New Broad-Led Center Aims to Make Gene Therapies Repeatable
With up to $34.5 million from ARPA-H, the new nonprofit aims to standardize gene-editing therapies for rare diseases, starting with childhood epilepsies.
The Broad Institute, Boston Children’s Hospital, and the Jackson Laboratory are teaming up to turn one-off, patient-specific gene therapies into repeatable treatments for rare diseases.
Announced July 21, the new Center for Therapeutic Genetics (CTG), will serve as a hub for design tools, disease models, manufacturing processes, safety data and clinical protocols across multiple disease programs.
“We receive messages every week from parents asking for help: Can we do for their child what has been done for other children who have received treatments?” David Liu, a researcher at the Broad Institute and Harvard University who developed base editing technology that will be used at CTG, said in a statement. “The honest answer today is usually ‘not yet’, often not because the science doesn’t exist, but because we don’t yet have the infrastructure to bring these treatments to many patients. CTG is our commitment to closing that gap.”
The center’s first program will develop precision gene-editing treatments for children with rare forms of genetic epilepsy, backed by up to $34.5 million from the Advanced Research Projects Agency for Health (ARPA-H) THRIVE (Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines) program. Winston Yan — a physician scientist who came out of the lab of CRISPR pioneer Feng Zhang — is founding director, working alongside Liu, Cat Lutz of the Jackson Laboratory, and Timothy Yu and Wendy Chung of Boston Children’s Hospital.
CTG said it plans to add institutions and partners over time and will work with the FDA, US Department of Health and Human Services and the Centers for Medicare & Medicaid Services to help shape regulatory and reimbursement frameworks suited to medicines designed for one or a few patients.
In its announcement, the CTG estimated that 350 million to 400 million people worldwide live with one of approximately 8,000 rare diseases, with fewer than one in 20 of those conditions having an approved treatment. The traditional drug development system is not built to address these patients; meantime, advances in gene-editing technologies have led to treatments in more than 200 patients already.
In 2018, a team led by CTG co-founder Timothy Yu at Boston Children’s developed a custom antisense drug for a child with a progressive neurodegenerative disease and researchers at the University of Pennsylvania and Children’s Hospital of Philadelphia have used Liu’s base-editing technology to treat an infant with a severe metabolic disorder.
“Scaling and sustaining treatment for ultra-rare disease is a hard problem that many scientists, clinicians, patients, and drug developers are working on,” Winston Yan said. “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.”