The Jackson Laboratory (JAX), in collaboration with the Broad Institute and partners across 12 institutions, has been selected for an up to USD 34.5 million ARPA-H THRIVE contract to develop a scalable gene-editing platform for severe pediatric neurological diseases — an area where effective treatments remain largely absent. The award, issued through ARPA-H's Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) initiative, funds the Pediatric Epilepsies & Rare CNS (PERC) Gene Editing Platform, with the Broad Institute serving as lead.
The platform will initially target two severe developmental and epileptic encephalopathies: alternating hemiplegia of childhood (AHC) and Dravet syndrome. Both conditions cause profound neurological impairment and carry high early mortality. Rather than pursuing each disease as an isolated program, PERC is designed as a repeatable infrastructure — integrating base editing, prime editing, and in vivo CNS delivery — intended to generate the preclinical evidence required for first-in-human studies while simultaneously establishing a regulatory pathway scalable to additional rare neurogenetic diseases.
The consortium spans JAX's Rare Disease Translational Center, led by Cat Lutz, PhD, MBA; Winston Yan, MD, PhD, of the Broad Institute, who serves as lead investigator; Boston Children's Hospital; the RARE Hope Foundation; and academic, clinical, and industry partners. JAX brings established preclinical expertise in AHC and Dravet syndrome, including published work using precision genome editing in disease models.
The award reflects a broader federal push to address the structural inefficiency of rare disease drug development, where small patient populations and high per-program costs have historically limited commercial and academic investment. ARPA-H's THRIVE initiative is explicitly oriented toward platform approaches that can compress timelines and share regulatory infrastructure across multiple indications — a model that, if validated, could alter how the rare CNS disease field is resourced and organized.