Capsida and Prevail Collaborate to Develop Non-Invasive Gene Therapies for CNS Diseases

Capsida Biotherapeutics and Lilly subsidiary Prevail Therapeutics entered a multi-year strategic collaboration to develop genetic medicines for serious diseases.

Photo: Peter Anastasiou, CEO of Capsida

As part of the collaboration, 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.

Delivering AAV gene therapy systemically to target the CNS, while limiting exposure to non-target organs (such as the liver), has been a significant challenge in the gene therapy field. Capsida has 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.

“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,” said Peter Anastasiou, CEO of Capsida.

Under the terms of the agreement, Capsida will receive $55 million consisting of an upfront payment and a commitment to participate in the company’s next financing round, plus the potential to receive up to $685 million in research and development and commercial milestones as well as tiered royalties. In addition, for one of the programs under the collaboration, Capsida will have an option to participate in development and commercialization in the United States 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.

“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.

Author: Rare Daily Staff