JP7453921B2 - Ｇｙｓ２発現を阻害するための組成物及び方法 - Google Patents

Ｇｙｓ２発現を阻害するための組成物及び方法 Download PDF

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JP7453921B2
JP7453921B2 JP2020568663A JP2020568663A JP7453921B2 JP 7453921 B2 JP7453921 B2 JP 7453921B2 JP 2020568663 A JP2020568663 A JP 2020568663A JP 2020568663 A JP2020568663 A JP 2020568663A JP 7453921 B2 JP7453921 B2 JP 7453921B2
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oligonucleotide
nucleotides
gys2
oligonucleotides
nucleotide
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JPWO2019168687A5 (https=
JP2021517826A5 (https=
JP2021517826A (ja
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ボブ ディー ブラウン
ナタリー パーセル
ヘンリク ティー ドュデク
チェン ライ
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Dicerna Pharmaceuticals Inc
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    • 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
    • C12N15/113—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
    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01001—Phosphorylase (2.4.1.1)
    • 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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/50—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
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01011—Glycogen(starch) synthase (2.4.1.11)

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Microbiology (AREA)
  • Biophysics (AREA)
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  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
JP2020568663A 2018-03-02 2019-02-15 Ｇｙｓ２発現を阻害するための組成物及び方法 Active JP7453921B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862637574P 2018-03-02 2018-03-02
US62/637,574 2018-03-02
PCT/US2019/018189 WO2019168687A1 (en) 2018-03-02 2019-02-15 Compositions and methods for inhibiting gys2 expression

Publications (4)

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JP2021517826A JP2021517826A (ja) 2021-07-29
JPWO2019168687A5 JPWO2019168687A5 (https=) 2022-02-24
JP2021517826A5 JP2021517826A5 (https=) 2022-02-24
JP7453921B2 true JP7453921B2 (ja) 2024-03-21

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US (2) US11572562B2 (https=)
EP (1) EP3740247A4 (https=)
JP (1) JP7453921B2 (https=)
KR (1) KR102804353B1 (https=)
CN (1) CN112055598B (https=)
AU (1) AU2019227510B2 (https=)
CA (1) CA3092092A1 (https=)
IL (2) IL325191A (https=)
MX (1) MX2020009072A (https=)
WO (1) WO2019168687A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019227510B2 (en) 2018-03-02 2026-02-12 Novo Nordisk A/S Compositions and methods for inhibiting GYS2 expression
WO2020226960A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
WO2022223515A2 (en) * 2021-04-19 2022-10-27 Novo Nordisk A/S Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
AU2024269205A1 (en) * 2023-05-11 2025-12-11 Keymed Biosciences (Us) Inc. Dsrna molecules for regulating masp2 gene activity

Citations (4)

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WO2001075067A2 (en) 2000-03-31 2001-10-11 Hyseq, Inc. Novel nucleic acids and polypeptides
JP2012504389A (ja) 2008-09-22 2012-02-23 ダイセルナ ファーマシューティカルズ， インコーポレイテッド 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法
JP2014530602A (ja) 2011-10-05 2014-11-20 プロティバ バイオセラピューティクス インコーポレイテッド アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法
WO2016183009A2 (en) 2015-05-08 2016-11-17 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of antithrombin 3 (at3) by double-stranded rna

Family Cites Families (13)

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US20030004122A1 (en) * 1997-11-05 2003-01-02 Leonid Beigelman Nucleotide triphosphates and their incorporation into oligonucleotides
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20050119210A1 (en) * 2003-05-20 2005-06-02 Xiaobing Be Compositions and methods for diagnosing and treating cancers
US20060078902A1 (en) * 2004-04-15 2006-04-13 Michaeline Bunting Method and compositions for RNA interference
EP1752536A4 (en) * 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
US8278287B2 (en) * 2008-04-15 2012-10-02 Quark Pharmaceuticals Inc. siRNA compounds for inhibiting NRF2
AU2009336191B2 (en) * 2008-12-18 2017-08-24 Novo Nordisk A/S Extended dicer substrate agents and methods for the specific inhibition of gene expression
WO2010105372A1 (en) 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
AU2015330670B2 (en) * 2014-10-10 2022-01-06 Novo Nordisk Health Care Ag Therapeutic inhibition of lactate dehydrogenase and agents therefor
JP7105065B2 (ja) * 2014-12-15 2022-07-22 ダイセルナ ファーマシューティカルズ， インコーポレイテッド リガンド修飾二本鎖核酸
WO2017141109A1 (en) * 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
CN111448316B (zh) * 2017-12-22 2025-02-14 罗氏创新中心哥本哈根有限公司 新的硫代亚磷酰胺
AU2019227510B2 (en) 2018-03-02 2026-02-12 Novo Nordisk A/S Compositions and methods for inhibiting GYS2 expression

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001075067A2 (en) 2000-03-31 2001-10-11 Hyseq, Inc. Novel nucleic acids and polypeptides
JP2012504389A (ja) 2008-09-22 2012-02-23 ダイセルナ ファーマシューティカルズ， インコーポレイテッド 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法
JP2014530602A (ja) 2011-10-05 2014-11-20 プロティバ バイオセラピューティクス インコーポレイテッド アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法
WO2016183009A2 (en) 2015-05-08 2016-11-17 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of antithrombin 3 (at3) by double-stranded rna

Non-Patent Citations (1)

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Title
Nature Communications，2017年06月19日，Vol.8，15868

Also Published As

Publication number Publication date
US12534730B2 (en) 2026-01-27
EP3740247A4 (en) 2022-06-08
KR102804353B1 (ko) 2025-05-13
CA3092092A1 (en) 2019-09-06
US11572562B2 (en) 2023-02-07
KR20200127211A (ko) 2020-11-10
MX2020009072A (es) 2020-10-08
CN112055598B (zh) 2026-04-14
CN112055598A (zh) 2020-12-08
IL277058A (en) 2020-10-29
EP3740247A1 (en) 2020-11-25
IL325191A (en) 2026-02-01
JP2021517826A (ja) 2021-07-29
US20210062197A1 (en) 2021-03-04
AU2019227510B2 (en) 2026-02-12
US20230365974A1 (en) 2023-11-16
WO2019168687A1 (en) 2019-09-06

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