Faster to Patient. Faster to Cure

Capsida Biotherapeutics is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening disorders.

In the past, nearly all gene therapies have been aimed at treating rare disorders in infants – partially because of tremendous unmet need in those patients but mainly because of the limitations of naturally occurring AAV capsids. At Capsida, we’re not limited by the way things have always been done. We’ve developed a high throughput platform to biologically screen and identify proprietary engineered capsids to significantly improve target specificity and penetration in disease tissues and cells, allowing the use of gene therapy in both rare and common diseases across all ages.

Through intravenous (IV) delivery of a single, engineered capsid, Capsida’s technology can target single or multiple organs simultaneously while limiting exposure to non-targeted organs.

WHAT WE OFFER

Capsida’s innovation and intellectual property can be broadly applied to all therapeutic areas. We’re starting in the Central Nervous System (CNS) because of the significant prevalence and unmet need in CNS disorders.

The Capsida platform generates capsids optimized to target specific tissue types and limits transduction of tissues and cell types that are not relevant to the disease, allowing for improved efficacy and safety. Getting to targeted organs, such as crossing the blood-brain barrier to target the brain, while limiting exposure to non-targeted organs (such as the liver), has been a significant challenge in the gene therapy field. However, with our patent-protected technology, we believe we are poised to overcome this challenge and deliver on the promise of gene therapy for patients in need.

Capsida’s proprietary technology is used to engineer capsids that can significantly improve targeted tissue penetration, thereby allowing use in both rare and common diseases across all ages – unlike traditional gene therapies aimed at treating rare disorders, usually in infants.

Certain diseases require delivery of gene therapy to multiple organs for maximum efficacy. Our technology allows the simultaneous targeting of multiple organs while still limiting the penetration of non-targeted organs, especially the liver.

All Capsida pipeline programs are delivered through intravenous (IV) administration. This delivery method allows the greatest potential for efficacy by using the body’s natural blood flow to deliver our gene therapy to the targeted organs.

Capsida has quickly developed into a fully-integrated gene therapy company. We have created internal capabilities and expertise for capsid engineering, cargo optimization, translational biology, clinical development, regulatory, process development, and state-of-the-art manufacturing.

HISTORY

Capsida’s AAV gene therapy platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a Caltech neuroscience professor.

Gradinaru’s lab is where Nick Goeden, then a post-doctoral researcher, met Nicholas Flytzanis, then a Ph.D. student. A few months into working together, the two began to envision a translational focus for this AAV gene therapy platform – an opportunity to purpose it for human therapeutics. A year into those conversations, the platform achieved its first proof-of-concept.

Over lunch – at a restaurant fittingly named Nick’s – Goeden and Flytzanis approached Gradinaru with a concept for a gene therapy company built around this revolutionary AAV engineering platform.

A few months later, Capsida was born.

Goeden, Chief Technology Officer, and Flytzanis, Chief Scientific Officer, continue to build their team by hiring scientists with new, diverse and curious approaches to solving problems. Gradinaru continues her involvement with Capsida as a member of the company’s Board of Directors.

HISTORY

Capsida’s AAV gene therapy platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a Caltech neuroscience professor.

Gradinaru’s lab is where Nick Goeden, then a post-doctoral researcher, met Nicholas Flytzanis, then a Ph.D. student. A few months into working together, the two began to envision a translational focus for this AAV gene therapy platform – an opportunity to purpose it for human therapeutics. A year into those conversations, the platform achieved its first proof-of-concept.

Over lunch – at a restaurant fittingly named Nick’s – Goeden and Flytzanis approached Gradinaru with a concept for a gene therapy company built around this revolutionary AAV engineering platform.

A few months later, Capsida was born.

Goeden, Chief Technology Officer, and Flytzanis, Chief Scientific Officer, continue to build their team by hiring scientists with new, diverse and curious approaches to solving problems. Gradinaru continues her involvement with Capsida as a member of the company’s Board of Directors.

Capsida Building

INVESTORS