Federal Grant Award Summary

Epicure Therapeutics, Inc. received a $1.5M Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853), awarded July 16, 2026, with completion targeted for June 30, 2028. The company is developing a cell class-specific adeno-associated virus (AAV) gene therapy targeting SLC6A1-related neurodevelopmental disorder, a severe developmental epileptic encephalopathy characterized by seizures, motor impairment, and intellectual disability in patients lacking functional copies of the SLC6A1 gene encoding the primary GABA transporter in the brain.

Under this Phase II Small Business Innovation Research (SBIR) award, Epicure Therapeutics will advance its circuit-selective gene replacement strategy by identifying minimal biodistribution parameters for targeting inhibitory neurons, assessing co-targeting approaches for both inhibitory neurons and astrocytes, and evaluating next-generation AAV capsids to optimize cell type specificity. This research builds on established proof-of-concept that restricting SLC6A1 expression to inhibitory neurons or astrocytes—rather than all neurons—represents a viable and safer therapeutic approach, positioning the company to develop a first-in-class disease-modifying treatment for this monogenic neurological disorder.