Chancellor announces support for Rare Therapies Launch Pad

https://www.paediatrics.ox.ac.uk/news/chancellor-announces-support-for-rare-therapies-launch-pad?utm_campaign=morning_rounds&utm_medium=email&_hsmi=299162334&_hsenc=p2ANqtz--cO6lxbJqaYmRE4rEUX3i1rvRWx97Ai-ExoJwMB4eJt_Z7SBSq6ggJjrDDovFUoKlUeZCsR97EcpgA5EUOc-Bm2kzGDQ&utm_content=299162334&utm_source=hs_email U.K. researchers and government agencies team up to accelerate individual genetic therapies New customized genetic therapies built on now-widespread genetic testing are far from everyday treatments, but they have entered the realm of the possible, raising hopes for children with diseases stemming from the rarest of mutations. To better find answers for more inherited conditions, researchers and government agencies in the U.K. have launched efforts to expand what have been bespoke successes to reach more patients. “A lot of the barriers are no longer scientific — we can make these drugs,” said Matthew Wood, a professor of pediatrics at the University of Oxford. “It’s more about, can we make them at a cost that is reasonable enough? And can we get the regulators to reduce the barriers to doing this so that when a patient shows up, you can actually make a drug quickly enough to benefit that patient, rather than it costing huge amounts of money and taking two or three years to do it?” STAT’s Andrew Joseph reports on two initiatives (Rare Therapies Launch Pad and UPNAT) and tells us how the story of milasen, an N-of-1 drug for a girl named Mila with Batten disease, resonates across the Atlantic. U.K. health officials and researchers try to carve a path for more individualized genetic treatments Andrew Joseph By Andrew Joseph March 21, 2024 https://www.statnews.com/2024/03/21/uk-individualized-genetic-treatments/?utm_campaign=morning_rounds&utm_medium=email&_hsmi=299162334&_hsenc=p2ANqtz-9OgHX7qQjY9O_4l0stEQJQJVNIyDaDWc_JESa5y4Uo4Hl30CjFRejMZ7ZD4SQQx5m1mwD5kao4_pvsD5V43hgEKXyEDA&utm_content=299162334&utm_source=hs_email Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease Meghana Keshavan By Meghana Keshavan Oct. 22, 2018 https://www.statnews.com/2018/10/22/a-tailor-made-therapy-may-have-halted-a-rare-disease/?utm_campaign=morning_rounds&utm_medium=email&_hsmi=299162334&_hsenc=p2ANqtz--6Wyiu1xNhNkfBc1D_Vi-ygm01C994CMZrePX4L5P8uI9PXEEK0l8G5TxheFJmUznrAjx4M0OD_qG7-Fb6sIdzzY-N6Q&utm_content=299162334&utm_source=hs_email