Broad Institute launches Center for Therapeutic Genetics for rare diseases

The Broad Institute, in collaboration with Boston Children's Hospital and The Jackson Laboratory, has launched the Center for Therapeutic Genetics (CTG), a nonprofit initiative dedicated to developing genetic medicines for rare and ultra-rare diseases.

CTG will use a platform-based approach where research, methods, data, and clinical practices are shared across multiple rare disease programs, making personalized genetic treatments more repeatable and scalable, similar to standardized medical procedures.

The Center for Therapeutic Genetics is in its founding phase and not yet accepting patient referrals or clinical care, but aims to expand access to bespoke genetic therapies for patients who currently have no treatment options.

CTG's initial focus includes severe pediatric neurological disorders such as rare genetic epilepsies, supported by an up to $34.5 million ARPA-H THRIVE grant for the Pediatric Epilepsies and Rare CNS (PERC) gene editing platform.

CTG is led by physician-scientist Winston Yan, MD, PhD, and involves prominent researchers including David R. Liu, Wendy Chung, and Tim Yu, reflecting a cross-institutional effort to advance precision genetic medicine for rare disease.