Gene therapy for haemophilia A and B, from basic principles to clinical implementation

An illustrated review

  • Ay, Cihan
  • Frenzel, Laurent
  • Pinachyan, Karen
  • Le Quellec, Sandra

  • 1Department of Medicine IClinical Division of Hematology and Hemostaseology Medical University of Vienna Vienna Austria

    2Laboratory of Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutical Implications, Labex GR‐Ex, Imagine Institute, Inserm Paris Descartes – Sorbonne Paris Cité University Paris France

    3Hematology unit care, Hemophilia Center Necker Hospital Paris France

    4 CSL Behring Europe Hattersheim am Main Germany

CorrespondenceCihan Ay, Department of Medicine I, Clinical Division of Hematology and Hemostaseology, Medical University of Vienna, Währinger Gürtel 18–20, 1090 Vienna, Austria. Email: [email protected]

Received in revised form November 21, 2023

Received July 17, 2023

Accepted November 28, 2023

This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

Haemophilia 30(1):p 5-15, January 2024. | DOI: 10.1111/hae.14907

AUTHOR CONTRIBUTIONS

All authors were involved in the preparation and the overall conceptualization of the review. Cihan Ay, Laurent Frenzel, and Sandra Le Quellec were also involved in the creation, visualization, and presentation of the published work. All authors validated and approved the published work.

CONFLICT OF INTEREST STATEMENT

Cihan Ay received personal fees for lectures and participation in advisory boards from Bayer, CSL Behring, Novo Nordisk, Pfizer, Roche, LFB, and SOBI. Laurent Frenzel received consultant fees from SOBI, Roche, and Pfizer. Karen Pinachyan and Sandra Le Quellec are full‐time employees of CSL Behring.

ETHICS APPROVAL STATEMENT

Not applicable.

PATIENT CONSENT STATEMENT

Not applicable.

PERMISSION TO REPRODUCE MATERIAL FROM OTHER SOURCES

Not applicable.

CLINICAL TRIAL REGISTRATION

Not applicable.

ACKNOWLEDGMENTS

The authors thank Dr.ir. Tonke L. de Jong of COR2ED Medical Affairs, Bottmingen, Switzerland for providing scientific and editorial support, and Dr. Yuva Oz of Art 4 Science, Amsterdam, the Netherlands for providing creative support, which was funded by CSL Behring Europe, Hattersheim am Main, Germany, in accordance with Good Publication Practice (GPP3) guidelines (http://www.ismpp.org/gpp3).

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