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Highlights
- Sangamo and Astellas forge a groundbreaking partnership to treat neurological diseases.
- Astellas gains exclusive worldwide rights to use STAC-BBB capsid for gene therapies.
- Sangamo to receive $20 million upfront, with potential for $1.3 billion in milestone payments.
Sangamo Therapeutics, Inc. (Nasdaq:SGMO), a leader in genomic medicine, has entered into a major licensing agreement with Astellas Pharma Inc., a prominent global pharmaceutical company. This partnership grants Astellas the exclusive worldwide rights to use Sangamo's proprietary STAC-BBB adeno-associated virus (AAV) capsid for gene therapies targeting neurological diseases.
The Power of STAC-BBB in Neuroscience
STAC-BBB, a novel and highly promising AAV capsid, is designed to address one of the most significant challenges in gene therapy: overcoming the blood-brain barrier (BBB). The BBB is a selective membrane that restricts the delivery of therapeutic agents to the brain, making it incredibly difficult to treat neurological diseases effectively. Sangamo’s STAC-BBB capsid has shown impressive results in nonhuman primates, demonstrating potent BBB penetration and successful neuronal transduction, which are critical for effective gene delivery to the central nervous system (CNS).
Sangamo’s Chief Executive Officer, Sandy Macrae, expressed confidence in the technology, stating that the STAC-BBB capsid is a groundbreaking solution to the hurdles in CNS gene therapy. He emphasized the potential of the capsid in delivering intravenous gene therapies directly to the brain, a breakthrough that could revolutionize the treatment of neurological diseases with high unmet medical needs.
Astellas’ Commitment to Gene Therapy Innovation
Astellas, led by President and CEO Naoki Okamura, is making significant strides in developing transformative gene therapies for genetic diseases. The company recognizes the importance of overcoming the blood-brain barrier to effectively treat conditions such as genetic neurological disorders. Adam Pearson, Chief Strategy Officer at Astellas, noted that technologies like STAC-BBB could play a critical role in advancing the company's pipeline of gene therapies, particularly for diseases that have been difficult to address with traditional approaches.
The partnership agreement grants Astellas the right to use the STAC-BBB capsid for one target, with the option to add up to four additional targets after paying additional licensed target fees. This collaboration could pave the way for the development of several potential gene therapies aimed at treating various neurological conditions, such as inherited genetic disorders and other serious brain diseases. Astellas will assume responsibility for all aspects of the research, preclinical and clinical development, regulatory affairs, manufacturing, and commercialization of any gene therapy products developed under this agreement.
Financial Terms and Future Prospects
Under the terms of the deal, Sangamo will receive a $20 million upfront payment from Astellas as part of the licensing arrangement. In addition, Sangamo is eligible for up to $1.3 billion in potential milestone payments and licensed target fees, depending on the number of targets Astellas chooses to develop. Additionally, Sangamo will receive tiered royalties on potential future sales of any gene therapy products derived from the STAC-BBB technology.
