Introduction

So far in these pages, we have described the technical aspects of aptamers and aptamer-like molecules as potential therapeutic agents. This promise has now been realized. In December 2004, pegaptanib sodium - an antivascular endothelial growth factor 165 (VEGF165) aptamer - became the first aptamer to be approved by the US Food and Drug Administration (FDA) for human therapeutic use. Pegaptanib is indicated for the treatment of neovascular age-related macular degeneration (AMD), a leading cause of blindness worldwide (Resnikoff et al., 2004), and is marketed under the trade name MacugenĀ®. This chapter will recount the story of the journey of Macugen from the laboratory to the clinic.

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