New Center Seeks to Make Tailored Genetic Therapies Reliable and Repeatable

Rare Daily Staff

The Broad Institute of MIT and Harvard, Boston Children’s Hospital, and The Jackson Laboratory have launched the Center for Therapeutic Genetics (CTG), a collaborative, non profit effort to develop precision genetic medicines for rare and ultra rare diseases. The center’s mission is to build a repeatable playbook—methods, data, and training that can be shared so clinicians and institutions can treat more patients using similar tools.

Over the past decade, scientists have begun to show that it is possible to design medicines for a single person by targeting the exact mutation that causes their disease. In 2018, a team led by Boston Children’s neurologist and CTG co founder Timothy Yu created a custom antisense drug for a young girl with a fatal neurodegenerative disorder—the first treatment tailored specifically to one patient’s unique genetic error.

More recently, clinicians at the University of Pennsylvania and Children’s Hospital of Philadelphia treated an infant with a severe metabolic disorder using a base editing medicine built for that child’s mutation, based on technology developed by CTG co founder David Liu and colleagues at the Broad Institute. These and similar cases have helped spur a new field some researchers call “interventional genetics,” in which gene editing or other programmable tools are used as modular platforms that can be adapted to different patients. CTG’s founders say their goal is to make these tailored treatments as reliable and repeatable as complex procedures like organ transplants, where the process is standardized even though each patient’s case is unique.

CTG brings together several leaders in genetic medicine and rare disease research. The founding team includes David Liu, a Broad Institute scientist whose lab invented base and prime editing; Boston Children’s physicians Timothy Yu and Wendy Chung, who have pioneered individualized genetic therapies and clinical genomic medicine; Cat Lutz, head of the Rare Disease Translational Center at The Jackson Laboratory; and Winston Yan, the center’s founding director, who has helped build both nonprofit and industry gene editing programs.

“What we are building together is scalable treatment, something our institutions will learn to do well and repeat again and again,” said Boston Children’s Chung. “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.”

Yan describes the effort as a shift in how the field thinks about treatment for ultra rare diseases: not as boutique products, but as standardized clinical procedures built on shared platforms. Liu and others involved in the center say they regularly hear from families asking whether similar custom treatments could be developed for their children, and that a major barrier is not the science itself but the lack of infrastructure to deliver such therapies at scale.

Central to CTG’s model is a platform strategy: design tools, disease models, manufacturing methods, safety data, and clinical protocols developed for one condition will be reused and adapted across many programs rather than rebuilt from scratch each time. The aim is that, over time, this shared infrastructure will make genetic medicines faster to develop, safer to test, and less expensive to deliver—even when only a handful of patients have a particular disease.

To jump start the work, CTG and its partners have secured up to $34.5 million through ARPA H’s THRIVE program, part of a broader federal “moonshot” investment of up to $160 million in custom gene editing treatments for rare diseases. The center’s first clinical focus will be on precision gene editing therapies for children with severe genetic epilepsies, leveraging emerging data that gene editing can correct disease causing mutations in brain cells and dramatically reduce seizures in animal models.

Building a system where medicines are, by design, made for one or a few patients will require regulatory and reimbursement frameworks different from those used for conventional drugs. CTG members plan to work closely with agencies such as the U.S. Food and Drug Administration and Centers for Medicare & Medicaid Services to generate evidence and help shape policies that can accommodate individualized therapies while maintaining rigorous safety standards.

Photo: Wendy Chung, Boston Children’s Hospital physician