Capsida Biotherapeutics is an industry-leading gene therapy company creating a new class of potentially curative and disease-modifying therapies for rare and more common disorders. Naturally occurring AAV capsids have limited the potential application of gene therapies for the CNS. Typically, AAV9-delivered treatments even when delivered via invasive brain surgery methods transduce a very small percentage of brain cells, increasing risks associated with delivery and restricting their use due to the limited transduction. At Capsida, we are developing intravenous (IV) delivered gene therapies for the CNS that have broad, brain-wide neuronal expression throughout the CNS and superior off target safety profiles, including significant detargeting of the liver and dorsal root ganglion (DRG), resulting in wider therapeutic index and potential for lower dosing compared to wild type serotypes. For diseases of the eye, Capsida is directing its platform to engineer capsids for local delivery.
Capsida is the only gene therapy company that has been able to achieve the combination of capsid engineering scale, CNS tropism, peripheral de-targeting, therapeutic expression, clinical translatability and manufacturability.
Capsida’s AAV gene therapy platform originated from groundbreaking research in the laboratory of Professor Viviana Gradinaru, Ph.D. at Caltech.
Professor Gradinaru’s lab is where Nick Goeden, Ph.D., 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.
Dr. Goeden, Chief Technology Officer, and Dr. Flytzanis, Chief Research and Innovation Officer, built their team by hiring scientists with new, diverse and curious approaches to solving problems. Professor Gradinaru continues her involvement with Capsida as a member of the company’s Board of Directors.
