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Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [ 5 ][ 6 ][ 7 ] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. [ 7 ] The gene is expressed in the liver to produce ...
If approved, Pfizer's hemophilia A gene therapy will compete with BioMarin Pharmaceutical's one-time treatment Roctavian, which was approved in the U.S. last year and is priced at $2.9 million.
681K1JDI8M. KEGG. D12434. Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A. [1][3] It was developed by BioMarin Pharmaceutical. [4][5][6] Valoctocogene roxaparvovec is made of a virus (AAV5) that has been modified to contain the gene for factor VIII, which is lacking in ...
D12908. Fidanacogene elaparvovec, sold under the brand name Beqvez among others, is a gene therapy delivered via adeno-associated virus used for the treatment of hemophilia B (congenital Factor IX deficiency). [ 1 ][ 5 ][ 8 ] Fidanacogene elaparvovec was approved for medical use in Canada in December 2023, [ 1 ] in the United States in April ...
(Reuters) -High cost, logistical issues and the prospect of potential treatment advances are holding back adoption of the first gene therapies for hemophilia, experts said this week during the ...
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
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