FDA-approved cell and gene therapies

In 2024, the US Food and Drug Administration (FDA; MD, USA) approved nine cell and gene therapy products, targeting various forms of cancer, alongside genetic conditions like hemophilia B and aromatic L-amino acid decarboxylase deficiency, offering hope to patients who previously had limited treatment options. The momentum has continued into 2025, with approvals such as Encelto for macular telangiectasia type 2 and Zevaskyn for recessive dystrophic epidermolysis bullosa. As the FDA maintains its projected pace of approving 10-20 cell and gene therapies annually, this overview examines the cell and gene therapies that are reshaping the landscape of modern medicine and offering...