Capsida Debuts with $140 Million to Advance Next Generation Gene Therapies

Rare Daily Staff    

Capsida Biotherapeutics emerged from stealth mode with $50 million in series A funding and a strategic collaboration with AbbVie to use to develop tissue-targeted gene therapies for serious neurodegenerative diseases.

Versant Ventures and Westlake Village BioPartners launched Capsida and are funding its series A round.

Capsida uses an adeno-associated virus (AAV) engineering and cargo development platform to develop tissue-targeted gene therapies for multiple types of diseases.

“Our high-throughput AAV engineering platform is designed to identify differentiated capsids and cargos that will successfully deliver gene therapies with superior cell and tissue targeting and safety profiles than current-generation products in both CNS and non-CNS diseases,” said Robert Cuddihy, CEO of Capsida.

Along with the launch, Capsida announced a multi-year strategic collaboration and option agreement with AbbVie that provides $90 million in upfront and equity investment capital in addition to potential future option, development, and commercial milestone payments. The collaboration is aimed at developing best-in-class, targeted gene therapies for three programs in serious neurodegenerative diseases.

Capsida is focused on the effective delivery of gene therapies to specific tissues. Although current gene therapy approaches have shown dramatic efficacy in several rare diseases, the medicines are hindered by imprecise targeting, an inability to transduce certain cell types and tissues effectively, and safety liabilities. Specifically, most current-generation approaches use naturally occurring serotypes of AAV that have limitations in their ability to transduce desired cell types with a therapeutic gene product. Due to lower transduction efficacy, these products may need to be delivered at higher doses and thus may be more likely to induce immunogenic responses and adverse events.

As a result, many monogenetic and sporadic neurodegenerative disorders are still unaddressed by this therapeutic modality.

Capsida said it is addressing these concerns with its 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, Capsida 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, 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 says its 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.

The company’s 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, Capsida will receive $80 million upfront in cash and a $10 million equity investment. For targets one and two, upon AbbVie exercising its option, Capsida is eligible to receive $530 million in option and development milestone payments excluding commercial milestone payments. Capsida 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 a 50/50 cost/profit share with Capsida with 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.

“By combining Capsida’s deep understanding of the structure-function relationship of AAV biology with AbbVie’s innovative cargo therapeutic technologies, we will strive to develop highly effective and transformative gene therapies for patients with devastating CNS disorders,” said Eric Karran, vice president, Neuroscience Discovery at AbbVie.

Besides the AbbVie collaboration, Capsida’s initial internal preclinical programs center on brain-targeting gene therapies for neurodevelopmental and neurodegenerative disorders. 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 state-of-the-art manufacturing facility this year and advance its platform into non-CNS disorders.

Photo: Robert Cuddihy, CEO of Capsida