MX2022011012A - Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas. - Google Patents

Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas.

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Publication number
MX2022011012A
MX2022011012A MX2022011012A MX2022011012A MX2022011012A MX 2022011012 A MX2022011012 A MX 2022011012A MX 2022011012 A MX2022011012 A MX 2022011012A MX 2022011012 A MX2022011012 A MX 2022011012A MX 2022011012 A MX2022011012 A MX 2022011012A
Authority
MX
Mexico
Prior art keywords
sina
molecules
nucleic acid
short interfering
interfering nucleic
Prior art date
Application number
MX2022011012A
Other languages
English (en)
Inventor
Markus Hossbach
Leonid Beigelman
Jin Hong
Laxman Eltepu
Vivek Kumar Rajwanshi
Rajendra K Pandey
Saul Martinez Montero
Costa N Tilani S De
Original Assignee
Aligos Therapeutics Inc
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 Aligos Therapeutics Inc filed Critical Aligos Therapeutics Inc
Publication of MX2022011012A publication Critical patent/MX2022011012A/es

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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
    • 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
    • C12N15/1131—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 against viruses
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • 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.
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2320/31—Combination therapy

Abstract

En la presente se describen moléculas de ácido nucleico interferente corto (siNA) que comprenden nucleótidos modificados y usos de estas. Las moléculas de siNA pueden ser de cadena doble y comprender nucleótidos modificados que se seleccionan de nucleótidos 2'-O-metilo y nucleótidos 2'-fluoro. En la presente se describen además moléculas de siNA que comprenden (a) un bloqueador de fosforilación, un resto conjugado o un casquete del extremo 5' estabilizado; y (b) un ácido nucleico interferente corto (siNA).
MX2022011012A 2020-03-06 2021-03-05 Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas. MX2022011012A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062986150P 2020-03-06 2020-03-06
US202063109196P 2020-11-03 2020-11-03
PCT/US2021/021199 WO2021178885A1 (en) 2020-03-06 2021-03-05 Modified short interfering nucleic acid (sina) molecules and uses thereof

Publications (1)

Publication Number Publication Date
MX2022011012A true MX2022011012A (es) 2022-10-07

Family

ID=75278356

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022011012A MX2022011012A (es) 2020-03-06 2021-03-05 Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas.

Country Status (15)

Country Link
US (4) US20220364096A1 (es)
EP (1) EP4121533A1 (es)
JP (1) JP2023515664A (es)
KR (1) KR20220163960A (es)
CN (1) CN115485384A (es)
AU (1) AU2021230601A1 (es)
BR (1) BR112022017287A2 (es)
CA (1) CA3170466A1 (es)
CL (1) CL2022002379A1 (es)
CO (1) CO2022014258A2 (es)
IL (1) IL296045A (es)
MX (1) MX2022011012A (es)
PE (1) PE20221578A1 (es)
TW (1) TW202208625A (es)
WO (1) WO2021178885A1 (es)

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WO2019053661A2 (en) * 2017-09-14 2019-03-21 Alios Biopharma, Inc. GALNAC DERIVATIVES
CN115485384A (zh) * 2020-03-06 2022-12-16 阿利戈斯治疗公司 经修饰的短干扰核酸(siNA)分子和其用途
CN116724038A (zh) 2020-10-21 2023-09-08 安力高医药股份有限公司 双环化合物
WO2022184852A1 (en) * 2021-03-03 2022-09-09 Silence Therapeutics Gmbh Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell
WO2022266193A1 (en) 2021-06-18 2022-12-22 Aligos Therapeutics, Inc. Bicyclic compounds
WO2023122317A2 (en) * 2021-12-23 2023-06-29 Aligos Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (SINA) MOLECULES FOR TARGETING β-CATENIN AND USES THEREOF
US20230399645A1 (en) * 2022-05-16 2023-12-14 University Of Massachusetts Optimized sirna scaffolds
WO2024033374A1 (en) 2022-08-11 2024-02-15 Syngenta Crop Protection Ag Novel arylcarboxamide or arylthioamide compounds
WO2024089216A1 (en) 2022-10-27 2024-05-02 Syngenta Crop Protection Ag Novel sulfur-containing heteroaryl carboxamide compounds
CN117534717A (zh) * 2024-01-09 2024-02-09 凯莱英生命科学技术(天津)有限公司 5′-(e)-乙烯基磷酸酯的合成方法

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CN103154014B (zh) 2010-04-28 2015-03-25 Isis制药公司 修饰核苷、其类似物以及由它们制备的寡聚化合物
CN113430196A (zh) * 2011-06-30 2021-09-24 箭头药业股份有限公司 用于抑制乙型肝炎病毒的基因表达的组合物和方法
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Publication number Publication date
TW202208625A (zh) 2022-03-01
AU2021230601A2 (en) 2022-10-20
EP4121533A1 (en) 2023-01-25
WO2021178885A1 (en) 2021-09-10
US20220364096A1 (en) 2022-11-17
CO2022014258A2 (es) 2022-12-30
BR112022017287A2 (pt) 2022-11-16
AU2021230601A1 (en) 2022-10-06
JP2023515664A (ja) 2023-04-13
US20220177888A1 (en) 2022-06-09
CA3170466A1 (en) 2021-09-10
IL296045A (en) 2022-10-01
US20230332153A1 (en) 2023-10-19
US20230365970A1 (en) 2023-11-16
KR20220163960A (ko) 2022-12-12
US11549110B2 (en) 2023-01-10
CN115485384A (zh) 2022-12-16
PE20221578A1 (es) 2022-10-06
CL2022002379A1 (es) 2023-07-28

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