Antisense oligonucleotides containing conformationally constrained 2',4'-(N-methoxy)aminomethylene and 2',4'-aminooxymethylene and 2'-O,4'-C-aminomethylene bridged nucleoside analogues show improved potency in animal models

J Med Chem. 2010 Feb 25;53(4):1636-50. doi: 10.1021/jm9013295.

Abstract

To identify chemistries and strategies to improve the potency of MOE second generation ASOs, we have evaluated gapmer antisense oligonucleotides containing BNAs having N-O bonds. These modifications include N-MeO-amino BNA, N-Me-aminooxy BNA, 2',4'-BNA(NC)[NMe], and 2',4'-BNA(NC) bridged nucleoside analogues. These modifications provided increased thermal stability and improved in vitro activity compared to the corresponding ASO containing the MOE modification. Additionally, ASOs containing N-MeO-amino BNA, N-Me-aminooxy BNA, and 2',4'-BNA(NC)[NMe] modifications showed improved in vivo activity (>5-fold) compared to MOE ASO. Importantly, toxicity parameters, such as AST, ALT, liver, kidney, and body weights, were found to be normal for N-MeO-amino BNA, N-Me-aminooxy BNA, and 2',4'-BNA(NC)[NMe] ASO treated animals. The data generated in these experiments suggest that N-MeO-amino BNA, N-Me-aminooxy BNA, and 2',4'-BNA(NC)[NMe] are useful modifications for applications in both antisense and other oligonucleotide based drug discovery efforts.

MeSH terms

  • Alanine Transaminase / metabolism
  • Animals
  • Aspartate Aminotransferases / metabolism
  • Body Weight / drug effects
  • Cell Line
  • Drug Stability
  • Hot Temperature
  • Male
  • Mice
  • Mice, Inbred BALB C
  • Molecular Conformation
  • Nucleic Acid Denaturation
  • Nucleic Acid Heteroduplexes / chemistry
  • Oligoribonucleotides, Antisense / chemical synthesis*
  • Oligoribonucleotides, Antisense / pharmacology
  • Oligoribonucleotides, Antisense / toxicity
  • Organ Size / drug effects
  • PTEN Phosphohydrolase / biosynthesis
  • PTEN Phosphohydrolase / genetics
  • RNA, Messenger / biosynthesis
  • Structure-Activity Relationship

Substances

  • Nucleic Acid Heteroduplexes
  • Oligoribonucleotides, Antisense
  • RNA, Messenger
  • Aspartate Aminotransferases
  • Alanine Transaminase
  • PTEN Phosphohydrolase