In a world where science can already create drugs for the specific mutation of a single child, millions of families still remain without help. It is precisely this gap between possibility and accessibility that three leading medical centers decided to close, announcing on July 21, 2026, the creation of the Center for Therapeutic Genetics — an organization that intends to turn the miracles of personalized genetic medicine into standard clinical practice.
The problem is immense and weighs on healthcare worldwide. It is estimated that 350 to 400 million people globally suffer from one of approximately 8,000 rare diseases. Children are disproportionately affected — nearly half of patients with rare diseases are children, many of whom do not live to see their fifth birthday. The diseases are relentlessly progressive and life-threatening, yet approved treatments exist for less than one in twenty such conditions. The traditional drug development model, designed for millions of patients and requiring hundreds of millions of dollars and many years of investment, simply doesn't work here: there may be only a few people in the world for each ultra-rare disease.
The Center brings together global pioneers in genetic medicine who have already proven that individual genetic drugs are real and save lives. The founders include David Liu (Broad Institute, inventor of base editing and prime editing), Timothy Yu (Boston Children's Hospital, pioneer of personalized gene therapies), Kat Lutz (The Jackson Laboratory, a leader in translational science and rare disease model development), Wendy Chung (Chief of Pediatric Genetics and Genomic Medicine at Boston Children's Hospital and a specialist in the clinical implementation of genomic medicine), and the Center's Director Winston Yan (an experienced organizer of biotech institutions who has already brought one gene therapy to clinic). This team is convinced that the question is not whether we can treat rare diseases, but whether we will build the infrastructure for it.
The key approach is a platform strategy of open collaboration. Design tools, disease models, manufacturing processes, safety data, and clinical protocols developed for one rare disease will be openly shared with other teams. This exchange is intended to accelerate the development of the next treatment, reduce its cost, and increase reliability. The Center is already working on creating gene editors for rare genetic epilepsies in children, supported by an ARPA-H grant of up to $34.5 million under the THRIVE program (Therapeutic research in hereditary rare diseases using in vivo precision gene editing). This grant focuses on two particularly severe conditions: Alternating Hemiplegia of Childhood and Dravet Syndrome.
The success story began even before the Center's official establishment. In 2018, Timothy Yu's team developed a custom antisense drug for a child with a progressive neurodegenerative disease — the first-ever drug made for a specific patient and their specific mutation. Later, David Liu's base editing technologies helped treat an infant with a severe metabolic disorder. Most recently, a personalized gene therapy based on Liu's technologies saved the life of a patient with a rare blood disorder. Now, these methods and new approaches are planned to be scaled up through close collaboration with regulators — the FDA, HHS, and CMS — on appropriate rules for personalized drugs, which require a fundamentally new approval logic.
The project participants emphasize the main point: science already allows for the treatment of many rare diseases; the issue is purely with infrastructure and scaling. The Center is not yet accepting patients and is in the formation phase, but it intends to openly share all achieved results and actively invite new partners from academia, industry, patient organizations, and regulatory bodies. The main goal is to create a system where each new treatment accelerates the development of the next, rather than remaining an isolated exception accessible only to a select few.
When the infrastructure allows for the repetition of complex medical procedures, personalized genetic therapy ceases to be a miracle and becomes accessible help — this is precisely what the three institutions intend to build in the coming years.
