Sangamo and Astellas Announce License for Neurological Genomic Medicines

Sangamo Therapeutics, a leading genomic medicine company, and Astellas Pharma, a prominent player in the life sciences industry, have announced a significant licensing agreement. This collaboration grants Astellas the exclusive global rights to utilize Sangamo's advanced neurotropic adeno-associated virus (AAV) capsid, known as STAC-BBB. This capsid is crucial due to its demonstrated capability to effectively cross the blood-brain barrier and achieve neuronal transduction in nonhuman primates, marking a promising advancement in the treatment of neurological diseases.

The agreement allows Astellas to target a specific neurological condition initially, with the potential to expand to four more targets by paying additional fees. This strategic move aims to harness the power of the STAC-BBB capsid for developing genomic medicines that can be administered intravenously, addressing challenging neurological disorders.

Sangamo will receive an upfront payment of $20 million from Astellas and has the opportunity to gain up to $1.3 billion in further fees and milestone payments, contingent on the realization of five potential disease targets. Additionally, Sangamo will benefit from tiered royalties on any net sales resulting from these treatments.

Sandy Macrae, CEO of Sangamo, expressed optimism about the STAC-BBB capsid's potential to tackle the complexities associated with delivering therapies to the central nervous system. The collaboration with Astellas signifies a growing interest in the innovative capabilities of the STAC-BBB capsid. This partnership is poised to advance treatments for neurological diseases that currently lack sufficient medical solutions.

Astellas is committed to expanding its gene therapy pipeline and enhancing its capabilities in research, development, manufacturing, and commercialization. Adam Pearson, Chief Strategy Officer at Astellas, highlighted the significant challenge of delivering therapies to the brain and central nervous system. He emphasized the importance of technologies like the STAC-BBB capsid in overcoming these hurdles and offering transformative treatment options for patients with severe genetic neurological conditions.

Under the terms of the agreement, Sangamo will carry out a technology transfer related to the STAC-BBB capsid to Astellas. Astellas will then be responsible for conducting research, preclinical and clinical development, regulatory interactions, manufacturing, and global commercialization of the gene therapy products derived from this collaboration.

Sangamo Therapeutics is dedicated to translating innovative scientific advances into medical treatments that significantly impact the lives of patients and families affected by serious neurological conditions. The company's expertise in zinc finger epigenetic regulators positions it to potentially address debilitating neurological disorders. Sangamo's work extends beyond immediate intrathecal delivery methods, aiming to develop capsids that enhance delivery to the central nervous system.

Astellas, a global leader in the life sciences sector, is focused on transforming scientific innovations into valuable treatments for patients. Their research and development efforts prioritize areas with significant unmet medical needs, including oncology, ophthalmology, urology, immunology, and women's health. Astellas Gene Therapies, a center of excellence within the company, is dedicated to developing genetic medicines for rare diseases affecting the eye, central nervous system, and neuromuscular system.

Overall, the partnership between Sangamo Therapeutics and Astellas Pharma represents a crucial step forward in the quest to develop effective treatments for neurological diseases, leveraging cutting-edge genomic technologies and extensive expertise in gene therapy. By combining their strengths, both companies aim to bring meaningful therapies to patients, addressing serious genetic conditions with significant unmet needs.