Japanese pharmaceutical company Astellas Pharma and US-based genomic medicine company Sangamo Therapeutics have signed a license agreement.
Under the terms of the agreement, Sangamo will complete the technology transfer related to its novel, in-house developed neurotropic adeno-associated virus (AAV) capsid, STAC-BBB.
Astellas will be responsible for all research, preclinical and clinical development, regulatory interactions, manufacturing, and marketing of gene therapy products worldwide.
Sangamo will receive $20m in upfront license fees and up to $1.3bn in additional licensed target fees and milestone payments across the five potential neurology disease targets.
The genomic medicine company is also eligible to receive tiered royalties on potential net sales of such products, subject to certain specified reductions.
Sangamo CEO Sandy Macrae said: “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.”
Sangamo develops medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate treatment options.
The agreement provides Astellas with the right to use Sangamo’s STAC-BBB, which showed potential in blood-brain barrier penetration and neuronal transduction.
Also, the deal provides Astellas with a global exclusive license to utilise the STAC-BBB capsid for one target, with an option to add up to four additional targets.
Astellas can add more targets by paying additional licensed target fees to deliver intravenously administered genomic medicines to treat certain neurological diseases.
Astellas chief strategy officer Adam Pearson said: “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.”
