BAR HARBOR, Maine, USA, July 9, 2026
The Jackson Laboratory (JAX), in collaboration with the Broad Institute and a multidisciplinary network of academic, clinical, industry, and patient advocacy partners, has been selected for an up to $34.5 million award from the Advanced Research Projects Agency for Health (ARPA-H) under its Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) initiative. The funding will support the development of the Pediatric Epilepsies & Rare CNS (PERC) Gene Editing Platform, an innovative program designed to accelerate precision genetic medicines for children affected by severe neurological disorders. Initially focusing on Alternating Hemiplegia of Childhood (AHC) and Dravet syndrome, the project aims to create a scalable gene-editing platform capable of advancing multiple rare central nervous system (CNS) diseases while shortening development timelines for future personalized therapies. The initiative represents a major milestone in gene-editing innovation, combining expertise in base editing, prime editing, in vivo gene delivery, disease modeling, and translational medicine to address some of the most challenging pediatric neurological disorders.
PERC Platform Accelerates Precision Gene Editing for Rare CNS Disorders
The Pediatric Epilepsies & Rare CNS (PERC) platform has been designed to establish a repeatable and scalable development pathway for precision genetic medicines, moving beyond the traditional one-disease-at-a-time model commonly used in rare disease research. Supported through the ARPA-H THRIVE program, the project will pursue three major objectives: advancing gene-editing strategies for AHC and Dravet syndrome, generating the robust preclinical evidence required for first-in-human clinical studies, and building a standardized regulatory framework that can be expanded to additional rare neurogenetic diseases.
The program integrates recent advances in base editing, prime editing, CNS-targeted gene delivery technologies, and precision disease biology, creating a comprehensive platform capable of accelerating therapeutic development for devastating childhood neurological disorders. Researchers believe this standardized approach could significantly reduce development complexity while improving efficiency in delivering personalized treatments for patients with ultra-rare genetic diseases.
Collaborative Research Strengthens Rare Disease Therapeutic Development
Advertisement
The PERC initiative brings together a broad coalition of 12 leading research institutions, hospitals, biotechnology organizations, and patient advocacy groups, including The Jackson Laboratory, Broad Institute, Boston Children’s Hospital, and The RARE Hope Foundation. Within the collaboration, The Jackson Laboratory’s Rare Disease Translational Center (RDTC) will lead preclinical development activities by leveraging its expertise in disease genetics, precision genome editing, animal disease models, translational research, and therapeutic evaluation.
The team has already conducted extensive research in Alternating Hemiplegia of Childhood and Dravet syndrome, providing a strong scientific foundation for evaluating advanced gene-editing approaches capable of correcting disease-causing genetic mutations. By integrating scientific discovery, translational biology, regulatory planning, and patient-centered collaboration, the consortium aims to establish a robust platform that can accelerate the transition of promising gene-editing therapies from laboratory research into clinical development.
ARPA-H Investment Advances the Future of Precision Genetic Medicine
The ARPA-H THRIVE award reflects the U.S. government’s continued investment in accelerating precision genetic medicine for patients living with severe rare neurological diseases. Approximately half of all known rare genetic disorders affect the central nervous system, with many causing profound neurological impairment and high mortality during early childhood. Traditional drug development approaches remain challenging for these ultra-rare conditions because of limited patient populations and complex clinical requirements.
Through the PERC platform, researchers aim to demonstrate that innovative gene-editing technologies, supported by scalable regulatory strategies and standardized development pipelines, can dramatically improve the speed and efficiency of therapeutic development across multiple diseases. If successful, the initiative could establish a new model for precision medicine, expanding access to personalized gene-editing therapies while transforming the future treatment landscape for rare pediatric neurological disorders worldwide.
Source: The Jackson Laboratory press release
