GENE THERAPY FOR ALL
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.
WHAT MAKES CAPSIDA DIFFERENT
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.
- Fully industrialized and roboticized platform
- Screening capabilities across cell types in NHPs and human cells
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- >99% specific to neurons
- >70% neurons transduced
- Broad IP covering capsids and capsid/cargo
- >16x liver & >50x DRG detargeted
- Superior off-target safety profile
- Broad IP protecting detargeting
- Expression in NHPs with potential for full disease correction
- Industry leading expression levels across pipeline programs
- Identified/patented novel human receptor with complete homology in NHPs and humans
- Validated expression in NHPs including primates >20 years old
- In-house process development and GMP manufacturing
- Productivity surpassing AAV9
- Quality specifications at or above FDA standards
HISTORY
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.
HISTORY
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.
INVESTORS
