About

“We are focused on successfully executing IND-enabling studies for our Parkinson’s disease and genetic epilepsy programs to support initiation of clinical trials in the first half of 2025 for the people who so desperately need them.”

Peter Anastasiou, CEO

Capsida Biotherapeutics is an industry-leading gene therapy company creating a new class of targeted, non-invasive IV administered gene therapies for rare and more common diseases across all ages. We have three wholly owned programs in CNS, including potential best-in-class treatments for genetic epilepsy due to STXBP1 mutations and Parkinson’s disease associated with GBA mutations. Our candidates achieve breakthrough levels of neuronal transduction throughout the brain, up to 70% in certain brain regions, while simultaneously detargeting the liver. We also have validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and expanded our partnership with AbbVie to include ophthalmology disorders.


Science & Innovation

THE CAPSIDA APPROACH

Capsid Engineering, Cargo Optimization, Translational Biology, Process Development and Manufacturing, and Clinical Development – All Under One Roof

Capsida Biotherapeutics is the only 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.

Built through a combination of in-house R&D and industry partnerships, our therapeutic approaches – including gene replacement and gene editing – are designed to bring therapies to patients with both monogenic and polygenic diseases.

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Capsid Engineering

At Capsida, the scale at which we can engineer and screen capsids is unrivaled. Using our automated, high-throughput biological screening method, our scientists assess large, diverse engineered capsid libraries to quickly identify optimal capsids to target tissues and cells in diseased organs.

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Discovery and Preclinical

To quickly advance our mission of providing meaningful therapeutic opportunities for patients with unmet medical need, Capsida optimizes cargo and evolves preclinical disease proof-of-concept in parallel with capsid engineering. Our engineered capsids can be optimized to deliver a broad range of cargos including gene replacement, gene editing, gene silencing and vectorized antibodies, among others.

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Manufacturing Operations

At Capsida, we are singularly focused on rapidly getting our high-quality, life-changing therapies to patients who are suffering from debilitating diseases. We have built capabilities to perform everything in-house, eliminating the need for contract development and manufacturing organizations (CDMOs). We streamline the entire process, from selecting our therapies for manufacturability through scalable process development. Ultimately, this helps us get therapeutics to patients sooner.

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Clinical Development

Capsida’s approach unlocks the potential to treat both rare and common diseases across all ages. First and foremost, we approach patients holistically, working closely with advocacy groups, patients, and caregivers to ensure we are meeting their needs.

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Pipeline

Capsida’s innovation and intellectual property can be broadly applied to all therapeutic areas. We began with a focus on the Central Nervous System (CNS) based on the expertise and passion of our founders and the significant prevalence and unmet need in CNS disorders, and have since expanded into serious eye diseases through our partnership with AbbVie.


Team

Capsida’s founding technology originated in the lab of Caltech’s Viviana Gradinaru, Ph.D., Professor of Neurosciences and Biological Engineering at the Tianqiao and Chrissy Chen Institute of Neuroscience. Founded in 2019, Capsida is financed by Versant Ventures and Westlake Village BioPartners and has entered into a strategic partnership with AbbVie for treating neurodegenerative disease.

Our leaders have decades of experience in the biopharmaceutical industry and expertise in AAV biology and biologics manufacturing.


Partnering

“We welcome the opportunity to collaborate with innovators who share our determination to develop transformative gene therapies for patients.”

Bethany Mancilla, Chief Business Officer

Capsida’s approach unlocks the potential to treat both rare and common diseases across all ages. Our goal is to apply the strength of our targeted gene therapy platform to develop a new generation of transformative therapies.

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Careers

BE PART OF OUR DNA

Capsida is a rapidly growing company based in the heart of the Greater Los Angeles biotech hub. We’re looking for passionate, dedicated colleagues to join us!

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News & Events

CAPSIDA NEWS & EVENTS

FDA Grants Orphan Drug Designation to Capsida Biotherapeutics for Potential Treatment of STXBP1 Developmental and Epileptic Encephalopathy

Wholly owned first-in-class program anticipated to enter clinical trials in first half of 2025 THOUSAND OAKS, Calif., October 30, 2024 – Capsida…

New Data Demonstrate Substantial Therapeutic Potential of Capsida’s IV Gene Therapy for Parkinson’s Disease Associated with GBA Mutations

CAP-003 achieved best-in-class GCase increases in NHPs exceeding levels needed for normalizing activity in patients Brainwide RNA expression greater than…