Capsida has received FDA IND clearance to initiate a clinical trial for our first-in-class IV-administered investigational gene therapy for STXBP1 Developmental and Epileptic Encephalopathy

Meet Reece and the Zink family, who embody incredible strength, resilience, and love. Reece has STXBP1-DEE. Read Reece’s inspirational story

UNLOCKING THE POTENTIAL OF GENE THERAPY FOR ALL


About

Capsida is a fully integrated next-generation genetic medicines company advancing potentially disease modifying and curative treatments for rare and more common diseases

We are focused on bringing our innovative therapies to the people who so desperately need them. Our program for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE) recently received FDA IND clearance and is initiating a clinical trial. Our program for Parkinson's caused by GBA mutations (PD-GBA) is also on track to enter clinical development this quarter.

Peter Anastasiou, CEO

Capsida Biotherapeutics is developing next generation, targeted, IV-administered genetic medicines for rare and more common diseases across all ages. The Investigational New Drug (IND) application for our potential first and best-in-class investigational therapy for STXBP1-DEE has received FDA clearance and we are now initiating a clinical trial. We are advancing a potential best-in-class investigational treatment for PD-GBA that is on schedule for IND submission in 2Q 2025. We are also working on a potential best-in-class investigational therapy for Friedreich’s ataxia (FA).


Pipeline

Our Pipeline is Making the Impossible Possible

Capsida’s intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels in desired tissues and cells, while limiting tropism to non-target organs, such as the liver and dorsal root ganglion (DRG).

Our program for STXBP1-DEE has received IND clearance from FDA and is initiating a clinical trial. This program is the first engineered IV-delivered gene therapy that crosses the blood-brain-barrier and detargets the liver and dorsal root ganglia (DRG) to enter the clinic.

Our investigational gene therapy for PD-GBA is on track for IND submission in the second quarter of 2025. Our third program is in IND-enabling studies for FA.

In our partnered programs with AbbVie, CRISPR Therapeutics, and Prevail Therapeutics, a part of Lilly’s Gene Therapy Division, we have the potential to co-develop and co-commercialize up to three CNS programs. Partnered programs target CNS and ophthalmology disease indications.


Science & Innovation

THE CAPSIDA APPROACH

Capsid Engineering, Cargo Optimization, Translational Biology, Process Development and Good Manufacturing Practices (GMP) Manufacturing, and Clinical Development – All Under One Roof to Develop Better Therapies Faster

Capsida Biotherapeutics is the only fully integrated next-generation genetic medicines company that has created all the capabilities needed to deliver on the promise of genetic medicine.

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Capsid Engineering

At Capsida, the scale at which we can engineer and screen capsids is unrivaled. Using our automated screening method, our scientists assess large, diverse engineered capsid libraries in non-human primates to quickly identify optimal capsids for disease targets and their corresponding human receptors.

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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 cargo modalities.

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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 state-of-the-art cGMP manufacturing capabilities to perform everything in-house. 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.


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 part of Lilly’s Gene Therapy Division, and Kate Therapeutics (acquired by Novartis).

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


Partnering

“Our partnerships with leading global biopharmaceutical companies are key to Capsida achieving its vision of being able to reach more potential patients in need with our enabling genetic medicines.”

Bethany Mancilla, Chief Business Officer

Our goal is to apply the strength of our targeted genetic medicine 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 part of Lilly’s Gene Therapy Division, and Kate Therapeutics (acquired by Novartis).

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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!

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News & Events

CAPSIDA NEWS & EVENTS

Capsida Presents New GLP Toxicology Data Supporting Recent FDA IND Clearance of Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy

Data show CAP-002 has potential to safely correct seizures, developmental disabilities, and motor abnormalities after single IV infusion Oral presentation…

Capsida Receives FDA IND Clearance for Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy

CAP-002 is the first engineered IV-delivered gene therapy that crosses the blood-brain-barrier and detargets the liver and dorsal root ganglia to enter the…