Broad Institute Unveils Rare Disease Gene Therapy Effort
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- Broad Institute, Boston Children's Hospital, and Maine's Jackson Laboratory announced a collaboration Tuesday to develop gene therapies for rare diseases and make them cheaper and more accessible to patients.
- The partners launched a non-profit called the Center for Therapeutic Genetics, which will create precision medicines and share methods, data, and training with other clinicians so they can replicate the work.
- Winston Yan, founding director of the new center, said the goal is to make personalized treatments function like clinical procedures such as organ transplants so doctors don't have to seek separate regulatory approvals for each use.
- With relatively few gene therapies currently on the market, families of children with rare genetic disorders must partner individually with researchers and raise millions of dollars to fund bespoke treatments for their conditions.
Why it matters: Families of children with rare genetic disorders raise millions individually to fund one-off treatments because few gene therapies are on the market. Winston Yan's new center will replace bespoke development per disorder with pooled methods, data, and training shared across its partner institutions.
