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Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel.

Blood advances - 14 Jan 2025

Mirza Adil, Ritsert Mona-Lisa, Tao Gloria, Thakar Himal, Lobitz Stephan, Heine Sabine, Koscher Leila, Dürken Matthias, Schmitt Anita, Schmitt Michael, Pavel Petra, Laier Sascha, Jakoby Donate, Greil Johann, Kunz Joachim, Kulozik Andreas

Abstract excerpt

ABSTRACT: Gene addition and editing strategies for transfusion-dependent β-thalassemia have gained momentum as potentially curative treatment options, with studies showcasing their efficacy and safety. We report, to our knowledge, the first real-world application of betibeglogene autotemcel (beti-cel; Zynteglo) during its period of active license in Europe from January 2020 to March 2022 for patients aged ≥12...

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PMID: 39418614
DOI: 10.1182/bloodadvances.2024014104

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