Gene therapy shows promise for haemophilia, but could be most expensive U.S. drug ever


20 June 2020 - Jack Grehan, who was born with hemophilia, used to inject himself every couple of days with a protein he needs for his blood to clot. But not anymore.

"It's been absolutely brilliant and life-changing for me," says Grehan, 26, of Billinge in North West England. He received an experimental gene therapy in 2017 that, at least for now, has eliminated his need for regular injections. "I can just go about my day and not have to worry."

Based on experiences like Grehan's, the company that developed the therapy is seeking approval in Europe and the United States to start selling the first gene therapy for haemophilia. That's generating excitement among patients, patient advocates and doctors.

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Michael Wonder

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Michael Wonder