Capsida Biotherapeutics Inc., a US biotechnology startup using an adeno-associated virus (AAV) engineering and cargo development platform to develop tissue-targeted gene therapies for multiple types of diseases, has received USD 140 mln financing from two venture capital firms and pharmaceutical group AbbVie. The company’s goal is to develop the best-in-class, targeted gene therapies for three programs in serious neurodegenerative diseases based on research conducted at Caltech by Romanian-born professor Viviana Gradinaru.

Capsida is developing 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 compared to current gene therapies. Capsida is also working on proprietary cargo that delivers effective gene replacement or enhancement customized for the specific disease of interest.

The platform originated from groundbreaking research in the laboratory of Romanian-born 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.

Capsida Biotherapeutics has received USD 50 million Series A funding from US venture capital firms Versant Ventures and Westlake Village BioPartners. It also announced a multi-year strategic collaboration and option agreement with AbbVie that provides USD 90 million in upfront and equity investment capital in addition to potential future option, development and commercial milestone payments.

With the anchor investment from Versant and Westlake, and the collaboration with AbbVie, Capsida 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.

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(Photo source: Caltech.edu)