Astellas is continuing to strengthen its neurological disease gene therapy capabilities, handing Sangamo Therapeutics $20 million upfront to help bypass the blood-brain barrier (BBB).
The agreement grants the Japanese pharma the worldwide license to initially use Sangamo's proprietary neurotropic adeno-associated virus (AAV) capsid, dubbed STAC-BBB, for one target. The idea is that the capsid—which has shown potential to penetrate the notoriously tricky BBB in preclinical studies—could help Astellas better deliver its own genomic medicines for neurological diseases.
Astellas has the option to expand the collaboration to cover another four neurological targets, subject to additional fees. If Astellas fully takes up this option, it could be in line to pay out up to $1.3 billion in milestone payments to Sangamo on top of tiered mid- to high-single-digit royalties on any marketed products.
Tokyo-based Astellas formally launched its Astellas Gene Therapies unit in 2021. The past couple of years have seen the company license gene therapies from the likes of Kate Therapeutics and 4D Molecular Therapeutics while also taking $170 million hit on an unsuccessful Duchenne muscular dystrophy candidate from Audentes Therapeutics.
The company paid the U.K.’s AviadoBio $50 million in upfront cash and equity investment in October for an AAV-based gene therapy in a phase 1/2 trial for dementia. At the time, Astellas’ chief strategy officer Adam Pearson put the deal in the context of the company’s attempts to expand its gene therapy pipeline “to help a broader range of people living with debilitating, neurodegenerative diseases.”
In this morning’s release, Pearson explained that “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,” Pearson added.
The deal is another win for Sangamo this year after a tough 2023 that saw the gene-based therapy biotech lose partnerships with both Biogen and Novartis as well as lay off staff. This year has seen the company’s fortunes turn around, as Sangamo has secured a pact with Roche’s Genentech unit and an accelerated approval pathway for its Fabry disease candidate in the last five months.
“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,” Sangamo CEO Sandy Macrae, Ph.D., said in this morning’s release. “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.”