- Agreement grants Astellas rights to employ Sangamo’s novel proprietary capsid, STAC-BBB, for up to five potential neurological disease targets

- Sangamo to receive a $20 million upfront license fee and is eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across all five potential disease targets, as well as tiered royalties on potential net sales

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”), today announced they have entered into a license agreement allowing Astellas to leverage Sangamo’s novel proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has demonstrated potent blood-brain barrier penetration and neuronal transduction in nonhuman primates. The agreement grants Astellas a worldwide exclusive license to utilize the STAC-BBB capsid for one target, with the right to add up to four additional targets after paying additional licensed target fees to deliver their intravenously administered genomic medicines to treat certain neurological diseases.

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Sandy Macrae, Chief Executive Officer, Sangamo

“We strongly believe in the potential of STAC-BBB, our industry-leading, intravenously delivered AAV capsid, to overcome the challenges associated with delivering therapies to the central nervous system. This agreement underscores the continued industry interest in our STAC-BBB capsid and reinforces our ongoing commitment to partnering with collaborators who understand its unique potential. We are delighted to license STAC-BBB to Astellas to advance potential treatments for neurological diseases with significant unmet medical needs.”

Adam Pearson, Chief Strategy Officer, Astellas

“Delivering treatments to the brain and central nervous system remains a highly complex challenge in the field of gene therapy. We believe that technologies such as Sangamo’s STAC-BBB capsid could prove critical in helping us deliver effective transformational treatments to patients suffering from serious genetic neurological conditions. We continue to build a world-class gene therapy pipeline and end-to-end discovery, development, manufacturing, and commercial capabilities. This agreement is another example of our commitment to delivering meaningful therapies for patients with genetic diseases.”

Under the terms of the agreement, Sangamo is responsible for completing a technology transfer related to the STAC-BBB capsid. Astellas is responsible for all research, preclinical and clinical development, regulatory interactions, manufacturing, and global commercialization of the resulting gene therapy products. Sangamo will receive a $20 million upfront license fee from Astellas and is eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across the five potential neurology disease targets, as well as tiered mid-to-high single digit royalties on potential net sales of such products, subject to certain specified reductions.

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.

About Astellas

Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. We provide transformative therapies in disease areas that include oncology, ophthalmology, urology, immunology and women's health. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need. Learn more at www.astellas.com.

About Astellas Gene Therapies

Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Our gene therapy drug discovery engine is built around innovative science, a validated AAV platform, and industry leading internal manufacturing capability with a particular focus on rare diseases of the eye, CNS and neuromuscular system. Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. Astellas Gene Therapies is based in South San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., Sanford, N.C. and Tsukuba, Japan.

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 Astellas to develop genomic medicines to treat neurodegenerative diseases by leveraging Sangamo’s capsid delivery capabilities, the potential for intravenously administered 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, the potential for Astellas to expand its license to include additional targets and to perform research, preclinical and clinical development, manufacturing and global commercialization of licensed gene therapy products for each licensed target, expectations concerning Sangamo’s receipt of an upfront license fee, the potential for Sangamo to receive additional licensed target fees, manufacturing, development and commercial milestone payments, and royalties, 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 Astellas to breach or terminate its agreement with Sangamo; and the potential for Sangamo to fail to realize its expected benefits from the Astellas agreement; 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 Astellas 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 Astellas’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 September 30, 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.

Astellas Cautionary Notes

In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

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