Capsida Biotherapeutics Inc. has entered a multi-year strategic collaboration with Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Co., to develop genetic medicines for serious diseases. Prevail will leverage Capsida’s novel adeno-associated virus (AAV) engineering platform to identify and advance clinically translatable capsids paired with Prevail’s cargo to develop best-in-class, IV-administered gene therapies directed to specified targets known to cause serious diseases that affect the central nervous system (CNS).
 
Capsida developed a high throughput platform to biologically screen and identify engineered AAV capsids that target specific tissues, such as the brain, and limit transduction of tissues and cell types that are not relevant to a given disease. This platform has the potential to improve the efficacy and safety of systemically administered AAV gene therapies and deliver on the promise of gene therapy for patients that are living with diseases that are difficult to treat with existing approaches.
 
Capsida will receive $55 million upfront and a commitment to participate in the company’s next financing round, plus the potential to receive up to $685 million in R&D and commercial milestones as well as royalties. In addition, for one of the programs under the collaboration, Capsida will have an option to participate in development and commercialization in the U.S. in exchange for a gross margin share in that program. For all programs, Capsida will lead capsid discovery efforts using its high throughput AAV engineering and screening platform and Prevail will be responsible for preclinical and Investigational New Drug-enabling studies with therapeutic payloads.
 
“We are thrilled to be entering into this strategic collaboration with Prevail. Prevail’s expertise in neuroscience, gene therapy R&D, and access to Lilly’s world-class commercialization capabilities complements Capsida’s fully integrated approach, including our next-generation AAV engineering platform. Prevail and Capsida are committed to developing highly effective and safe gene therapies that have the potential to be transformative for patients living with serious CNS diseases,” said Peter Anastasiou, chief executive officer of Capsida.
 
“Prevail is committed to developing therapeutics for difficult to treat diseases, and we are excited to enter this collaboration with Capsida to advance IV-administered gene therapies for diseases of the central nervous system with significant unmet need. Their expertise and capabilities complement our innovative approaches, and together we aspire to unlock the full potential of gene therapy technologies to help patients who are most in need of treatments,” said Mansuo Shannon, chief scientific officer of Prevail.