AR116832A1 - Ácido ribonucleico bicatenario capaz de suprimir la expresión de c5 de complemento - Google Patents

Ácido ribonucleico bicatenario capaz de suprimir la expresión de c5 de complemento

Info

Publication number
AR116832A1
AR116832A1 ARP190103059A ARP190103059A AR116832A1 AR 116832 A1 AR116832 A1 AR 116832A1 AR P190103059 A ARP190103059 A AR P190103059A AR P190103059 A ARP190103059 A AR P190103059A AR 116832 A1 AR116832 A1 AR 116832A1
Authority
AR
Argentina
Prior art keywords
ribonucleic acid
bicatenary
complement
suppressing
expression
Prior art date
Application number
ARP190103059A
Other languages
English (en)
Inventor
Kazuhiro Tahara
Yoshinori Takahashi
Sotaro Motoi
Yuta Suzuki
Original Assignee
Eisai R&D Man Co Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Eisai R&D Man Co Ltd filed Critical Eisai R&D Man Co Ltd
Publication of AR116832A1 publication Critical patent/AR116832A1/es

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005—Wall or coating material
    • A61K9/5015—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/10—Drugs for disorders of the urinary system of the bladder
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Dispersion Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Cosmetics (AREA)
  • Anti-Oxidant Or Stabilizer Compositions (AREA)
  • Dc Digital Transmission (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)

Abstract

Se proporciona un ácido ribonucleico bicatenario que comprende una combinación de hebra codificante y hebra no codificante, en donde la combinación de la hebra codificante y la hebra no codificante se selecciona entre el grupo que consiste en las combinaciones: SEQ ID Nº 159 y 160, SEQ ID Nº 141 y 142, SEQ ID Nº 143 y 144, SEQ ID Nº 145 y 146, SEQ ID Nº 147 y 148 y SEQ ID Nº 153 y 154.
ARP190103059A 2018-10-26 2019-10-24 Ácido ribonucleico bicatenario capaz de suprimir la expresión de c5 de complemento AR116832A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP2018201777 2018-10-26

Publications (1)

Publication Number Publication Date
AR116832A1 true AR116832A1 (es) 2021-06-16

Family

ID=69058781

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP190103059A AR116832A1 (es) 2018-10-26 2019-10-24 Ácido ribonucleico bicatenario capaz de suprimir la expresión de c5 de complemento

Country Status (20)

Country Link
US (1) US10526603B1 (es)
EP (1) EP3871680B1 (es)
JP (1) JP6725776B1 (es)
KR (1) KR20210086608A (es)
CN (1) CN112771163B (es)
AR (1) AR116832A1 (es)
AU (1) AU2019367536A1 (es)
BR (1) BR112021006520A2 (es)
CA (1) CA3115249A1 (es)
CL (2) CL2021000826A1 (es)
CO (1) CO2021004029A2 (es)
IL (1) IL281902B (es)
JO (1) JOP20210066A1 (es)
MX (1) MX2021003910A (es)
PE (1) PE20210811A1 (es)
PH (1) PH12021550736A1 (es)
SG (1) SG11202102881PA (es)
TW (2) TWI818225B (es)
WO (1) WO2020085456A1 (es)
ZA (1) ZA202102104B (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6918197B2 (ja) * 2019-12-26 2021-08-11 エーザイ・アール・アンド・ディー・マネジメント株式会社 脂質複合体を含む医薬組成物及び脂質ナノ粒子を含む医薬組成物
BR112022010742A2 (pt) 2019-12-26 2022-08-16 Eisai R&D Man Co Ltd Composição farmacêutica contendo ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar
WO2021154941A1 (en) * 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
WO2023245126A2 (en) * 2022-06-15 2023-12-21 Sirnaomics, Inc. Products and compositions
WO2024169908A1 (zh) * 2023-02-17 2024-08-22 苏州时安生物技术有限公司 一种调节补体C5表达的siRNA、其缀合物和药物组合物及用途
WO2024255747A1 (zh) * 2023-06-16 2024-12-19 施能康医药科技(苏州)有限公司 靶向补体成分5的核酸及其用途

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL288931B2 (en) 2013-03-14 2025-05-01 Alnylam Pharmaceuticals Inc Compositions comprising a complementary portion of C5 IRNA and methods of using them
EP3093283B1 (en) 2014-01-09 2018-12-19 Eisai R&D Management Co., Ltd. Cationic lipid
WO2016044419A1 (en) * 2014-09-16 2016-03-24 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
US10081598B2 (en) 2014-12-26 2018-09-25 Eisai R&D Management Co., Ltd. Cationic lipid
US10036017B2 (en) 2015-02-17 2018-07-31 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of complement component 5(C5) by double-stranded RNA
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3469083A1 (en) * 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
WO2017222016A1 (ja) 2016-06-24 2017-12-28 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質

Also Published As

Publication number Publication date
CO2021004029A2 (es) 2021-04-30
PE20210811A1 (es) 2021-04-28
CL2023001965A1 (es) 2023-12-15
CN112771163A (zh) 2021-05-07
US10526603B1 (en) 2020-01-07
SG11202102881PA (en) 2021-04-29
WO2020085456A1 (ja) 2020-04-30
JPWO2020085456A1 (ja) 2021-02-15
TW202135833A (zh) 2021-10-01
MX2021003910A (es) 2021-06-04
IL281902B (en) 2022-09-01
EP3871680A4 (en) 2022-09-21
CN112771163B (zh) 2024-07-12
JOP20210066A1 (ar) 2021-04-06
ZA202102104B (en) 2022-08-31
KR20210086608A (ko) 2021-07-08
CL2021000826A1 (es) 2021-09-10
TW202031269A (zh) 2020-09-01
JP6725776B1 (ja) 2020-07-22
TWI718717B (zh) 2021-02-11
EP3871680B1 (en) 2024-03-20
AU2019367536A1 (en) 2021-04-29
PH12021550736A1 (en) 2021-10-25
CA3115249A1 (en) 2020-04-30
TWI818225B (zh) 2023-10-11
IL281902A (en) 2021-05-31
EP3871680A1 (en) 2021-09-01
BR112021006520A2 (pt) 2021-07-06

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