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‘Too Rare to Care’? A New Center for Rare Diseases Hopes to Change That
The New York Times • • business
Key Points:
- Researchers at the Broad Institute, Boston Children’s Hospital, and Jackson Laboratory have launched a nonprofit, the Center for Therapeutic Genetics, to develop reusable gene therapy treatments for rare diseases.
- The center aims to treat rare genetic disorders by creating gene-editing therapies that can be adapted for multiple diseases without starting from scratch each time.
- This approach addresses the current challenge where developing gene therapies for ultra-rare diseases is economically unfeasible, often described as “too rare to care.”
- The project received a $34.5 million contract from the Advanced Research Projects Agency for Health and will initially focus on neurological disorders causing seizures, with hopes to extend to other genetic brain diseases.
- Approximately 400 million people worldwide suffer from rare diseases, half of whom are children, highlighting the potential broad impact of this new gene therapy development model.
