Sangamo and Astellas Collaborate to Advance Neurological Gene Therapies Using AAV Capsid Technology

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a leader in genomic medicine, and Astellas Pharma Inc. (TSE: 4503), a global innovator in life sciences, have partnered under a new license agreement. This collaboration centers around Sangamo’s cutting-edge neurotropic AAV capsid, STAC-BBB, designed for potent blood-brain barrier penetration and neuronal transduction.

A Strategic Collaboration for Neurological Disease Treatment

The agreement grants Astellas a worldwide exclusive license to use the STAC-BBB capsid for one target with the potential to add up to four additional targets. Sangamo will receive an upfront payment of $20 million and stands to earn up to $1.3 billion in milestone payments and additional target fees. Royalties on net sales will range from mid-to-high single digits.

Pioneering Potential in Gene Therapy

Sandy Macrae, CEO of Sangamo, emphasized the unique value of STAC-BBB in addressing delivery challenges in the central nervous system. “This agreement underscores the industry interest in our STAC-BBB capsid and our commitment to advancing potential treatments for neurological diseases with significant unmet medical needs.”
Adam Pearson, Chief Strategy Officer of Astellas, highlighted the complexity of targeting the brain with gene therapies, noting that technologies like STAC-BBB are vital for achieving transformational outcomes for patients.

Division of Responsibilities

Sangamo will oversee the transfer of STAC-BBB technology, while Astellas will handle research, development, regulatory interactions, manufacturing, and global commercialization of therapies derived from the collaboration.

About the STAC-BBB Technology

The STAC-BBB capsid is part of Sangamo’s innovative platform to enhance the delivery of genetic medicines to the central nervous system. Its ability to cross the blood-brain barrier and achieve effective neuronal transduction positions it as a game-changer in gene therapy.

Company Highlights

• • Sangamo Therapeutics focuses on leveraging zinc finger epigenetic regulators and advanced capsid platforms to develop treatments for severe neurological disorders.
  • Astellas Pharma is dedicated to providing transformative therapies in high-need areas, with its gene therapy division advancing a robust pipeline targeting rare diseases in the CNS, eye, and neuromuscular systems.

This collaboration marks a significant milestone in gene therapy, harnessing the potential of advanced AAV capsid technology to address unmet needs in neurological disease treatment.

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