title: Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease.
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.
authors.affiliation: Eyetech Pharmaceuticals, Inc., 3 Times Square, 12th Floor, New York, New York 10036, USA.
authors.forename: Eugene W M
authors.initials: EW
authors.lastname: Ng
authors.forename: David T
authors.initials: DT
authors.lastname: Shima
authors.forename: Perry
authors.initials: P
authors.lastname: Calias
authors.forename: Emmett T
authors.initials: ET
authors.lastname: Cunningham
authors.forename: David R
authors.initials: DR
authors.lastname: Guyer
authors.forename: Anthony P
authors.initials: AP
authors.lastname: Adamis
mesh_terms: D020533:Angiogenesis Inhibitors / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use*; D000818:Animals; D052157:Aptamers, Nucleotide / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D003930:Diabetic Retinopathy / Q000188:drug therapy; D016503:Drug Delivery Systems; D005123:Eye / Q000098:blood supply; D005128:Eye Diseases / Q000188:drug therapy*; D006801:Humans; D009389:Neovascularization, Pathologic / Q000188:drug therapy*; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors*
chemical_list: D020533:Angiogenesis Inhibitors; D052157:Aptamers, Nucleotide; D042461:Vascular Endothelial Growth Factor A; C495058:pegaptanib