Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years

Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years

Abstract: Etranacogene dezaparvovec (CSL222, formerly AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing the highly active factor IX (FIX) Padua variant controlled by a liver-specific promoter. This phase 2b, open-label, single-dose, single-arm, multicenter trial ev...

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Main Authors: Annette von Drygalski, Esteban Gomez, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan U. Lattimore, Frank W. G. Leebeek, Wolfgang A. Miesbach, Michael Recht, Paul E. Monahan, Sandra Le Quellec, Steven W. Pipe
Format: Article
Language:English
Published: Elsevier 2025-07-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952925002022
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http://www.sciencedirect.com/science/article/pii/S2473952925002022

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