Broad Institute, Hospitals Form Gene Therapy Nonprofit
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- The Broad Institute, Boston Children's Hospital, and Jackson Laboratory in Maine announced a collaboration Tuesday to develop gene therapies for rare diseases and make the life-saving treatments cheaper and more accessible.
- The Center for Therapeutic Genetics, a newly formed nonprofit, will create precision medicines and share its methods, data, and training with other clinicians so they can replicate the work.
- Winston Yan, named founding director of the center, said the goal is to make personalized gene therapies function like clinical procedures such as organ transplants, so doctors wouldn't need separate regulatory approvals for each individual patient.
- With relatively few gene therapies on the market, families of children with rare genetic disorders currently have few options but to partner individually with researchers and raise millions of dollars to develop a treatment for their condition.
Why it matters: Families of children with rare genetic disorders currently raise millions of dollars individually for bespoke gene therapies because so few approved treatments exist. The Broad-led nonprofit targets that gap directly: by standardizing production and sharing data and training, it aims to convert costly one-off therapies into routine clinical procedures — though the impact depends on whether outside clinicians adopt the shared methods.
