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Mol Ther. 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.

Author information

1
Department of Medical Oncology and Experimental Therapeutics, City of Hope Medical Center, 1500 East Duarte Road, Duarte, CA 91010, USA. Electronic address: cstein@coh.org.
2
Department of Medical Oncology and Experimental Therapeutics, City of Hope Medical Center, 1500 East Duarte Road, Duarte, CA 91010, USA.

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

PMID:
28366767
PMCID:
PMC5417833
DOI:
10.1016/j.ymthe.2017.03.023
[Indexed for MEDLINE]
Free PMC Article

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