CN114761557B - 具有最小氟含量的小干扰rna的化学修饰 - Google Patents

具有最小氟含量的小干扰rna的化学修饰 Download PDF

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CN114761557B
CN114761557B CN202080082980.8A CN202080082980A CN114761557B CN 114761557 B CN114761557 B CN 114761557B CN 202080082980 A CN202080082980 A CN 202080082980A CN 114761557 B CN114761557 B CN 114761557B
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modified
antisense strand
nucleotides
oligonucleotide
positions
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CN114761557A (zh
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W·王
N·纳泽夫
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Novo Nordisk AS
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Dicerna Pharmaceuticals Inc
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    • 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
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    • 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
    • C12N15/1137—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 enzymes
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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
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    • 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

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
CN202080082980.8A 2019-10-02 2020-10-02 具有最小氟含量的小干扰rna的化学修饰 Active CN114761557B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962909278P 2019-10-02 2019-10-02
US62/909278 2019-10-02
PCT/US2020/053999 WO2021067744A1 (en) 2019-10-02 2020-10-02 Chemical modifications of small interfering rna with minimal fluorine content

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CN114761557A CN114761557A (zh) 2022-07-15
CN114761557B true CN114761557B (zh) 2024-11-15

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US (1) US20220389430A1 (https=)
EP (1) EP4038191A1 (https=)
JP (1) JP2022551269A (https=)
KR (1) KR20220069103A (https=)
CN (1) CN114761557B (https=)
AU (1) AU2020358016A1 (https=)
CA (1) CA3153026A1 (https=)
CL (1) CL2022000825A1 (https=)
IL (1) IL291841A (https=)
MX (1) MX2022004032A (https=)
TW (1) TW202126809A (https=)
WO (1) WO2021067744A1 (https=)

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PE20250400A1 (es) 2021-04-12 2025-02-11 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir cetohexoquinasa
CA3209418A1 (en) * 2021-04-14 2022-10-20 Utsav SAXENA Compositions and methods for modulating pnpla3 expression
EP4372085A4 (en) * 2021-07-16 2026-01-21 Suzhou Ribo Life Science Co Ltd Nucleic acid, composition and conjugate, container, preparation process and use
EP4405368A1 (en) * 2021-09-22 2024-07-31 Sanegene Bio USA Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
WO2023069495A1 (en) * 2021-10-19 2023-04-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides with 2'-deoxy-2'-f-2'-c-methyl nucleotides
JP2024543195A (ja) 2021-12-01 2024-11-19 ディセルナ ファーマシューティカルズ インコーポレイテッド Ａｐｏｃ３発現を調節するための組成物及び方法
CN119183476A (zh) * 2022-04-26 2024-12-24 上海拓界生物医药科技有限公司 氘代化学修饰和包含其的寡核苷酸
TW202526017A (zh) * 2023-09-14 2025-07-01 美商Ionis製藥公司 用於減少ａｐｏｃｉｉｉ表現的化合物及方法
WO2025191337A2 (en) * 2024-03-11 2025-09-18 Synerk Biotech Limited Oligonucleotide terminal phosphate derivatives and method of use

Citations (1)

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WO2019079781A2 (en) * 2017-10-20 2019-04-25 Dicerna Pharmaceuticals, Inc METHODS OF TREATING HEPATITIS B TYPE INFECTIONS

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CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
DK2341943T3 (en) 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
US8691971B2 (en) 2008-09-23 2014-04-08 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
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WO2021067744A1 (en) 2021-04-08
CA3153026A1 (en) 2021-04-08
AU2020358016A1 (en) 2022-04-21
CL2022000825A1 (es) 2023-01-27
CN114761557A (zh) 2022-07-15
JP2022551269A (ja) 2022-12-08
US20220389430A1 (en) 2022-12-08
KR20220069103A (ko) 2022-05-26
EP4038191A1 (en) 2022-08-10
MX2022004032A (es) 2022-09-21
TW202126809A (zh) 2021-07-16
IL291841A (en) 2022-06-01

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