The Broad Institute, Boston Children’s Hospital, and Jackson Laboratory launched a Center for Therapeutic Genetics to develop rare-disease gene therapies and establish standard protocols for broader clinical use. The collaboration is tied to a $34.5 million ARPA-H grant awarded to a Broad-led coalition earlier this month. The center is designed to address a core bottleneck in personalized and programmatic gene therapy development: aligning regulatory and healthcare delivery processes with scientific innovation. The partners said they aim to treat the first patient within three years, with early work focused on genetic treatments for rare pediatric epilepsies including alternating hemiplegia of childhood (ATP1A3) and Dravet syndrome (SCN1A). Beyond epilepsy, the initiative plans to expand into other indications, using a strategic cadence approach to translate learnings from one patient program to the next. The announcement marks another high-profile attempt to make gene therapy development more repeatable and scalable within real-world healthcare workflows.
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