Review
Nature Reviews Drug Discovery 5, 123-132 (February 2006) | doi:10.1038/nrd1955
Article series: Case Histories
Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease
Eugene W. M. Ng1, David T. Shima1, Perry Calias1, Emmett T. Cunningham, Jr.1, David R. Guyer1 About the authors & Anthony P. Adamis1
Abstract
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.
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Author affiliations
- Eyetech Pharmaceuticals, Inc., 3 Times Square, 12th Floor, New York, New York 10036, USA
Correspondence to: Anthony P. Adamis1 Email: Tony.Adamis@eyetech.com
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