A $160 million 'moonshot' backs Boston effort to revolutionize rare disease treatment. By Allyson Chiu Globe Staff, Updated July 21, 2026, 8:00 a.m. The Broad Institute, Boston Children's Hospital, and Maine's Jackson Laboratory announced a collaboration Tuesday to develop gene therapies for people with rare diseases and to make the life-saving therapies cheaper and more accessible to patients. With relatively few gene therapies on the market, families of children with rare genetic disorders have few options but to partner individually with researchers and raise millions of dollars to develop treatments for their conditions. The newly formed nonprofit Center for Therapeutic Genetics will create precision medicines and share those methods, data, and training with other clinicians so they can do the same. The goal is to make these personalized treatments more like clinical procedures, such as organ transplants, so doctors don't have to seek separate regulatory approvals for each use, said Dr. Winston Yan, founding director of the new center. "Rare disease families, they carry too much on their shoulders today, and it just feels so unfair," Yan said. "If we achieve our vision, we think there's a world in which those families can just show up to such a center and have this be the standard of care. You get a genetic diagnosis? Well, here is a procedure that will get you your genetic treatment, and that feels really exciting to me." One in 10 Americans lives with a rare disease, half of whom are children, according to the National Organization for Rare Disorders. Less than 5 percent of known rare diseases, of which there are more than 10,000, have approved treatments. Developing treatments for these diseases has been a challenge because of a lack of scientific tools and investment. But the science now exists to create transformative treatments, largely because of breakthroughs in gene editing and gene therapy. Getting treatments to patients, however, remains a problem, Yan said. "There's a lot of headwinds for biotech," Yan said, noting that many rare diseases often don't affect enough people to drive commercial investment in developing treatments. "That means that patients fundamentally don't have access to life-saving technologies, even though in many cases the science actually works. You can actually treat their diseases meaningfully with these technologies." The center's goal, he said, is to create treatment platforms, including disease models, manufacturing processes, safety data, and clinical programs that can be shared and replicated by others. The center will initially focus on developing precision gene-editing treatments for children with rare forms of genetic epilepsies, an effort supported by a federal grant through ARPA-H, the country's "moonshot" agency for health research. Earlier this month, the agency announced that it will spend up to $160 million to advance custom gene editing treatments for a number of rare diseases. "Our model is not to be family-funded for particular diseases," said Timothy Yu, one of the center's founding partners and an attending physician in the division of genetics and genomics at Boston Children's Hospital. "Our model is to try to raise money philanthropically to support the field, to pick the conditions that will advance the field the furthest, the quickest, and with the most confidence." "I don't want to set the expectation that people should be calling us to put their name on a waiting list," Yu added. The center is also currently in its "founding phase" and not yet accepting patient referrals or providing clinical care, according to Tuesday's announcement. Pamela Gavin, chief executive of the National Organization for Rare Disorders, said the center's work could address a "phenomenal need." "We're excited about what they're building because it addresses one of the greatest unmet needs in rare disease," Gavin said. "They're not only advancing the science of genetic medicine, they're also developing repeatable approaches that could make these therapies more scalable and ultimately more accessible for patients with very small populations." The new collaboration would help make critical resources available to patients and advocacy groups as well as pharmaceutical and biotechnology companies, said Patricia Musolino, a critical care and vascular neurologist at Mass General Brigham, who has been involved in gene therapy for 15 years. "We want to change the way we're thinking about genetic medicines," said Musolino, who also recently received a grant from ARPA-H to work on genomic medicines to treat rare genetic vascular diseases. In the future, gene therapy would ideally be treated more like surgical procedures, she said. "The surgeon doesn't have to go to the FDA to get approved for exactly what he's going to be doing to fix your gallbladder or your lungs," she said. "He's going to use the approved resources." But expanding access to treatment for rare diseases is a complex challenge, Gavin said. She noted that her organization and others around the country have been working to address similar issues. The National Organization for Rare Disorders runs a network of nearly 50 leading US medical and research institutions that are dedicated to advancing rare disease diagnosis, care, and research. "The science is advancing remarkably quickly," Gavin said. "The harder challenge now is building the systems that allow those scientific advances to reach patients safely, efficiently, and at scale. That's not something any one institution can solve alone."

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