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CSL EXPERT HIVE

Webinars on-demand

Gene therapy 1: Clinical studies

Pathology:

Haemophilia A Haemophilia B

Watch the full video recording of the first instalment of the two-part Gene therapy: The journey from science to clinical practice webinar, Part 1: What have we learnt from clinical studies? held on May 16, 2023.

This webinar was moderated by Frank Leebeek (The Netherlands) and featured presentations from Michiel Coppens (The Netherlands) and Daan Breederveld (The Netherlands). This webinar described the clinical development of a gene therapy from the Phase I/II trial through to approval, including efficacy data reported in the Phase III HOPE-B study. The impact of gene therapy on quality of life from the patients’ perspective was also provided. Finally, the safety considerations for patients were outlined.