Broad-Led Center to Reuse Gene Therapy Across Rare Diseases
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- Center for Therapeutic Genetics, a new nonprofit from the Broad Institute, Boston Children's Hospital, and the Jackson Laboratory, will develop gene-therapy treatments meant to be reused across diseases by swapping only the instructions to a gene editor.
- David Liu of the Broad Institute said he receives more than 20 messages per week from parents of children with ultrarare genetic disorders, and that he typically must tell them treatment is not economically feasible despite existing science.
- The project is funded by a $34.5 million contract from the Advanced Research Projects Agency for Health (ARPA-H), a federal agency backing high-risk, high-reward medical research.
- The center will initially target neurological disorders that cause seizures, with researchers hoping to demonstrate that one brain-cell editing system can be repurposed for additional genetic brain diseases including Huntington's.
- Dr. Wendy Chung of Boston Children's Hospital coined the phrase "too rare to care" to describe the access gap the center is designed to close.
- About 400 million people worldwide and 25–30 million in the U.S. have a rare disease, per the article, with half of those affected being children and a third dying before age 5.
Why it matters: A platform approach reframes rare-disease gene therapy — which the article notes typically takes years and hundreds of millions of dollars per treatment — into a reusable editing system aimed at the 400 million people worldwide currently priced out of bespoke drug development. The $34.5 million ARPA-H contract gives the Broad-led team a concrete runway starting with seizure disorders.
