About
Science & Innovation
THE CAPSIDA APPROACH
Capsid Engineering, Cargo Optimization, Translational Biology, Process Development and Manufacturing, and Clinical Development – All Under One Roof to Develop Better Therapies Faster
Capsida Biotherapeutics is the only fully integrated next-generation gene therapy company that has created all the capabilities and results needed to deliver on the promise of gene therapy. Capsida’s approach unlocks the potential to treat both rare and common diseases across all ages.
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.
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.
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.
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.
Pipeline
Our Pipeline is Making the Impossible Possible
Capsida’s pipeline consists of next generation intravenously (IV) administered gene therapies. Capsida’s gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver and dorsal root ganglion (DRG). Capsida has developed IV-delivered capsids with >70% neuronal expression and ~5000-fold difference in central nervous system (CNS) expression versus liver targeting relative to naturally occurring AAV9, substantially increasing the potential therapeutic index of our gene therapy candidates. Capsida is currently progressing three wholly owned programs in CNS disorders, including STXBP1 developmental and epileptic encephalopathy and Parkinson’s disease associated with GBA mutations expected to enter clinical development in the first half of 2025. Our third program is in IND-enabling studies for Friedreich’s ataxia and is aiming to target CNS, cardiac, and sensory manifestations with a single candidate.
In our partnered programs we have the potential to co-develop and co-commercialize up to three CNS programs. Partnered programs target CNS and ophthalmology disease indications.
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 strategic collaborations with AbbVie, CRISPR Therapeutics, Prevail Therapeutics (a wholly owned subsidiary of Eli Lilly), and Kate Therapeutics.
Our leaders have decades of experience in the biopharmaceutical industry and expertise in AAV biology and biologics manufacturing.
Partnering
“The opportunity to partner with leading global biopharmaceutical companies is key to Capsida achieving its vision of gene therapy for all.”
Bethany Mancilla, Chief Business Officer
Our goal is to apply the strength of our targeted gene therapy platform to develop a new generation of transformative therapies. Since our inception, we have entered into strategic collaborations with AbbVie, CRISPR Therapeutics, Prevail Therapeutics (a wholly owned subsidiary of Eli Lilly), and Kate Therapeutics.
Careers
BE PART OF OUR DNA
Capsida is a vibrant company based in the heart of the Greater Los Angeles biotech hub. We’re looking for passionate, dedicated colleagues to join us!