USA —Sangamo Therapeutics, a genomic medicine biotech, has secured a partnership with Prevail Therapeutics, a subsidiary of Eli Lilly, focused on its preclinical neurology adeno-associated virus (AAV) capsids.
This collaboration comes after Sangamo experienced setbacks when both Biogen and Novartis terminated their partnerships earlier this year.
Under the agreement, Prevail will evaluate specific AAV capsids developed by Sangamo, with the option to exclusively license these capsids for multiple undisclosed neurological targets.
Sangamo’s AAV capsid engineering platform has shown promising results in delivering gene therapy to the central nervous system in preclinical animal models, according to the company.
The company aims to utilize these capsids to enhance therapeutic delivery in previously challenging areas.
As part of the deal, Prevail will make an upfront payment to Sangamo for the evaluation of the capsids. If Prevail decides to move forward with certain capsids, it will take the lead on further development, manufacturing, and commercialization.
Sangamo stands to receive developmental milestones of up to US$415 million and commercial payments of up to US$775 million, along with tiered royalties, if Prevail exercises its option for all targets and obtains regulatory approvals for each target in the U.S. and Europe.
This partnership is a positive turn of events for Sangamo, which faced setbacks when both Biogen and Novartis discontinued their collaborations earlier this year.
Novartis withdrew from their autism spectrum disorder and intellectual disability collaboration, while Biogen terminated their partnership involving tauopathies, Parkinson’s disease, and myotonic dystrophy.
These terminations resulted in the loss of potential milestone payments amounting to US$2.37 billion.
Following the dissolution of these collaborations, Sangamo underwent a restructuring, which involved a layoff of approximately 120 employees, representing 27% of its U.S. workforce.
The company announced a refocus on three key areas: its neurology epigenetic regulation portfolio, a phase 3-ready asset for Fabry disease, and a phase 1 CAR-Treg therapy.
As part of its new strategic direction, Sangamo halted all preclinical work except for its internally owned neurology pipeline.
The company continued to identify engineered AAV capsids aimed at improving therapeutic delivery to the central nervous system, attracting the interest of Prevail.
The partnership between Sangamo and Prevail follows a previous agreement signed in May between Prevail and Scribe Therapeutics to leverage CRISPR X-Editing (XE) technologies in the development of genetic medicines for serious neurological and neuromuscular diseases.
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