GENE THERAPY FOR ALL
Capsida Biotherapeutics is an industry-leading genetic medicine 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 advancing 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.
The Investigational New Drug (IND) application for our potential first and best-in-class treatment for STXBP1-DEE has received FDA clearance and we are now 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.
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).
WHAT MAKES CAPSIDA DIFFERENT
Capsida is the only genetic medicine 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
-
- >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 Good Manufacturing Practices (GMP) manufacturing
- Productivity surpassing AAV9
- Quality specifications at or above FDA requirements
LEADERSHIP TEAM
Peter Anastasiou
Chief Executive Officer
View Biography
Nicholas Flytzanis, Ph.D.
Founder, Chief Research and Innovation Officer
View Biography
Nick Goeden, Ph.D.
Founder, Chief Technology Officer
View Biography
Julie Hakim
Chief Financial Officer
View Biography
Bethany Mancilla
Chief Business Officer
View Biography
Rob Murphy
Chief Manufacturing and Quality Officer
View Biography
Swati Tole, M.D.
Chief Medical Officer
View Biography
BOARD OF DIRECTORS
Peter Anastasiou
Chief Executive Officer
View Biography
Rita Balice-Gordon, Ph.D.
View Biography
Viviana Gradinaru, Ph.D.
Founder
View Biography
Clare Ozawa, Ph.D.
View Biography
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.
Let's Connect
General Information
For general inquiries, please use the form below.
Patients and Families
If you are a patient or family member who would like to get in touch with us, please use the form below.
Healthcare Provider
If you are a healthcare provider who would like to get in touch with us, please send us a message using the form below.
Media
Contact Kathy Vincent at
Greig Communications
kathy@greigcommunications.com