First conditional marketing authorization approval in the European Union for hemophilia “A” gene therapy
Thierry VandenDriessche, Steven W. Pipe, Glenn F. Pierce, Radosław Kaczmarek
Topics & Concepts
Factor IXMedicineHaemophiliaGenetic enhancementClotting factorHaemophilia BHaemophilia AMarketing authorizationCoagulationInternal medicinePediatricsBioinformaticsGeneGeneticsBiologyVirus-based gene therapy researchCAR-T cell therapy researchCRISPR and Genetic Engineering