Capsida announces strategic collaboration to develop non-invasive gene therapies for CNS diseases

Capsida joins forces with Eli Lilly subsidiary Prevail in $55 million deal.

Capsida Biotherapeutics Inc has announced a multi-year strategic collaboration with Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, to develop transformative genetic medicines for serious diseases.

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 (CNS).

Capsids are the protein shell of a virus, enclosing its genetic material; Capsida engineers capsids that can be optimised to deliver a broad range of cargos including gene replacement, gene editing, gene silencing and vectorised antibodies, among others. The company’s initial targets are in the CNS because the founders feel there is significant prevalence and unmet need in CNS disorders; aging is the primary risk factor for most neurodegenerative diseases, including Alzheimer’s and Parkinson’s.

Longevity.Technology: Last year, we identified Capsida as one of the new biotechs on which we’d be keeping eye – and we’re glad we did. Although current gene therapy approaches have shown dramatic efficacy in several rare diseases, the medicines are hindered by imprecise targeting, an inability to transduce a number of cell types and tissues effectively and have safety liabilities. As a result, many monogenetic and sporadic neurodegenerative disorders remain unaddressed by this therapeutic modality.

Capsida is addressing these concerns with its AAV engineering platform that generates capsids optimised to target specific tissue types and limits transduction of tissues and cell types that are not relevant to the target disease, allowing for improved efficacy and safety. In addition, Capsida is developing proprietary cargo that delivers effective gene replacement.

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.

“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,” said Peter Anastasiou, chief executive officer of Capsida. “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 [1].”

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 (R&D) and commercial milestones as well as tiered royalties.

Last year, we reported that Capsida launched with $50 million Series A funding from Versant Ventures and Westlake Village BioPartners; a later AbbVie collaboration swelled the coffers to the tune of an additional $90 million for gene therapies for NS disease targets.

Under the newly-announced collaboration agreement, Capsida will also have an option to participate in development and commercialisation in the US of one of the programs 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.

“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,” said Mansuo Shannon, chief scientific officer of Prevail. “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 [1].”