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.