Global Genes Releases NEXT Report 2024: Rewriting the Rules
Despite a difficult financing environment, the rare disease sector made significant advances with a sharp increase in the approval of new medicines, which included the first CRISPR Cas9 gene editing therapy for sickle cell disease, the first gene therapies for hemophilia A and Duchenne muscular dystrophy, and the first redosable gene therapy for dystrophic epidermolysis […]