July 21, 2026 —
The Broad Institute, Boston Children’s Hospital, and The Jackson Laboratory announced the launch of the Center for Therapeutic Genetics, or CTG, a collaborative initiative designed to make genetic medicines for rare diseases more repeatable, scalable, and accessible.
CTG aims to move patient-tailored genetic medicines from isolated one-off successes toward a standardized clinical practice. The center will develop genetic medicines, treat patients with rare diseases, and share methods, data, and training so that other institutions can adopt similar approaches.
Rare diseases collectively affect an estimated 350 million to 400 million people worldwide, spanning approximately 8,000 conditions. Many rare diseases are progressive, life-threatening, or debilitating, and children are disproportionately affected. Yet fewer than one in 20 rare diseases has an approved treatment.
Traditional drug development models often require many years and hundreds of millions of dollars, making them difficult to apply to ultra-rare diseases that may affect only a few patients. CTG is designed to address this gap by treating genetic medicine development more like a repeatable clinical procedure than a traditional product-by-product development pathway.
The center will focus on programmable genetic medicines, including base editing and prime editing, which can be tailored to specific disease-causing mutations. These approaches build on recent clinical examples of individualized genetic medicine, including a custom antisense therapy developed by Boston Children’s Hospital researchers for a child with a progressive neurodegenerative disease, and a mutation-specific base-editing treatment used for an infant with a severe metabolic disorder.
CTG was founded by leaders in genetic medicine and rare disease translation, including David Liu of the Broad Institute, inventor of base and prime editing; Cat Lutz of The Jackson Laboratory, a leader in rare disease model development; Timothy Yu of Boston Children’s Hospital, a pioneer in individualized genetic therapies; Wendy Chung of Boston Children’s Hospital, a leader in clinical genomic medicine; and Winston Yan, CTG’s founding director.
A central feature of CTG’s model is a shared platform strategy. Design tools, disease models, manufacturing processes, safety data, and clinical protocols developed for one program will be reused and adapted across multiple disease programs. Over time, this approach is intended to create scientific and regulatory infrastructure that can reduce development time, improve safety, and lower costs.
The center also plans to expand by welcoming additional institutions and partners. By sharing learnings across organizations, CTG aims to help build a broader ecosystem for precision genetic medicine rather than keeping each rare disease program isolated.
Regulatory and reimbursement frameworks will be a major focus. Because many patient-tailored genetic medicines may be designed for one or a few patients, CTG members plan to work with the FDA, HHS, CMS, and other authorities to generate evidence that can inform future policy models.
CTG’s initial programs will include precision gene-editing treatments for children with rare genetic epilepsies, supported by a recent award of up to $34.5 million from the ARPA-H THRIVE program.
The launch reflects a broader shift in rare disease medicine: from asking whether individualized genetic treatments are scientifically possible to building the infrastructure needed to make them reliable, sustainable, and more widely available.
