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

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.