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1. WO2020233680 - NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PREPARATION METHOD, AND USE

Publication Number WO/2020/233680
Publication Date 26.11.2020
International Application No. PCT/CN2020/091614
International Filing Date 21.05.2020
IPC
C12N 15/113 2010.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C07H 15/04 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
15Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
02Acyclic radicals, not substituted by cyclic structures
04attached to an oxygen atom of a saccharide radical
A61K 31/713 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
713Double-stranded nucleic acids or oligonucleotides
CPC
A61K 31/713
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
713Double-stranded nucleic acids or oligonucleotides
C07H 15/04
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
15Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
02Acyclic radicals, not substituted by cyclic structures
04attached to an oxygen atom of the saccharide radical
C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
Applicants
  • 苏州瑞博生物技术股份有限公司 SUZHOU RIBO LIFE SCIENCE CO., LTD. [CN]/[CN]
Inventors
  • 张鸿雁 ZHANG, Hongyan
  • 高山 GAO, Shan
  • 康代武 KANG, Daiwu
Agents
  • 北京信诺创成知识产权代理有限公司 SINO-CREATIVITY INTELLECTUAL PROPERTY LAW FIRM
Priority Data
201910430606.122.05.2019CN
Publication Language Chinese (ZH)
Filing Language Chinese (ZH)
Designated States
Title
(EN) NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PREPARATION METHOD, AND USE
(FR) ACIDE NUCLÉIQUE, COMPOSITION PHARMACEUTIQUE, CONJUGUÉ, PROCÉDÉ DE PRÉPARATION ET UTILISATION
(ZH) 核酸、药物组合物与缀合物及制备方法和用途
Abstract
(EN)
An siRNA which inhibits kininogen (KNG) gene expression, a pharmaceutical composition containing the siRNA, and an siRNA conjugate. Each nucleotide in the siRNA is independently a modified or unmodified nucleotide. The siRNA contains a sense strand and an antisense strand. The sense strand contains nucleotide sequence I, nucleotide sequence I having the same length as the nucleotide sequence shown in SEQ ID NO: 1, with no more than three nucleotide differences. The antisense strand contains nucleotide sequence II, nucleotide sequence II having the same length as the nucleotide sequence shown in SEQ ID NO: 2, with no more than three nucleotide differences. The siRNA, the pharmaceutical composition thereof and the siRNA conjugate can effectively treat and/or prevent septicemia.
(FR)
La présente invention concerne un ARNsi qui inhibe l'expression génique de kininogène (KNG), une composition pharmaceutique contenant l'ARNsi, ainsi qu'un conjugué d'ARNsi. Chaque nucléotide dans l'ARNsi est, indépendamment, un nucléotide modifié ou non modifié. L'ARNsi contient un brin sens et un brin antisens. Le brin sens contient la séquence nucléotidique I, la séquence nucléotidique I ayant la même longueur que la séquence nucléotidique représentée dans SEQ ID NO : 1, sans plus de trois différences de nucléotides. Le brin antisens contient la séquence nucléotidique II, la séquence nucléotidique II ayant la même longueur que la séquence nucléotidique présentée dans SEQ ID NO : 2, avec pas plus de trois différences de nucléotides. L'ARNsi, la composition pharmaceutique associée et le conjugué d'ARNsi permettent de traiter et/ou de prévenir efficacement une septicémie.
(ZH)
一种抑制激肽原(KNG)基因表达的siRNA,含有该siRNA的药物组合物和siRNA缀合物。所述siRNA中的每个核苷酸各自独立地为修饰或未修饰的核苷酸,该siRNA含有正义链和反义链,所述正义链含有核苷酸序列I,所述核苷酸序列I与SEQ ID NO:1所示的核苷酸序列长度相等,且不多于3个核苷酸差异,所述反义链含有核苷酸序列II,所述核苷酸序列II与SEQ ID NO:2所示的核苷酸序列长度相等,且不多于3个核苷酸差异。所述siRNA及其药物组合物和siRNA缀合物,可以有效治疗和/或预防败血症。
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