The Broad Institute, Boston Children’s Hospital, and The Jackson Laboratory launched the Center for Therapeutic Genetics (CTG) on July 21, 2026. The non-profit initiative aims to standardize the development of personalized genetic medicines for rare diseases, moving away from expensive, one-off treatments toward a scalable, repeatable clinical infrastructure.
Standardizing Care for Rare Genetic Conditions
For millions of people living with rare diseases, the path to treatment has historically been fragmented and prohibitively expensive. An estimated 350 to 400 million people worldwide live with one of approximately 8,000 rare diseases, yet fewer than 5 percent of these conditions have an approved treatment, according to the National Organization for Rare Disorders. The new Center for Therapeutic Genetics (CTG) seeks to bridge this gap by treating genetic medicine as a standardized clinical procedure rather than a series of isolated, high-cost breakthroughs.
“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.”
Dr. Winston Yan, founding director of the CTG
Founding Leadership and Institutional Collaboration
The center is the result of a long-standing collaboration between pioneers in genetic medicine. Its leadership includes David Liu, a core institute member at the Broad Institute known for developing base and prime editing; Timothy Yu, a physician at Boston Children’s Hospital who pioneered custom antisense drug development; and Cat Lutz, Vice President of The Jackson Laboratory’s Rare Disease Translational Center. Wendy Chung, Chief of Pediatrics at Boston Children’s, also serves as a founding partner.
By pooling resources, the institutions aim to create a shared platform for disease models, manufacturing protocols, and clinical safety data. This infrastructure is intended to reduce the regulatory and financial hurdles that currently prevent many rare disease treatments from reaching clinical use.
Funding and Initial Clinical Focus
While the center is currently in its founding phase and not yet accepting patient referrals, it has already secured significant federal support. The CTG’s initial programs will focus on precision gene-editing treatments for rare forms of genetic epilepsy, supported by a recent award of up to $34.5 million from the ARPA-H THRIVE program.
This funding is part of a broader federal push to address the headwinds for biotech
that often discourage commercial investment in diseases affecting very small populations, as reported by The Boston Globe. The ARPA-H agency recently announced it would spend up to $160 million to advance custom gene-editing treatments for a variety of rare conditions.
Moving Beyond the “One-Off” Model
The current model of rare disease treatment often forces families to raise millions of dollars to fund individual research efforts. The CTG aims to shift this burden by creating a sustainable system where methods developed for one patient can be adapted for others. As noted in broader research contexts, the ripple effect of these breakthroughs often extends beyond rare conditions, providing insights that can be transferred to more common diseases.
“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.”
Dr. Timothy Yu, founding partner and staff physician at Boston Children’s Hospital
Regulatory and Reimbursement Challenges
A primary goal for the CTG is to work with agencies like the FDA, HHS, and CMS to establish frameworks suitable for medicines designed for small patient populations. Because current regulatory and reimbursement systems are largely built for blockbuster drugs serving large populations, the center hopes to generate the rigorous data needed to update these policies. According to The Boston Globe, Pamela Gavin, CEO of the National Organization for Rare Disorders, the new center addresses a phenomenal need
by developing repeatable approaches that could make genetic therapies both more scalable and more accessible.
As the CTG matures, it intends to welcome additional institutional partners, further expanding the network of clinicians and researchers capable of deploying these precision genetic tools. For now, the center’s leadership emphasizes that they are building the systems necessary to ensure that genetic medicine becomes a reliable, routine aspect of modern healthcare.
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