THOUSAND OAK, California, April 29, 2021 / PRNewswire / – Versant Ventures and Westlake Village BioPartners today announced the emergence of stealth mode from Capsida Biotherapeutics Inc., a biotechnology company using an adeno-associated virus (AAV) engineering and development platform of cargo to develop tissue-targeted gene therapies for several types of diseases. In addition to a $ 50 million Series A commitment of the two companies, Capsida also announced a multi-year strategic collaboration and option agreement with AbbVie which provides $ 90 million in initial investment capital and equity, in addition to any future payments of options, development and business milestones. The collaboration aims to develop the best targeted gene therapies for three programs in severe neurodegenerative diseases.
“Our high-throughput AAV engineering platform is designed to identify the differentiated capsids and cargoes that will successfully deliver gene therapies with cell and tissue targeting and superior safety profiles than current generation products. in CNS and non-CNS diseases, “said Robert cuddihy, MD, CEO of Capsida. “We are very pleased to make our debut today with the support of experienced business builders at Versant and Westlake, as well as a significant pharmaceutical partnership with AbbVie.”
A new generation AAV and cargo platform
Although current gene therapy approaches have shown dramatic effectiveness in several rare diseases, drugs are hampered by imprecise targeting, an inability to effectively transduce a number of cell and tissue types, and safety risks. Specifically, most of the current generation approaches use natural AAV serotypes that have limitations in their ability to transduce desired cell types with a therapeutic gene product. Due to lower transduction efficiency, these products may need to be administered at higher doses and therefore may be more likely to induce immunogenic responses and adverse events.
Consequently, many monogenetic and sporadic neurodegenerative disorders still remain untreated by this therapeutic modality.
Capsida addresses 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 irrelevant to the target disease, enabling efficiency. and improved security. In addition, Capsida is developing a proprietary pipeline that offers efficient and personalized gene replacement or enhancement for the specific disease of interest.
The platform is the result of groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., Professor of Neuroscience and Biological Engineering, Researcher at the Heritage Medical Research Institute and Director of the Center for Molecular and Cellular Neuroscience at the Tianqiao and Chrissy Chen Neuroscience Institute at Caltech. The platform uses machine learning, structural biology, non-human primate models, and human tissue models to screen billions of artificial capsids so that it can precisely target desired tissue types.
Company-designed capsids demonstrated significantly improved tissue tropism for neurons compared to astrocytes, glia and other CNS cell types, demonstrating the potential to unlock treatments for disorders requiring neuronal transduction which exceeds the performance of first generation AAV9 therapies.
“Given our long history in the field of gene therapy, it is gratifying to help launch Capsida, which takes a new approach to developing genetic drugs,” said Clare Ozawa, Ph.D., Managing Director of Versant and member of the Board of Directors of Capsida.
Collaboration with AbbVie
Capsida’s recently announced collaboration with AbbVie will use the biotechnology platform to identify and advance clinically translatable capsids, coupled with AbbVie’s innovative therapeutic approach to create tissue-targeted gene therapies for three disease targets. SNC.
Under the terms of the agreement, Capsida will receive $ 80 million in advance in cash and a $ 10 million A fair investment. For targets one and two, as soon as AbbVie exercises its option, Capsida is eligible to receive $ 530 million in options and development milestone payments excluding trade milestone payments. Capsida is also eligible to receive single-digit royalty payments on future product sales. Following the exercise by AbbVie of its options for these programs, AbbVie will be solely responsible for further development and commercialization.
For the third target disease, once AbbVie exercises its option, Capsida will have the right to develop through human proof of concept, and AbbVie will drive late stage development and commercialization. Following human proof of concept, the parties would enter into a 50/50 cost: benefit sharing with Capsida having the ability to co-promote in the United States.
For all three programs, Capsida will lead capsid discovery efforts using its high-throughput AAV engineering and screening platform and AbbVie will bring innovative therapeutic approaches. Capsida will also be responsible for process development and early clinical manufacturing of all programs.
“We are delighted to enter into this licensing agreement with Capsida. By combining Capsida’s deep understanding of the structure-function relationship of AAV biology with AbbVie’s innovative therapeutic technologies, we will strive to develop highly effective and transformative gene therapies for patients with devastating CNS disorders. ,” mentionned Eric karran, Ph.D., Vice President, Neuroscience Discovery at AbbVie.
Capsida operating plans
Capsida’s first internal preclinical programs focus on neurodevelopmental and neurodegenerative disorders, areas in which gene therapies have not yet gained ground due to difficulties in targeting the brain. Based on the progress made to date, Capsida plans to begin work enabling IND on its first development candidates in 2021 and to begin clinical trials in 2022.
In addition to continuing preclinical work on its main assets, the company plans to use the product of its $ 50 million Series A funding to open its state-of-the-art manufacturing facility this year and advance its platform in non-CNS disorders.
The company operates from its site in Thousand Oaks, California.
“Capsida is another example of the rapidly growing biotechnology cluster in the Greater Los Angeles region, “said Beth seidenberg, MD, Founding Managing Director of Westlake Village BioPartners and Member of the Board of Directors of Capsida. “Westlake Village BioPartners is proud to catalyze this growth. Capsida is led by an exceptional team of executives and we look forward to growing the team to support our internal pipeline and the AbbVie collaboration.”
With the anchor investment from Versant and Westlake, and collaboration with AbbVie, Capsida plans to expand its 50-member team to around 100 scientists this year. To seize the growing opportunities in the field of gene therapy, Capsida will pursue additional strategic opportunities in parallel with financial investors over the coming months.
About Capsida Biotherapeutics
Capsida Biotherapeutics Inc. develops tissue-targeted gene therapies using its high throughput adeno-associated virus (AAV) engineering and proprietary freight development platform. As a fully integrated gene therapy company, Capsida combines its differentiated AAV engineering and screening capabilities with state-of-the-art cargo development and manufacturing to establish a proprietary pipeline of breakthrough gene therapies across a range of therapeutic areas for indications. inaccessible. with current technologies. The company’s leadership is supported by decades of successful experience in manufacturing biologics and deep expertise in AAV biology. Visit us at www.capsida.com to find out more.
About Versant Ventures
Versant Ventures is a leading healthcare venture capital firm committed to helping exceptional entrepreneurs build the next generation of great businesses. The company focuses on biotechnology companies that are discovering and developing new therapies. With $ 4.2 billion under management and offices in the United States, Canada and Europe, Versant has assembled a team with deep investment, operations and R&D expertise that enables a hands-on approach to starting a business. Since the company was founded in 1999, more than 75 Versant companies have successfully completed acquisitions or IPOs. For more information, please visit www.versantventures.com.
About Westlake Village BioPartners
Westlake Village BioPartners is a Los Angeles-based venture capital firm focused on incubating and building life science businesses with entrepreneurs who have the potential to bring transformative therapies to patients. Westlake manages more startup-only venture capital from the greater Los Angeles area than any other company. The Westlake model is based on the founding team’s unique experience in identifying and developing breakthrough therapies and building organizations, based on their extensive R&D experience. For more information, please visit www.westlakebio.com.
SOURCE Capsida Biotherapeutics