Capsida Biotherapeutics Inc., a Thousand Oaks, CA-based biotechnology company using an adeno-associated virus (AAV) engineering and cargo development platform to develop tissue-targeted gene therapies for multiple types of diseases, raised $140m in Series A funding.
Backers included Versant Ventures and Westlake Village BioPartners, which together provided $50m. Capsida also announced a multi-year strategic collaboration and option agreement with AbbVie that provides $90m in up front and equity investment capital in addition to potential future option, development and commercial milestone payments. The collaboration is aimed at developing targeted gene therapies for three programs in serious neurodegenerative diseases.
Led by Robert Cuddihy, M.D., chief executive officer, Capsida provides an AAV engineering platform that generates capsids optimized 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, the company is developing proprietary cargo that delivers effective gene replacement or enhancement customized for the specific disease of interest.
The platform originated from research in the laboratory of Viviana Gradinaru, Ph.D., Professor of Neuroscience and Biological Engineering, Heritage Medical Research Institute Investigator, and Director of the Center for Molecular and Cellular Neuroscience at the Tianqiao and Chrissy Chen Institute for Neuroscience at Caltech. The platform uses machine learning, structural biology, non-human primate models, and human tissue models to screen billions of engineered capsids for the ability to target desired tissue types precisely.
The company’s engineered capsids have demonstrated markedly enhanced tissue tropism for neurons versus astrocytes, glia, and other CNS cell types, thus demonstrating potential to unlock treatments for disorders requiring neuronal transduction that exceeds the performance of first-generation AAV9-based therapies.
Capsida’s newly announced collaboration with AbbVie will use the biotech’s platform to identify and advance clinically translatable capsids paired with an innovative therapeutic approach from AbbVie to create tissue-targeted gene therapies for three CNS disease targets.
Under the terms of the agreement, the company will receive $80m up front in cash and a $10m equity investment. For targets one and two, upon AbbVie exercising its option, Capsida is eligible to receive $530m in option and development milestone payments excluding commercial milestone payments. the company is also eligible to receive up to high single-digit royalty payments on future product sales. Following AbbVie’s exercise of its options for these programs, AbbVie will be solely responsible for further development and commercialization.
For the third disease target, upon AbbVie exercising its option, Capsida will have the right to develop through human proof-of-concept, and AbbVie would lead late-stage development and commercialization. Following human-proof-of concept, the parties would enter into a 50/50 cost:profit share with Capsida having the option to co-promote in the US.
For all three programs, Capsida will lead capsid discovery efforts using its high throughput AAV engineering and screening platform and AbbVie will contribute innovative therapeutic approaches. Capsida will also be responsible for process development and early clinical manufacturing of all programs.
Capsida’s initial internal preclinical programs center on neurodevelopmental and neurodegenerative disorders, areas in which gene therapies have yet to gain significant traction due to the difficulties of targeting the brain. Based on progress to date, Capsida expects to start IND-enabling work on its first development candidates during 2021 and to start clinical trials in 2022.
In addition to continuing preclinical work on its lead assets, the company plans to use proceeds from its $50 million Series A financing to open its manufacturing facility this year and advance its platform into non-CNS disorders.
The company is operating from its site in Thousand Oaks, California.
With the anchor investment from Versant and Westlake, and the collaboration with AbbVie, the company plans to expand its 50-member team to about 100 scientists this year. To capture the expanding opportunities in the field of gene therapy, Capsida will pursue additional strategic opportunities in parallel with financial investors over the coming months.