July 20, 2026 —
EpiCure Therapeutics announced that it has received a $1.5 million NIH R44 grant to advance an AAV gene therapy for SLC6A1-related disorder, a rare developmental and epileptic encephalopathy with no approved disease-modifying treatment.
The Seattle-based company, an Allen Institute spinout, received the SBIR Phase II award through the National Institute of Neurological Disorders and Stroke, or NINDS. The funding will support preclinical studies aimed at advancing the program toward selection of a clinical development candidate.
SLC6A1-related disorder is caused by loss of one functional copy of SLC6A1, the gene encoding GAT1, the brain’s primary GABA transporter. The condition is typically diagnosed around age two and is associated with seizures, motor impairment, language delay, and intellectual disability.
EpiCure’s approach focuses on restoring SLC6A1 expression selectively in inhibitory neurons and astrocytes, where GAT1 is enriched. This cell-type-restricted strategy differs from broader pan-neuronal expression approaches and is intended to improve both efficacy and safety by targeting the cell populations most relevant to disease biology.
Under the NIH award, EpiCure will evaluate next-generation AAV capsids and regulatory elements to refine biodistribution and cell-type specificity. Planned studies include safety and expression assessments in mouse models and non-human primates.
The program builds on prior work by Bryan B. Gore and colleagues, who established proof-of-concept for circuit-selective SLC6A1 gene replacement. The new funding supports continued development of that approach toward translational readiness.
According to the grant abstract, no FDA-approved therapy currently induces SLC6A1 expression, and no gene-replacement therapy for SLC6A1-related disorder has entered clinical testing. If advanced successfully, EpiCure’s program could represent a first-in-class therapeutic approach for this genetic epilepsy.
The award also reflects continued NIH interest in using SBIR and STTR funding mechanisms to bridge academic-origin gene therapy platforms toward clinical development, particularly for monogenic neurodevelopmental diseases with small patient populations and limited treatment options.
More broadly, the program highlights a key technical direction in CNS gene therapy: achieving precise, cell-type-restricted AAV delivery. For genetic epilepsies, balancing sufficient therapeutic expression with safety in defined neuronal circuits remains a central challenge ahead of first-in-human studies.
