Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular 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.
Ng
Eugene W M
EW
Eyetech Pharmaceuticals, Inc., 3 Times Square, 12th Floor, New York, New York 10036, USA.
Shima
David T
DT
Calias
Perry
P
Cunningham
Emmett T
ET
Guyer
David R
DR
Adamis
Anthony P
AP
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*
D020533:Angiogenesis Inhibitors; D052157:Aptamers, Nucleotide; D042461:Vascular Endothelial Growth Factor A; C495058:pegaptanib