WO2016021673A1 - Procédé permettant de prédire l'efficacité médicamenteuse d'une composition pharmaceutique contenant un petit arn - Google Patents

Procédé permettant de prédire l'efficacité médicamenteuse d'une composition pharmaceutique contenant un petit arn Download PDF

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Publication number
WO2016021673A1
WO2016021673A1 PCT/JP2015/072303 JP2015072303W WO2016021673A1 WO 2016021673 A1 WO2016021673 A1 WO 2016021673A1 JP 2015072303 W JP2015072303 W JP 2015072303W WO 2016021673 A1 WO2016021673 A1 WO 2016021673A1
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WO
WIPO (PCT)
Prior art keywords
small rna
pharmaceutical composition
containing pharmaceutical
sirna
complex
Prior art date
Application number
PCT/JP2015/072303
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English (en)
Japanese (ja)
Inventor
史一 篠原
俊正 春元
Original Assignee
協和発酵キリン株式会社
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Publication date
Application filed by 協和発酵キリン株式会社 filed Critical 協和発酵キリン株式会社
Publication of WO2016021673A1 publication Critical patent/WO2016021673A1/fr

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.

Definitions

  • sugar-modified nucleotides include cross-linked artificial nucleic acids (BridgedridgeNucleic Acid, BNA) having two cyclic structures by introducing a cross-linked structure into the sugar portion, and specifically, an oxygen atom at the 2 ′ position And 4'-position carbon atoms cross-linked via methylene (Locked Nucleic Acid, LNA), ethylene-bridged artificial nucleic acid (Ethylene bridged nucleic acid, ENA) [Nucleic Acid Research, 32, In addition, peptide nucleic acids (PNA) [Acc. Chem. Res., 32, 624 (1999)], oxypeptide nucleic acids (OPNA) [J. Am. Chem. Soc., 123, 4653 (2001) ], Peptide ribonucleic acid (PRNA) [J. Am. Chem. Soc., 122, 6900 (2000)].
  • BNA cross-linked artificial nucleic acids
  • LNA Locked Nucleic Acid
  • ENA
  • siRNA used in the present invention for example, a nucleic acid molecule (WO2005 / 089287) that generates siRNA by the action of a ribonuclease such as Dicer, or a blunt end is formed without a 3 ′ or 5 ′ end overhang.
  • siRNA siRNA with a protruding sense strand (US2012-0040459), single-stranded siRNA reported by Walt et al. (Cell, 150, 883-894, 2012), and the like can also be used.
  • the dosage of the pharmaceutical composition used in the present invention is preferably determined in consideration of the drug, dosage form, patient condition such as age and weight, administration route, nature and degree of disease, etc.
  • the mass is 0.1 mg to 10 g / day, preferably 1 mg to 500 mg / day per day for an adult. In some cases, this may be sufficient, or vice versa. It can also be administered once to several times a day, and can be administered at intervals of 1 to several days.
  • the drug effect in the method for selecting a cancer type of the present invention refers to an effect of ameliorating or curing a cancer symptom possessed by a small RNA-containing pharmaceutical composition.
  • administration of a small RNA-containing pharmaceutical composition to a subject means that the tumor becomes smaller than before administration.
  • the cancer type of the subject can be selected as a cancer that is effective as the cancer type of the subject to which the small RNA-containing pharmaceutical composition is administered.
  • the present invention provides a method for predicting the efficacy of a small RNA-containing drug and a method for evaluating the knockdown effect of a target gene of a small RNA-containing drug. Further, according to the present invention, it becomes possible to select an effective small-RNA-containing drug, and to select patients who effectively exhibit the medicinal effect of the small-RNA-containing drug.

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

　L'invention concerne un procédé d'évaluation de l'effet d'une composition pharmaceutique contenant un petit ARN en matière d'inhibition d'un gène cible. Grâce à la présente invention, l'effet d'une composition pharmaceutique contenant un petit ARN en matière d'inhibition d'un gène cible peut être évalué en isolant un complexe protéine Ago-petit ARN à partir d'un échantillon prélevé chez un sujet auquel est administrée la composition pharmaceutique contenant un petit ARN, puis en mesurant la quantité de petit ARN contenue dans le complexe.
PCT/JP2015/072303 2014-08-06 2015-08-06 Procédé permettant de prédire l'efficacité médicamenteuse d'une composition pharmaceutique contenant un petit arn WO2016021673A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2014160487 2014-08-06
JP2014-160487 2014-08-06

Publications (1)

Publication Number Publication Date
WO2016021673A1 true WO2016021673A1 (fr) 2016-02-11

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/JP2015/072303 WO2016021673A1 (fr) 2014-08-06 2015-08-06 Procédé permettant de prédire l'efficacité médicamenteuse d'une composition pharmaceutique contenant un petit arn

Country Status (1)

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WO (1) WO2016021673A1 (fr)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020512272A (ja) * 2016-11-08 2020-04-23 ラモット アット テル アビブ ユニバーシティ， リミテッド 核酸送達用カチオン性脂質及びその調製物

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012174282A2 (fr) * 2011-06-16 2012-12-20 Caris Life Sciences Luxembourg Holdings, S.A.R.L. Compositions de biomarqueur et procédés associés

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012174282A2 (fr) * 2011-06-16 2012-12-20 Caris Life Sciences Luxembourg Holdings, S.A.R.L. Compositions de biomarqueur et procédés associés

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
WEI, J. ET AL.: "RNA-Induced Silencing Complex- Bound Small Interfering RNA Is a Determinant of RNA Interference-Mediated Gene Silencing in Mice", MOLECULAR PHARMACOLOGY, vol. 79, no. 6, 22 March 2011 (2011-03-22), pages 953 - 963, ISSN: 0026-895X *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020512272A (ja) * 2016-11-08 2020-04-23 ラモット アット テル アビブ ユニバーシティ， リミテッド 核酸送達用カチオン性脂質及びその調製物
JP7075669B2 (ja) 2016-11-08 2022-05-26 ラモット アット テル アビブ ユニバーシティ， リミテッド 核酸送達用カチオン性脂質及びその調製物
US11851389B2 (en) 2016-11-08 2023-12-26 Ramot At Tel-Aviv University Ltd. Cationic lipids for nucleic acid delivery and preparation thereof

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