Capsida Biotherapeutics is a clinical-stage, fully integrated, 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 intravenously (IV) delivered gene therapies for the CNS that have broad, brain-wide neuronal expression. Studies in relevant preclinical models have shown substantial detargeting of peripheral organs such as liver and dorsal root ganglia (DRG) and potential to lower dose compared to other IV gene therapy approaches.

We are developing a potential first-in-class investigational therapy for STXBP1-developmental and epileptic encephalopathy (STXBP1-DEE) and potential best-in-class investigational therapy for Parkinson’s disease associated with GBA mutations (PD-GBA). We are also developing a potential best-in-class investigational therapy for Friedreich’s ataxia (FA), which is currently in IND-enabling studies and aiming to target CNS, cardiac, and sensory manifestations with a single IV infusion. For diseases of the eye, Capsida is directing its platform to engineer capsids for local delivery.

WHAT MAKES CAPSIDA DIFFERENT

Capsida is a clinical-stage genetic medicine company that has been able to achieve a pioneering combination of capsid engineering scale, manufacturability, identification of human receptors to support clinical translatability and, in relevant preclinical models, CNS tropism, peripheral de-targeting, and therapeutic expression.