The Broad Institute, Boston Children’s Hospital, and The Jackson Laboratory launched a new Center for Therapeutic Genetics aimed at scaling genetic therapies for rare diseases and standardizing development approaches. The center is designed to deliver gene editing treatments through a more procedure-like pathway, seeking to reduce the need for separate regulatory processes for each individual use. Winston Yan, the center’s founding director, said the program will build repeatable protocols and focus on selecting patient populations and programs that can establish a “cadence of successes.” Timothy Yu of Boston Children’s described the effort as building momentum over time, with the immediate goal of treating a first patient within three years. A $34.5 million ARPA-H grant awarded earlier this month to a Broad-led coalition will fund work inside the new center, including a gene-editing platform for alternating hemiplegia of childhood (ATP1A3) and Dravet syndrome (SCN1A), with later expansion planned for liver diseases and other indications.
Get the Daily Brief