Capsida Biotherapeutics is developing next generation, targeted genetic medicines for rare and more common neurological and ocular diseases across all ages.

Naturally occurring AAV capsids have limited the potential application of gene therapies for the CNS and ophthalmology because of their limited ability to cross biological barriers. CNS gene therapies are typically delivered via invasive brain surgery methods that result in inconsistent expression. At Capsida, we are advancing intravenously (IV) delivered gene therapies for the CNS that have broad, brain-wide neuronal expression.

We are developing an investigational therapy for STXBP1-developmental and epileptic encephalopathy (STXBP1-DEE) and investigational therapy for Parkinson’s disease associated with GBA mutations (PD-GBA). We are also developing an 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.

Traditional gene therapy for ocular diseases has been hampered by procedural burden of subretinal surgery and physiological and physical barriers that prevent efficient transduction of retinal cell types.  For diseases of the eye, Capsida is directing its platform to engineer capsids with improved expression profiles through less invasive intravitreal (IVT) and suprachoroidal (SCS) administration .

WHAT MAKES CAPSIDA DIFFERENT