Broad Institute, Boston Children’s Hospital and the Jackson Laboratory have launched the Center for Therapeutic Genetics, a non-profit effort to develop genetic medicines for rare and ultra-rare diseases.
The new collaboration aims to develop precision medicines, including base and prime editing, to treat patients with rare diseases, using infrastructure and repeatable practices the center will share with others. CTG programs will include the development of precision gene-editing treatments for children with rare forms of genetic epilepsies, an effort supported by a recent award of up to $34.5 million from the ARPA-H THRIVE program.
The center is founded by pioneers in genetic medicine and long-standing scientific collaborators, including David Liu, inventor of base and prime editing; Cat Lutz, a leader in rare disease model development and translational science; Timothy Yu, a pioneer in individualized genetic therapies; Wendy Chung, a leader in the clinical implementation of genomic medicine; and Winston Yan, who has expertise building both nonprofit and industry biotech organizations and has brought a gene-editing therapy to the clinic.
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