MX2021006745A - Constructos de iarn modificados quimicamente y usos de estos. - Google Patents

Constructos de iarn modificados quimicamente y usos de estos.

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Publication number
MX2021006745A
MX2021006745A MX2021006745A MX2021006745A MX2021006745A MX 2021006745 A MX2021006745 A MX 2021006745A MX 2021006745 A MX2021006745 A MX 2021006745A MX 2021006745 A MX2021006745 A MX 2021006745A MX 2021006745 A MX2021006745 A MX 2021006745A
Authority
MX
Mexico
Prior art keywords
chemically
rnai constructs
modified
modified rnai
vivo
Prior art date
Application number
MX2021006745A
Other languages
English (en)
Inventor
Justin K Murray
Bradley J Herberich
Bin Wu
Yuan Cheng
Original Assignee
Amgen 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 Amgen Inc filed Critical Amgen Inc
Publication of MX2021006745A publication Critical patent/MX2021006745A/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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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 N.A.
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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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
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/34—Spatial arrangement of the modifications
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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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
    • C12N2310/3523—Allyl
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

Abstract

La presente invención se refiere a constructos de iARN modificados químicamente para reducir la expresión de un gen diana. En particular, la invención se refiere a patrones específicos de nucleótidos modificados que se incorporarán a los constructos de iARN para mejorar su estabilidad y eficacia in vivo. También se describen composiciones farmacéuticas que comprenden los constructos de iARN modificados químicamente y métodos para inhibir la expresión del gen diana in vivo administrando los constructos de iARN modificados químicamente, por ejemplo, para tratar o mejorar diversas afecciones patológicas.
MX2021006745A 2018-12-10 2019-12-09 Constructos de iarn modificados quimicamente y usos de estos. MX2021006745A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862777677P 2018-12-10 2018-12-10
PCT/US2019/065294 WO2020123410A1 (en) 2018-12-10 2019-12-09 CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF

Publications (1)

Publication Number Publication Date
MX2021006745A true MX2021006745A (es) 2021-07-15

Family

ID=69024730

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021006745A MX2021006745A (es) 2018-12-10 2019-12-09 Constructos de iarn modificados quimicamente y usos de estos.

Country Status (14)

Country Link
US (1) US20220049252A1 (es)
EP (1) EP3894554A1 (es)
JP (1) JP2022511866A (es)
KR (1) KR20210102313A (es)
CN (1) CN113166759A (es)
AU (1) AU2019395341A1 (es)
BR (1) BR112021011043A2 (es)
CA (1) CA3122393A1 (es)
CL (1) CL2021001490A1 (es)
EA (1) EA202191630A1 (es)
IL (1) IL283550A (es)
MX (1) MX2021006745A (es)
SG (1) SG11202105900UA (es)
WO (1) WO2020123410A1 (es)

Families Citing this family (9)

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WO2022036126A2 (en) 2020-08-13 2022-02-17 Amgen Inc. RNAi CONSTRUCTS AND METHODS FOR INHIBITING MARC1 EXPRESSION
CN116456990A (zh) * 2020-11-05 2023-07-18 美国安进公司 用靶向LPA的RNAi构建体治疗动脉粥样硬化性心血管疾病的方法
CA3225054A1 (en) * 2021-07-08 2023-02-12 Sun Woo Hong Rnai agent targeting marc1 gene, and use thereof
CN114703184A (zh) * 2022-03-11 2022-07-05 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
WO2023207615A1 (zh) * 2022-04-26 2023-11-02 南京明德新药研发有限公司 一类含由天然核苷酸组成突出端的双链RNAi化合物
US20240084301A1 (en) 2022-07-25 2024-03-14 Amgen Inc. Rnai constructs and methods for inhibiting fam13a expression
WO2024023256A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
WO2024023254A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
WO2024061185A1 (zh) * 2022-09-20 2024-03-28 上海舶望制药有限公司 特定修饰的RNAi试剂和组合物

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Also Published As

Publication number Publication date
IL283550A (en) 2021-07-29
SG11202105900UA (en) 2021-07-29
CL2021001490A1 (es) 2022-01-14
AU2019395341A1 (en) 2021-06-17
EP3894554A1 (en) 2021-10-20
EA202191630A1 (ru) 2021-11-29
BR112021011043A2 (pt) 2021-08-31
US20220049252A1 (en) 2022-02-17
CA3122393A1 (en) 2020-06-18
KR20210102313A (ko) 2021-08-19
CN113166759A (zh) 2021-07-23
WO2020123410A1 (en) 2020-06-18
JP2022511866A (ja) 2022-02-01

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