Capsida Biotherapeutics Announces Publication in Nature Nanotechnology Identifying Novel Capsid With Significantly Increased Brain Transduction

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Jul 11, 2023, 07:45 ET

Foundational research by Capsida and Caltech demonstrates ability to engineer AAVs with improved efficacy over AAV9 to transduce brain in non-human primates

Peer-reviewed publication highlights early work leading to Capsida's recent breakthrough data presented at ASGCT

THOUSAND OAKS, Calif., July 11, 2023 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced research published in Nature Nanotechnology describing an adeno-associated virus (AAV) variant that can more effectively transduce the brain in non-human primates (NHPs) than AAV9. The data reinforce the ability of Capsida's platform to engineer AAVs with improved efficacy over AAV9.

The publication entitled, "Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain," was published online on July 10, 2023. It is co-authored by scientists at Capsida and Caltech.

This new paper reports on AAV.CAP-Mac, an engineered variant identified by screening across multiple NHP species. It showed increased transduction over wild type AAV9 delivered systemically with uniform transduction across the NHP brain and predominantly neuronal targeting. These data highlight the potential for the non-invasive administration of novel, engineered AAVs from Capsida's platform to improve upon tissue targeting and the extent of transduction achieved with wild type AAVs.

"The work described in the Nature Nanotechnology paper highlights early groundbreaking research from Capsida and Caltech that has enabled us to make significant progress in delivering on the promise of gene therapy for both rare and more common CNS diseases across all ages," said Peter Anastasiou, Chief Executive Officer at Capsida.

At the recent American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, Capsida shared preclinical data on the company's next-generation engineered AAV capsids showing transduction in up to 68% of neurons across multiple brain regions while reducing liver transduction five-fold compared to wild-type AAVs.

"The roadmap for engineering AAVs outlined in this work showcases the strength of Capsida's platform, which utilizes non-human primate screening to identify novel capsids that cross the blood-brain barrier with non-invasive administration and increased efficacy to reach the brain," said co-author Nicholas Flytzanis, Ph.D., co-founder and Chief Research and Innovation Officer at Capsida. "Detailed characterization across non-human primate species and human cells provides a comprehensive picture of how this approach can open up new avenues for both research and therapeutic applications."

"The data from this early generation capsid provides additional validation of the power of directed evolution and Capsida's engineering strategy," added Nick Goeden, Ph.D., co-founder and Chief Technology Officer at Capsida and study co-author. "We can yield capsids that can be delivered systemically with specifically engineered cell-type tropism. Capsida has further progressed these efforts to identify next-generation engineered AAV capsids with greater transduction including protein production and significantly less exposure to the liver."

About Capsida Biotherapeutics
Capsida Biotherapeutics is a fully integrated next-generation gene therapy platform company. Capsida's approach unlocks the potential to treat both rare and common diseases across all ages. We create customized therapies that selectively target specific organ systems and simultaneously limit exposure to non-targeted organs. The company has wholly owned programs in central nervous system (CNS) and strategic collaborations with AbbVie (CNS and eye care), Lilly (CNS), and CRISPR Therapeutics (CNS), providing independent validation of Capsida's capabilities. Capsida is backed by Versant Ventures and Westlake Village BioPartners. Its platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at www.capsida.com to learn more.

SOURCE Capsida Biotherapeutics