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UK Hospital, Led by Dr. Booth, Seeks ADA-SCID Gene Therapy Approval

Apr 25, 2024, 07:37 PM

A UK hospital is taking an unprecedented step to seek approval for an ADA-SCID gene therapy, a rare disease, after many biopharma companies have abandoned similar efforts. This initiative, led by Dr. Booth, marks a significant shift from traditional pharmaceutical company applications to a hospital-led approach. The move has sparked discussions about the NHS's systems for approving new medicines, with criticism from Tanya Curry, CEO of the Motor Neurone Disease Association, who stated, 'It’s appalling that people with a particular genetic subtype of motor neurone disease face being denied what could be a life-changing treatment because of the NHS’s outdated systems for approving new medicine.'