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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JP2021517826A (ja
JP2021517826A5 (enExample
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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
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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)
    • C12Y204/01001—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
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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
    • 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
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/533—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
    • 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)
    • C12Y204/01011—Glycogen(starch) synthase (2.4.1.11)

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  • Health & Medical Sciences (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (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)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (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 (3)

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

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020009072A (es) 2018-03-02 2020-10-08 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de gys2.
WO2020226960A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
IL307154A (en) * 2021-04-19 2023-11-01 Novo Nordisk As Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
JP2026520116A (ja) * 2023-05-11 2026-06-22 大睿生物 ＭＡＳＰ２遺伝子活性を制御するためのｄｓＲＮＡ分子
WO2026162643A1 (en) * 2025-01-31 2026-08-06 Novo Nordisk A/S Nucleic acid constructs for inhibiting gys2 expression

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

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US20030004122A1 (en) * 1997-11-05 2003-01-02 Leonid Beigelman Nucleotide triphosphates and their incorporation into oligonucleotides
EP2305813A3 (en) * 2002-11-14 2012-03-28 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
CA2566286A1 (en) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
WO2009144704A2 (en) * 2008-04-15 2009-12-03 Quark Pharmaceuticals, Inc. siRNA COMPOUNDS FOR INHIBITING NRF2
KR20110110776A (ko) * 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
US10351854B2 (en) * 2014-10-10 2019-07-16 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
EP3865576A1 (en) * 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
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
MX2020009072A (es) 2018-03-02 2020-10-08 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de gys2.

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

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