The Broad Institute, Boston Children’s Hospital, and Jackson Laboratory launched a new Center for Therapeutic Genetics aimed at scaling gene therapies for rare diseases while standardizing development processes. The effort is framed as a push to make bespoke therapies more like “clinical procedures,” reducing the need for separate regulatory paths for each individual use. The center plans to treat its first patient within three years, supported by a $34.5 million ARPA-H grant awarded earlier this month. Focus indications include rare pediatric epilepsies driven by ATP1A3 mutations and Dravet syndrome (SCN1A), with additional expansion planned for liver diseases and other conditions. A key operational theme is developing gene-editing platforms that can create reusable tools, data, and training for the broader field. The initiative also highlights an emerging alternative to traditional trial structures when patient populations are small.
Get the Daily Brief