Breakthrough treatments are fortunately quite frequent but in 2023, the most excitement for an innovative product was reserved for Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics AG's Casgevy (exagamglogene autotemcel), the first-ever CRISPR gene-edited medicine to get approval, firstly in the UK and then in the US, for sickle cell disease and transfusion-dependent beta thalassemia. (Also see "Vertex Achieves Gene-Editing Milestone With First Ever CRISPR Therapy Approval" - Scrip, 16 November, 2023.)
The thumbs-up stateside came on the same day in December that the US Food and Drug Administration approved bluebird...
