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Sangamo Therapeutics and Astellas Collaborate on Innovative Gene Therapy for Neurological Diseases

NextEdge Admin

21 Dec 2024

2 min 13 sec

Astellas,Gene Therapy,Neurological Diseases

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, and Astellas Pharma Inc. (TSE: 4503) have entered a licensing agreement to leverage Sangamo’s proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB. This advanced capsid has shown strong blood-brain barrier penetration and neuronal transduction in nonhuman primates. Under the agreement, Astellas gains an exclusive global license to use STAC-BBB for one target, with options for up to four additional targets for treating neurological diseases via intravenous gene therapy.

Sandy Macrae, CEO of Sangamo, highlighted STAC-BBB’s potential to address central nervous system delivery challenges, emphasizing the agreement as a testament to its capabilities. Adam Pearson, Astellas’ Chief Strategy Officer, noted the importance of such technologies in tackling the complexities of gene therapy for neurological conditions and reaffirmed Astellas’ commitment to building a world-class gene therapy pipeline.

Sangamo will receive a $20 million upfront fee and is eligible for up to $1.3 billion in milestone payments across five potential targets, alongside tiered royalties on net sales. Sangamo will manage technology transfer, while Astellas assumes responsibility for research, development, regulatory interactions, manufacturing, and commercialization.

Sangamo specializes in genomic medicine, focusing on neurological disorders with unmet needs, while Astellas is a global leader in transformative therapies, including gene therapy for rare CNS, ocular, and neuromuscular diseases. This collaboration represents a significant step forward in addressing serious genetic neurological conditions.