New Center for Therapeutic Genetics Targets Rare Disease at Scale

The Broad Institute, Boston Children’s Hospital, and The Jackson Laboratory are moving away from traditional, high-cost drug development models. Instead, the Center for Therapeutic Genetics (CTG) will focus on creating a shared platform of design tools, disease models, and manufacturing protocols. By treating genetic medicine as a systemic process rather than a series of one-off breakthroughs, the coalition intends to reduce the time and expense required to bring therapies to patients with rare mutations.

Founding members include pioneers such as David Liu, who developed base and prime editing, and Timothy Yu, known for creating custom antisense drugs for individual patients. The center’s initial work is already bolstered by a $34.5 million grant from the ARPA-H THRIVE program, which will support the development of precision gene-editing treatments for rare forms of genetic epilepsy. Beyond the laboratory, the organization is engaging with regulatory bodies like the FDA and CMS to establish sustainable reimbursement and safety frameworks for therapies designed for small patient populations. While the center is currently in its founding phase, it plans to expand its reach by integrating additional institutional partners to accelerate the transition from discovery to clinical application.