Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease
Top Cited Papers
- 1 February 2006
- journal article
- review article
- Published by Springer Nature in Nature Reviews Drug Discovery
- Vol. 5 (2) , 123-132
- https://doi.org/10.1038/nrd1955
Abstract
Pegaptanib, an RNA aptamer that binds to vascular endothelial growth factor, was approved for the treatment of age-related macular degeneration in December 2004, becoming both the first approved aptamer therapeutic and the first anti-angiogenic agent for treating ocular disease. Aptamers are oligonucleotide ligands that are selected for high-affinity binding to molecular targets. Pegaptanib sodium (Macugen; Eyetech Pharmaceuticals/Pfizer) is an RNA aptamer directed against vascular endothelial growth factor (VEGF)-165, the VEGF isoform primarily responsible for pathological ocular neovascularization and vascular permeability. After nearly a decade of preclinical development to optimize and characterize its biological effects, pegaptanib was shown in clinical trials to be effective in treating choroidal neovascularization associated with age-related macular degeneration. Pegaptanib therefore has the notable distinction of being the first aptamer therapeutic approved for use in humans, paving the way for future aptamer applications.Keywords
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