Federal Project Grant Award Summary

The University of North Carolina at Chapel Hill received a Project Grant of $439,588 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 June 22, 2026, with a completion date of May 31, 2028. The grant supports the development of gene therapy approaches to treat cerebellar CACNA1A disorders, a group of neurodevelopmental and neurological conditions caused by pathogenic mutations in the Cav2.1 calcium channel gene. CACNA1A disorders manifest as severe movement disorders, progressive cerebellar atrophy, and seizures, with no existing curative treatments targeting the underlying genetic cause.

The funded research will develop and advance helper-dependent adenoviral vector (HDAD) technology as a therapeutic delivery platform for CACNA1A gene therapy. HDAD vectors offer a critical advantage over standard adeno-associated viral (AAV) vectors by providing significantly larger cargo capacity (~36 kilobases), enabling delivery of the full 7.5-kilobase CACNA1A protein-coding sequence that exceeds AAV packaging limits. Building on established preclinical safety and efficacy data in animal models and Phase I human trials, this project will explore HDAD's application to cerebellar CACNA1A disorders, establishing a foundation for translational advancement toward clinical treatment options for affected patients.