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STAT April 25, 2024
LONDON — Claire Booth, a gene therapy researcher in London, had hoped that a biotech company would take her team’s work on an experimental medication for an ultra-rare children’s disease and get it to market. It didn’t happen.
Now, in an unusual step, the hospital where she works is trying to get the medicine approved on its own.
Booth, who leads the stem cell gene therapy program at London’s Great Ormond Street Hospital...
