Pediatric Epilepsies & Rare CNS Gene Editing Platform (PERC) aims to accelerate the development of precision genetic medicines for severe childhood neurologic diseases with limited or no effective treatment options
BAR HARBOR, Maine, July 9, 2026 /PRNewswire/ — The Jackson Laboratory (JAX) today announced it has been selected, with the Broad Institute and other partners, for an up to $34.5 million contract from the Advanced Research Projects Agency for Health (ARPA-H), within the U.S. Department of Health and Human Services (HHS), through its Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines (THRIVE) initiative. The funding will support the Pediatric Epilepsies & Rare CNS (PERC) Gene Editing Platform, a bold, multi-institutional project led by the Broad Institute.
PERC directly addresses the goals of ARPA-H’s THRIVE initiative by advancing a platform-based approach to precision genetic medicine for rare diseases. The project will initially focus on two severe pediatric developmental and epileptic encephalopathies: alternating hemiplegia of childhood (AHC) and Dravet syndrome. The project will pursue three core objectives: advancing gene-editing approaches for AHC and Dravet syndrome; generating the evidence needed to move toward first-in-human studies; and building a reproducible, repeatable platform and regulatory pathway that can be scaled to additional rare neurogenetic diseases. THRIVE is led by ARPA-H Program Manager Daria Fedyukina, Ph.D.
“PERC gives us an opportunity to stop thinking about each rare disease program as something that has to start from scratch and instead build a process that is more systematic, repeatable, and adaptable,” said Cathleen (Cat) Lutz, PhD, MBA, vice president and Evnin Family Chair of the Rare Disease Translational Center (RDTC) at The Jackson Laboratory. “AHC and Dravet syndrome are devastating childhood neurologic diseases, but they reflect a much larger challenge in genetic medicine. The long-term vision is to bring together the right disease models, editing strategies, preclinical evidence, and regulatory path so that correcting a genetic variant can become a matter of precision and speed for patients and families who urgently need new options.”
Rare diseases collectively affect hundreds of millions of people worldwide, and children are disproportionately affected. Roughly half of known rare genetic diseases involve the central nervous system, and 90% of rare childhood disorders have major neurological effects[i]. Children with these rare CNS conditions often experience severe neurologic impairment and high mortality before the age of 5
The conventional drug development model is poorly suited to rare and ultra-rare diseases that may affect only a small number of people. For rare CNS diseases, the challenge is even more complex: potential therapies must reach the brain, demonstrate safety and efficacy in rigorous preclinical models, move through regulatory review, and be evaluated in small patient populations where traditional clinical trial models may not apply
The THRIVE award will support the collaborative efforts of The Jackson Laboratory, Broad Institute, and their partners to address a central challenge in rare disease medicine: how to turn advances in genetic diagnosis and precision genetic medicine into a scalable platform for developing, evaluating, and delivering precision genetic medicines across many diseases. PERC is designed to meet that challenge by integrating recent advances in base editing, prime editing, in vivo delivery of genetic therapies to the brain, and rare disease biology.
“PERC builds on a longstanding collaboration among the Broad Institute, The Jackson Laboratory, Boston Children’s Hospital, RARE Hope, and other partners that brings together the scientific, technical, clinical, and translational capabilities needed to advance the next generation of precision medicines,” said Winston Yan, MD, PhD, Co-founder & Director of the Center for Therapeutic Genetics and Lead Investigator for PERC at the Broad Institute. “With support from ARPA-H, we have an opportunity to help establish new treatments for patients affected by AHC and Dravet syndrome, while also building a platform that can be extended to additional rare neurologic diseases. The combination of serving urgent patient needs today and creating a more scalable path for the future is exactly what this collaboration is meant to achieve.”
The Jackson Laboratory Rare Disease Translational CenterThe Jackson Laboratory Rare Disease Translational Center, under the leadership of Cat Lutz, will drive the project’s preclinical and translational work. The Center is fully focused on empowering rare disease solutions through partnerships, innovation, and scaled preclinical pipelines that help move targeted therapies from the laboratory toward the clinic. Through RDTC, The Jackson Laboratory brings deep expertise in disease genetics, model generation, preclinical therapeutic development, and rigorous testing of potential therapies for rare diseases.
JAX RDTC researchers and collaborators have conducted extensive work in AHC and Dravet syndrome, including published and ongoing research using precision genome editing in disease models. This body of work provides a critical foundation for advancing the PERC platform and evaluating whether gene-editing approaches can correct or compensate for disease-causing genetic changes in ways that support further therapeutic development
PERC: A broad coalition across science, advocacy and therapeutic developmentThe PERC project brings together a broad team of research, clinical, patient advocacy, and therapeutic development partners across 12 institutions and organizations. The collaboration includes the Broad Institute, The Jackson Laboratory, Boston Children’s Hospital, The RARE Hope Foundation, and other academic, clinical, industry, and advocacy partners working together to combine gene-editing innovation, preclinical translation, patient insight, CNS delivery technology, and clinical development expertise in a platform no single institution could build alone.
Part of a broader federal push to accelerate clinical researchThe award comes amid a broader federal effort to strengthen U.S. global leadership in clinical research and accelerate the development of lifesaving treatments. In June, HHS announced a coordinated effort to modernize clinical research, reduce unnecessary delays, increase participation in clinical trials, and ensure that the next generation of medical breakthroughs is developed in the United States. THRIVE was cited as one of ARPA-H’s major initiatives within that effort, with a focus on developing new approaches to test multiple treatments and diseases simultaneously and improving trial efficiency before patient enrollment begins.
About The Jackson LaboratoryThe Jackson Laboratory (JAX) is an independent, nonprofit biomedical research institution with a National Cancer Institute-designated Cancer Center. JAX leverages a unique combination of research, education, and resources to achieve its bold mission: to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health. Established in Bar Harbor, Maine, in 1929, JAX is a global organization with nearly 3,000 employees worldwide and campuses and facilities in Maine, Connecticut, California, Florida, New York, and Japan. For more information, please visit www.jax.org.
SOURCE The Jackson Laboratory
