Broad Institute, Boston Children's Hospital, and The Jackson Laboratory launch the Center for Therapeutic Genetics, a non-profit effort to develop genetic medicines for rare and ultra-rare diseases. The new collaboration aims to develop precision medicines, including base and prime editing, to treat patients with rare diseases, using infrastructure and repeatable practices the center will share with others. July 21, 2026 Broad Institute, Boston Children's Hospital, and The Jackson Laboratory today announced the Center for Therapeutic Genetics (CTG), a collaboration that will develop genetic medicines and treat patients for rare disease not as one-off breakthroughs but as a repeatable practice, and share the methods, data, and training so others can do the same. An estimated 350-400 million people worldwide live with one of approximately 8,000 rare diseases. Children are disproportionately affected, and many of these conditions are progressive, life-threatening, or debilitating, yet fewer than one in 20 has an approved treatment. Traditional drug development typically takes many years and costs hundreds of millions of dollars - a model designed for diseases affecting large populations, not for rare and ultra-rare diseases that may each affect only a few people. Recent advances in programmable genetic medicines, including base and prime editing, are paving the way toward a new model for treating rare disease. These medicines can be tailored to the specific mutation that causes a given rare disease and have already been used to treat more than 200 patients. For example, in 2018, a team led by CTG co-founder Timothy Yu at Boston Children's Hospital developed a custom antisense drug for a child with a progressive neurodegenerative disease - the first ever drug tailored for a single patient's specific disease-causing mutation. More recently, the University of Pennsylvania and Children's Hospital of Philadelphia treated an infant with a severe metabolic disorder, using a gene-editing medicine built for that child's specific mutation. This treatment used base-editing technology developed by CTG co-founder David Liu and his laboratory at the Broad Institute. CTG aims to make these patient-tailored genetic treatments for rare disease as precise, reliable, and repeatable as today's most complex medical procedures, and to make them available at scale to patients in a sustainable way. The center is founded by pioneers in genetic medicine and long-standing scientific collaborators, including David Liu, Core Institute Member and Merkin Professor at the Broad Institute and inventor of base and prime editing; Cat Lutz, Vice President, Rare Disease Translational Center, The Jackson Laboratory and a leader in rare disease model development and translational science; Timothy Yu, Staff Physician, Division of Genetics and Genomics, Boston Children's Hospital and a pioneer in individualized genetic therapies; Wendy Chung, Chief, Department of Pediatrics, Boston Children's Hospital and a leader in the clinical implementation of genomic medicine; and Winston Yan, Director of CTG, who has expertise building both nonprofit and industry biotech organizations and has brought a gene-editing therapy to the clinic. Central to the CTG model is a platform strategy, in which design tools, disease models, manufacturing processes, safety data, and clinical protocols developed for one program are shared across multiple disease programs. The center also aims to expand by welcoming additional institutions and partners as it continues to develop. Over time, the result is a scientific and regulatory infrastructure that makes genetic medicine faster, safer, less costly, and more accessible to patients. Bringing these treatments to patients will require regulatory and reimbursement frameworks suited to medicines that are, by design, made for one or a few patients. CTG members will continue to work alongside the FDA, HHS, CMS, and other authorities to generate rigorous evidence to inform the evolution of such frameworks. CTG programs will include the development of precision gene-editing treatments for children with rare forms of genetic epilepsies, an effort supported by a recent award of up to $34.5 million from the ARPA-H THRIVE program. "Scaling and sustaining treatment for ultra-rare disease is a hard problem that many scientists, clinicians, patients, and drug developers are working on. What we have here is a group of leaders who believe that by approaching genetic medicines not as products, but as a standardized clinical procedure, and by sharing what we learn openly across institutions, we can make precision genetic medicine faster, safer, less expensive, and more accessible to patients and families in need." - Winston Yan, Founding Director, Center for Therapeutic Genetics "We receive messages every week from parents asking for help: Can we do for their child what has been done for other children who have received treatments? The honest answer today is usually 'not yet', often not because the science doesn't exist, but because we don't yet have the infrastructure to bring these treatments to many patients. CTG is our commitment to closing that gap, so that eventually every family who needs this kind of treatment has a path forward." - David Liu, Broad Institute "What we are building together is scalable treatment, something our institutions will learn to do well and repeat again and again. At Boston Children's, we see children every day for whom a diagnosis is only the beginning of a much longer journey. CTG exists to close a major gap in care and ensure that what we learn from one disease accelerates treatment for the next." - Wendy Chung, Boston Children's Hospital "We are at a moment in genomic medicine where, for many rare diseases, the question is no longer whether we can treat them, but whether we will build the systems to do it. CTG answers that call." - Cat Lutz, The Jackson Laboratory "We used to see precisely tailored therapies for children with genetic diseases as remarkable exceptions. CTG is built on the conviction that they don't have to be - that the methods we develop for one rare disease will carry to the next, and eventually to the many." - Timothy Yu, Boston Children's Hospital CTG updates and information. The Center for Therapeutic Genetics (CTG) is currently in its founding phase and is not yet accepting patient referrals or providing clinical care. CTG is committed to communicating openly as the center evolves. Individuals interested in CTG's progress, potential collaborations, research opportunities, or future clinical programs are encouraged to visit centerfortherapeuticgenetics.org.

JAX Webinar Series 2026 - Topic 1: beginning a journey of knowledge sharing with the research community. On 21 July 2026, i-DNA Biotechnology Pte Ltd, in collaboration with The Jackson Laboratory (JAX) and the Singapore Association for Laboratory Animal Science (SALAS), successfully hosted Topic 1 of the JAX Webinar Series 2026, titled "Supporting Reproducibility & the 3Rs with Effective Mouse Colony Management." The webinar brought together researchers and professionals from universities, research institutes, hospitals, biotechnology companies, and laboratory animal facilities across the region. Through an insightful presentation by Dr. Neeraj Kumar Tiwari from The Jackson Laboratory, participants gained practical knowledge on effective mouse colony management to strengthen research reproducibility, improve operational efficiency, and support the implementation of the 3Rs principles (Replacement, Reduction & Refinement) in laboratory animal research. i-DNA Biotechnology sincerely thanks The Jackson Laboratory (JAX), SALAS, Dr. Neeraj Kumar Tiwari, and all participants for making Topic 1 a great success. The success of Topic 1 marks the beginning of a meaningful collaboration between JAX, SALAS, and i-DNA to foster scientific knowledge sharing and support the research community. [ Comprehensive solution] Might be you like

The Jackson Laboratory, with the Broad Institute and partners, selected for ARPA-H THRIVE award to advance Gene Editing Platform for Pediatric Epilepsies and Rare CNS diseases. Jul 09, 2026, 11:18 ET 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 Center The 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 development The 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 research The 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 Laboratory The 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