BBB capsid's demonstrated efficacy in non-human primates is promising, but human trials will be crucial
• Targeting multiple neurodegenerative diseases suggests a platform approach, which could yield broader applications

While the science is exciting, it's important to remember that many promising approaches have faltered in neurodegenerative disease trials. The true test will be in clinical efficacy and safety in humans.

Financial Analyst

This deal significantly bolsters Sangamo's financial position and potential. Key financial implications include:

• Immediate boost from $50 million in near-term payments
• Substantial long-term potential with up to $1.9 billion in milestones
• Tiered royalties on net sales provide ongoing revenue potential
• Genentech bearing clinical development and commercialization costs reduces Sangamo's financial risk

For investors, this partnership mitigates some risk associated with the costly and lengthy development process for neurodegenerative therapies. However, it's important to note that milestone payments are contingent on success and the timeline to potential commercialization is likely long. The deal's structure allows Sangamo to benefit from Genentech's expertise and resources while retaining significant upside potential.

• 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

RICHMOND, Calif.--(BUSINESS WIRE)--

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240806084723/en/

Investor Relations & Media Inquiries

Louise Wilkie

ir@sangamo.com

media@sangamo.com

Source: Sangamo Therapeutics, Inc.

FAQ

What is the focus of Sangamo's license agreement with Genentech?

The agreement focuses on developing intravenously administered genomic medicines to treat certain neurodegenerative diseases, specifically targeting the tau gene involved in Alzheimer's disease and other tauopathies, as well as an undisclosed second neurology target.

How much is Sangamo (SGMO) expected to receive from this agreement?

Sangamo is expected to receive $50 million in near-term upfront license fees and milestone payments. Additionally, they are 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.

What technology is Sangamo licensing to Genentech in this agreement?

Sangamo is licensing its proprietary zinc finger repressors directed at the tau gene and an undisclosed second neurology target. They are also licensing their proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has shown potent blood-brain barrier penetration.

What are the responsibilities of Sangamo and Genentech in this partnership?

Sangamo is responsible for completing technology transfer and certain preclinical activities. Genentech will handle all clinical development, regulatory interactions, manufacturing, and global commercialization of the products developed under this agreement.