CN117425664A - Rna沉默剂及使用方法 - Google Patents

Rna沉默剂及使用方法 Download PDF

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Publication number
CN117425664A
CN117425664A CN202280034139.0A CN202280034139A CN117425664A CN 117425664 A CN117425664 A CN 117425664A CN 202280034139 A CN202280034139 A CN 202280034139A CN 117425664 A CN117425664 A CN 117425664A
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Prior art keywords
nucleic acid
nucleotide
antisense strand
target
sense strand
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CN202280034139.0A
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Chinese (zh)
Inventor
李珍
志清·乔尔·周
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Adalx Pharmaceutical Co ltd
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Adalx Pharmaceutical Co ltd
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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
    • A61K31/7105—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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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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]
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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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    • C12N2310/00—Structure or type of the nucleic acid
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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
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    • C12N2310/33—Chemical structure of the base
    • C12N2310/331—Universal or degenerate base
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    • C12N2310/33—Chemical structure of the base
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity

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  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CN202280034139.0A 2021-04-13 2022-04-13 Rna沉默剂及使用方法 Pending CN117425664A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163174507P 2021-04-13 2021-04-13
US63/174,507 2021-04-13
PCT/US2022/024692 WO2022221457A1 (en) 2021-04-13 2022-04-13 Rna silencing agents and methods of use

Publications (1)

Publication Number Publication Date
CN117425664A true CN117425664A (zh) 2024-01-19

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CN202280034139.0A Pending CN117425664A (zh) 2021-04-13 2022-04-13 Rna沉默剂及使用方法

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US (1) US20240191229A1 (enExample)
EP (1) EP4323376A4 (enExample)
JP (1) JP2024513972A (enExample)
CN (1) CN117425664A (enExample)
AU (1) AU2022257002A1 (enExample)
CA (1) CA3216732A1 (enExample)
WO (1) WO2022221457A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
US20240401045A1 (en) * 2021-10-01 2024-12-05 Adarx Pharmaceuticals, Inc. Angiotensinogen-modulating compositions and methods of use thereof

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090209626A1 (en) * 2008-02-11 2009-08-20 Dharmacon, Inc. Duplex Oligonucleotides with Enhanced Functionality in Gene Regulation
US20100173974A1 (en) * 2008-12-18 2010-07-08 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
US20130171242A1 (en) * 2010-08-24 2013-07-04 Lee Lim Single-Stranded RNAi Agents Containing an Internal, Non-Nucleic Acid Spacer
WO2016011123A1 (en) * 2014-07-16 2016-01-21 Arrowhead Research Corporation Organic compositions to treat apoc3-related diseases
US20190040383A1 (en) * 2014-12-17 2019-02-07 Proqr Therapeutics Ii B.V. Targeted rna editing

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2264169A1 (en) * 2009-06-15 2010-12-22 Qiagen GmbH Modified siNA
KR101696704B1 (ko) * 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090209626A1 (en) * 2008-02-11 2009-08-20 Dharmacon, Inc. Duplex Oligonucleotides with Enhanced Functionality in Gene Regulation
US20100173974A1 (en) * 2008-12-18 2010-07-08 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
WO2010080129A2 (en) * 2008-12-18 2010-07-15 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
US20130171242A1 (en) * 2010-08-24 2013-07-04 Lee Lim Single-Stranded RNAi Agents Containing an Internal, Non-Nucleic Acid Spacer
WO2016011123A1 (en) * 2014-07-16 2016-01-21 Arrowhead Research Corporation Organic compositions to treat apoc3-related diseases
US20170137814A1 (en) * 2014-07-16 2017-05-18 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat apoc3-related diseases
US20190040383A1 (en) * 2014-12-17 2019-02-07 Proqr Therapeutics Ii B.V. Targeted rna editing

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US20240191229A1 (en) 2024-06-13
CA3216732A1 (en) 2022-10-20
EP4323376A4 (en) 2025-07-09
WO2022221457A1 (en) 2022-10-20
JP2024513972A (ja) 2024-03-27
AU2022257002A1 (en) 2023-10-26
EP4323376A1 (en) 2024-02-21

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