VIDEO: Updated data on hemophilia A gene therapies presented at ASH
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In this video, Lindsey A. George, MD, discussed updated clinical trial data around hemophilia gene therapy presented at ASH Annual Meeting and Exposition.
George, assistant professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania, and director of clinical in vivo gene therapy and attending hematologist at the Children’s Hospital of Philadelphia, talked about two presentations in first-generation approaches in hemophilia A gene therapy.
In one presentation, researchers reported on a gene therapy for hemophilia A that uses a factor VIII V3 construct.
“What I think was really interesting about their data is that they showed initially high levels of expression, but these patients actually stabilized in the low-mild range of hemophilia A for up to 5 years,” George said.
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