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Review
. 2017 May 3;25(5):1069-1075.
doi: 10.1016/j.ymthe.2017.03.023. Epub 2017 Mar 31.

FDA-Approved Oligonucleotide Therapies in 2017

Cy A Stein  1 , Daniela Castanotto  2
Affiliations
Review

FDA-Approved Oligonucleotide Therapies in 2017

Cy A Stein et al. Mol Ther. .

Abstract

Oligonucleotides (oligos) have been under clinical development for approximately the past 30 years, beginning with antisense oligonucleotides (ASOs) and apatmers and followed about 15 years ago by siRNAs. During that lengthy period of time, numerous clinical trials have been performed and thousands of trial participants accrued onto studies. Of all the molecules evaluated as of January 2017, the regulatory authorities assessed that six provided clear clinical benefit in rigorously controlled trials. The story of these six is given in this review.

Keywords: Defitelio; Eteplirsen; Kynamro; Macugen; Spinraza; Vitravene; aptamer; oligonucleotide; phosphorothioate; therapeutics.

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