Patent · US Active

Formats for organic compounds for use in RNA interference

US9988627B2 · kind B2 · utility

5Cited by
59References
21Claims
0Family size

Assignee

Inventors

Key dates

Filing dateOct 6, 2014
Grant dateJun 5, 2018
Priority date
Expiry dateOct 6, 2034

Classification

  • Technology area (CPC C)Chemistry; Metallurgy
  • CPC primaryC12N2310/3515
  • WIPO fieldBiotechnology
  • WIPO sectorChemistry

Abstract

The disclosure relates to compositions comprising a RNAi agent having a novel format including a spacer subunit. The disclosure relates to compositions comprising a RNAi agent having a novel format: an 18-mer format with at least one internal spacer. These RNAi agents comprise a first and a second 18-mer strand, wherein the first strand is 18 ribonucleotides or 18 total ribonucleotides and spacer subunit(s), and the second strand is 18 total ribonucleotides and spacer subunit(s), wherein: each spacer subunit consists of: (a) a phosphate or modified internucleoside linker and (b) a spacer; a spacer subunit can be at any position in the strand; the two strands form a duplex with at least one blunt end; and the 3 end of one or both strands terminates in a phosphate or modified internucleoside linker and further comprises, in 5 to 3 order: a second spacer; a second phosphate or modified internucleoside linker, and a 3 end cap. In various embodiments, the RNAi agents comprise a first and a second strand, wherein each strand is a 30-mer or shorter, the first strand comprises ribonucleotides, and the second strand comprises ribonucleotides and one or more spacer subunit(s), wherein: each …

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