Capsida Biotherapeutics has unveiled data showing that its AAV-delivered gene therapy for developmental and epileptic encephalopathy (DEE), the most severe form of epilepsy, corrected cognitive and motor deficits and reduced seizures in mice up to one year after treatment.
The California-based biotech presented the results on Dec. 7 at the annual meeting of the American Epilepsy Society in Los Angeles.
Capsida recently met with the FDA for a pre-Investigational New Drug meeting and received an Orphan Drug Designation for the therapy, called CAP-002, the company said in a Dec. 6 release. Capsida plans to bring CAP-002 into the clinic in the first half of 2025.
The therapy restored syntaxin-binding protein 1 (STXBP1) protein levels in 70% of primate neurons, according to the abstract. STXBP1 is one of the genes that can cause DEE when mutated. CAP-002 also restored STXBP1 protein levels and neuronal network activity in human neurons cultured in the lab, Capsida said.
“STXBP1-DEE is a life-altering condition for patients and their families, and there are no approved treatments,” Capsida CEO Peter Anastasiou said in the release. “These new data demonstrate the potential of CAP-002 to safely address all of the manifestations of the disease, and we look forward to progressing this program into the clinic in the first half of 2025.”
DEE often manifests in infancy, with children experiencing frequent seizures that are difficult to control with typical anti-seizure medications. Patients commonly have other conditions alongside DEE, like sleep problems, movement disorders and autism spectrum disorder.