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1. WO2020063198 - SIRNA CONJUGATE, PREPARATION METHOD THEREFOR AND USE THEREOF

Publication Number WO/2020/063198
Publication Date 02.04.2020
International Application No. PCT/CN2019/101653
International Filing Date 20.08.2019
IPC
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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
A61P 31/20 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
12Antivirals
20for DNA viruses
CPC
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 31/20
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
12Antivirals
20for DNA viruses
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
  • 陈庚容 CHEN, Gengrong
Agents
  • 北京英创嘉友知识产权代理事务所(普通合伙) INNOTRACK INTELLECTUAL PROPERTY LAW FIRM
Priority Data
201811165363.530.09.2018CN
Publication Language Chinese (ZH)
Filing Language Chinese (ZH)
Designated States
Title
(EN) SIRNA CONJUGATE, PREPARATION METHOD THEREFOR AND USE THEREOF
(FR) CONJUGUÉ D'ARN INTERFÉRENT COURT, SON PROCÉDÉ DE PRÉPARATION ET UTILISATION ASSOCIÉE
(ZH) 一种siRNA缀合物及其制备方法和用途
Abstract
(EN)
An siRNA conjugate having a structure as represented by formula (1) for inhibiting hepatitis B virus gene expression, comprising siRNA and a conjugated group, wherein the sense strand of the siRNA comprises a nucleotide sequence 1, and the antisense strand comprises a nucleotide sequence 2; the nucleotide sequence 1 and the nucleotide sequence 2 are, at least in part, reversely complementary to form a double-stranded region; the nucleotide sequence 1 and SEQ ID NO: 1 are equal in length and differ by no more than three nucleotides; the nucleotide sequence 2 and SEQ ID NO: 2 are equal in length and differ by no more than three nucleotides. The siRNA conjugate can specifically target liver cells and effectively solve the problem of siRNA delivery in vivo, and shows excellent activity and low toxicity to inhibit HBV gene expression while maintaining high stability of siRNA.
(FR)
L'invention concerne un conjugué d'ARN interférent court ayant une structure telle que représentée par la formule (1) permettant d'inhiber l'expression du gène du virus de l'hépatite B, comprenant un ARN interférent court et un groupe conjugué, le brin sens de l'ARN interférent court comprenant une séquence nucléotidique 1, et le brin antisens comprenant une séquence nucléotidique 2 ; la séquence nucléotidique 1 et la séquence nucléotidique 2 sont, au moins en partie, inversement complémentaires pour former une région bicaténaire ; la séquence nucléotidique 1 et la SEQ ID No : 1 sont de même longueur et diffèrent d'un nombre inférieur ou égal à trois nucléotides ; la séquence nucléotidique 2 et la SEQ ID No : 2 sont de même longueur et diffèrent d'un nombre inférieur ou égal à trois nucléotides. Le conjugué d'ARN interférent court peut cibler spécifiquement les cellules hépatiques et résoudre efficacement le problème de l'administration d'ARN interférent court in vivo, et présente une excellente activité et une faible toxicité pour inhiber l'expression du gène du VHB tout en maintenant une stabilité élevée de l'ARN interférent court.
(ZH)
具有如式(1)所示结构的抑制乙型肝炎病毒基因表达的siRNA缀合物,包含siRNA和缀合基团,其中,该siRNA的正义链包含核苷酸序列1,反义链包含核苷酸序列2,核苷酸序列1和核苷酸序列2至少部分地反向互补形成双链区,核苷酸序列1与SEQ ID NO:1长度相等,且不多于3个核苷酸差异;核苷酸序列2与SEQ ID NO:2长度相等,且不多于3个核苷酸差异。该siRNA缀合物能够特异性地靶向肝细胞,有效解决siRNA体内递送问题,在保持siRNA高度稳定的同时,还显示出优异的抑制HBV基因表达的活性和低毒性。
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