STAT March 21, 2024
LONDON — Widespread genetic testing as well as the development of cutting-edge, customized genetic therapies have opened the door to treating many more inherited conditions than previously possible. A few recent cases, in which bespoke medicines were created for children with exceedingly rare mutations, have raised hopes for furthering the approach.
Now, researchers and government agencies in the U.K. are trying to figure out the nitty-gritty to replicate those early one-off successes for as many patients as possible — what sort of preclinical testing a tailor-made treatment needs to go through before being used, what regulatory thresholds each treatment needs...
Today's Sponsors
Today's Sponsor