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 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.

Our investigational therapies for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE) and Parkinson’s disease caused by GBA mutations (PD-GBA) have received IND clearance from FDA and we are initiating clinical trials. These are the first engineered, IV-delivered gene therapies that cross the blood-brain-barrier and detarget the liver and dorsal root ganglia (DRG) to enter clinical development.

Our third program is in IND-enabling studies for Friedreich’s ataxia (FA) and is aiming to target CNS, cardiac, and sensory manifestations with a single IV infusion.

WHAT MAKES CAPSIDA DIFFERENT

Capsida is the only clinical-stage 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.