Science & Nature
The European regulator has given gene therapy Hemgenix from uniQure/CSL Behring a conditional approval, making it the first in the region for treating hemophilia B. Hemgenix (etranacogene dezaparvovec) offers patients a possible one-time alternative to lifelong intravenous injections of factor IX. In affected individuals, the mutated F9 gene causes partial or complete lack of the clotting protein factor IX, leading to coagulation problems and, in severe cases, bleeding into joints. Hemgenix is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the highly active Padua variant of a codon-optimized human F9 controlled by a liver-specific promoter. It is administered intravenously at a dose of 2 × 1013 genome copies per kilogram body weight.
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EU gets first hemophilia B gene therapy.
Nat Biotechnol 41, 438 (2023). https://doi.org/10.1038/s41587-023-01760-5
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DOI: https://doi.org/10.1038/s41587-023-01760-5
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Tama Kazmierczak