EpiCure Therapeutics, Inc., a Seattle, Washington-based Allen Institute spinout, has received a USD 1.5 million NIH R44 grant to advance an AAV gene therapy for SLC6A1-related disorder, a rare developmental and epileptic encephalopathy for which no disease-modifying therapy currently exists. The SBIR Phase II award, funded through the National Institute of Neurological Disorders and Stroke (NINDS), supports preclinical work aimed at reaching a clinical development candidate.
SLC6A1-related disorder arises from loss of one functional copy of SLC6A1, the gene encoding GAT1, the brain's primary GABA transporter. Patients are typically diagnosed around age two with seizures, motor impairment, language delay and intellectual disability. Because the transporter is enriched in inhibitory neurons and astrocytes, EpiCure's approach focuses on restoring SLC6A1 expression selectively in those cell types rather than broadly across neurons, an approach the company says has shown preclinical safety and efficacy advantages over pan-neuronal expression. Under the new award, the company will test next-generation AAV capsids and regulatory elements to refine biodistribution and cell-type specificity, with safety and expression studies planned in mouse models and non-human primates.
The project reflects continued NIH interest in using SBIR/STTR mechanisms to bridge academic-origin gene therapy platforms toward clinical translation, particularly for monogenic neurodevelopmental diseases with small patient populations and no approved treatments. According to the grant abstract, no FDA-approved therapy currently induces SLC6A1 expression, and no gene-replacement approach for the disorder has yet entered clinical testing, underscoring the first-in-class positioning of EpiCure's program if it advances.