Novel Single-Dose Gene Therapy Improves Clotting Factor IX in Hemophilia B

Small, preliminary trial suggests it may free hemophilia B patients from transfusions
Small, preliminary trial suggests it may free hemophilia B patients from transfusions

HealthDay News — A single dose of an experimental gene therapy may benefit patients with hemophilia B, according to trial results presented at the annual meeting of the American Society of Hematology, held from December 3 to 6 in San Diego.

In the combined phase 1/phase 2 trial, researchers gave a single dose of the gene therapy to nine adult hemophilia B patients who ranged in age from 18 to 52. 

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The patients' levels of factor IX rose to a level of around 30. Patients who got the gene therapy were able to stop getting infusions of the clotting factor.

"At these new levels, hemophilia patients do not typically need to self-treat with factor [IX] to avoid bleeding events," lead investigator Lindsey George, MD, hematologist at Children's Hospital of Philadelphia, said in a hospital new release. "This represents a potential dramatic improvement in their quality of life and a shift in the way we think about treating hemophilia."

Children's Hospital of Philadelphia has a financial interest in a gene therapy company that spun off from the hospital. The senior author of the study was formerly a researcher at the hospital and now is president and chief scientific officer of the company.

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