First conditional marketing authorization approval in the European Union for hemophilia "A" gene therapy

Thierry VandenDriessche(1), Steven W Pipe(2), Glenn F Pierce(3), Radoslaw Kaczmarek(4)
1. Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel, Brussels, Belgium; Center for Molecular & Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, Leuven, Belgium. Electronic address:
2. Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI, USA.
3. World Federation of Hemophilia, Montreal, QC, Canada.
4. Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.

Molecular Therapy
DOI: 10.1016/j.ymthe.2022.09.020