Single-dose gene therapy could change lives for adults with hemophilia B

with adults hemophilia B After a single injection of , the number of bleeding episodes was reduced by an average of 71%. gene therapyaccording to international organizations Phase III clinical trial. The result is New England Medical Journal According to researchers Perelman School of Medicine and a multicenter group of researchers.

Hemophilia is a genetic disorder that limits the blood's ability to clot and affects approximately 30,000 people in the United States, most of them men. If left untreated, it can lead to spontaneous bleeding, especially in the joints, which can lead to painful joint damage and mobility problems over time. Hemophilia B is caused by a deficiency of clotting factor IX. Gene therapy allows the liver to produce factor IX, which helps blood clot and protects patients from frequent bleeding.

“What we saw from the patients in this study was that within a few days of receiving the gene therapy infusion, the gene therapy took hold and their bodies started producing factor IX for the first time in their lives,” said the study. says the lead author. adam cooker Chief of Hematology Department and Chief of Clinical Practice Penn Blood Disorders Center and Penn's Comprehensive Hemophilia Program. “We always want to be careful in using the word 'cure,' especially until we have long-term follow-up data, but for many of these patients, it was life-changing.”

Based on the results of this study, FDA approves gene therapy (fidanacogene elaparvovec) in April 2024. Cuker was the site director for the clinical trial at Penn Medicine, one of the sites with the highest enrollment in the study.

“I hear from people born with hemophilia that even when the disease is well-managed, they always have this burden in the back of their minds: frequent IV drips, treatment costs, and IV drip plans while traveling. There's always the need to do this, what to do if bleeding occurs, etc.,” Cukor said. “Now that patients treated in this study have been virtually cured of hemophilia, they are telling us about achieving a new 'hemophilia-free state of mind.' It's great to see patients so happy with their new reality.”

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