The US Food and Drug Administration (FDA) approved an almost record-setting 56 drugs in 2017, including December's first approval for an adeno-associated virus (AAV) gene therapy (Luxturna) and October's registration for a second chimeric antigen receptor T-cell (CAR-T) therapy (Yescarta). Promising results were reported for bluebird bio's lentiviral-based gene therapy in adrenomyeloneuropathy, two of four monoclonal antibodies (mAbs) against calcitonin gene-related peptide (CGRP) in migraine, and Vertex's combination of its small-molecule cystic fibrosis transmembrane regulator (CFTR) corrector therapy with Kalydeco.
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DeFrancesco, L. Drug pipeline: 4Q17. Nat Biotechnol 36, 130 (2018). https://doi.org/10.1038/nbt.4073
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DOI: https://doi.org/10.1038/nbt.4073
