The Broad Institute, Boston Children’s Hospital, and the Jackson Laboratory announced the Center for Therapeutic Genetics, a new effort to develop gene therapies for rare diseases and standardize approaches for broader access. The partners aim to treat a first patient within three years and will build treatment platforms intended to work more like hospital procedures rather than bespoke development for every patient. The center is supported by a $34.5 million ARPA-H grant awarded earlier this month to a Broad-led coalition. The initial platform work targets rare pediatric epilepsies, including alternating hemiplegia of childhood due to ATP1A3 mutations and Dravet syndrome from SCN1A mutations, with expansion planned into additional indications. The initiative also cites the feasibility gap: small patient populations and regulatory challenges can limit traditional randomized trials, pushing efforts toward scalable and protocol-driven evidence generation.