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Title:
DOUBLE-STRANDED RNA COMPRISING NUCLEOTIDE ANALOG CAPABLE OF REDUCING OFF-TARGET TOXICITY
Document Type and Number:
WIPO Patent Application WO/2024/002007
Kind Code:
A1
Abstract:
The present invention provides a double-stranded RNA comprising a nucleotide analog. The double-stranded RNA of the present invention exhibits one or more of enhanced stability, reduced off-target toxicity, and enhanced effectiveness.

Inventors:
HUANG JINYU (CN)
ZOU HAO (CN)
GUO HONGLI (CN)
Application Number:
PCT/CN2023/102403
Publication Date:
January 04, 2024
Filing Date:
June 26, 2023
Export Citation:
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Assignee:
SHANGHAI RONA THERAPEUTICS CO LTD (CN)
International Classes:
C07H19/167; A61K31/713; C12N15/113; C12N15/85
Domestic Patent References:
WO2020198509A22020-10-01
Foreign References:
CN116003494A2023-04-25
CN112805383A2021-05-14
CN102264374A2011-11-30
Other References:
KEN YAMADA ET AL.: "Structurally Constrained Phosphonate SIGMAnternucleotide Linkage Impacts Oligonucleotide-Enzyme Interaction, and Modulates siRNA Activity and Allele Specificity", NUCLEIC ACIDS RESEARCH, vol. 49, no. 21, 25 November 2021 (2021-11-25), pages 12069 - 12088, XP093003120, ISSN: 1362-4962, DOI: 10.1093/nar/gkab1126
DANIEL HUTTE ET AL.: "From Phosphate to Bis(methylene) Sulfone: Non-Ionic Backbone Linkers in DNA", HELVETICA C&T!TMICA ACTA, vol. 85, no. 9, 31 December 2002 (2002-12-31), pages 2777 - 2806, XP071268918, ISSN: 0018-019X, DOI: 10.1002/1522-2675(200209)85:9<2777::AID-HLCA2777>3.0.CO;2-1
SAHAR ABBAS ET AL.: "Commercially Available 5'-DMT Phosphoramidites as Reagents for the Synthesis of Vinylphosphonate-Linked Oligonucleic Acids", ORGANIC LETTERS, vol. 3, no. 21, 25 September 2001 (2001-09-25), pages 3365 - 3367, XP055055129, ISSN: 1523-7060, DOI: 10.1021/ol0166045
HORIBA MASAHIKO, YAMAGUCHI TAKAO, OBIKA SATOSHI: "Synthesis and Properties of Oligonucleotides Having Ethynylphosphonate Linkages", THE JOURNAL OF ORGANIC CHEMISTRY, AMERICAN CHEMICAL SOCIETY, vol. 85, no. 4, 21 February 2020 (2020-02-21), pages 1794 - 1801, XP093127803, ISSN: 0022-3263, DOI: 10.1021/acs.joc.9b01318
Attorney, Agent or Firm:
LIU , SHEN & ASSOCIATES (CN)
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