AU2021382621A1 - Oligonucleotides for dgat2 modulation - Google Patents

Oligonucleotides for dgat2 modulation Download PDF

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Publication number
AU2021382621A1
AU2021382621A1 AU2021382621A AU2021382621A AU2021382621A1 AU 2021382621 A1 AU2021382621 A1 AU 2021382621A1 AU 2021382621 A AU2021382621 A AU 2021382621A AU 2021382621 A AU2021382621 A AU 2021382621A AU 2021382621 A1 AU2021382621 A1 AU 2021382621A1
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Australia
Prior art keywords
dsrna
antisense strand
nucleotides
strand
sense strand
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Pending
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AU2021382621A
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AU2021382621A9 (en
Inventor
Michael P. Czech
Anastasia Khvorova
Batuhan Orbay YENILMEZ
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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Publication of AU2021382621A1 publication Critical patent/AU2021382621A1/en
Publication of AU2021382621A9 publication Critical patent/AU2021382621A9/en
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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
    • C12N15/113—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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2021382621A 2020-11-23 2021-11-22 Oligonucleotides for dgat2 modulation Pending AU2021382621A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063117005P 2020-11-23 2020-11-23
US63/117,005 2020-11-23
PCT/US2021/060356 WO2022109398A1 (en) 2020-11-23 2021-11-22 Oligonucleotides for dgat2 modulation

Publications (2)

Publication Number Publication Date
AU2021382621A1 true AU2021382621A1 (en) 2023-06-22
AU2021382621A9 AU2021382621A9 (en) 2024-02-08

Family

ID=81709762

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021382621A Pending AU2021382621A1 (en) 2020-11-23 2021-11-22 Oligonucleotides for dgat2 modulation

Country Status (5)

Country Link
US (1) US20220228141A1 (https=)
EP (1) EP4247391A4 (https=)
JP (1) JP2023550485A (https=)
AU (1) AU2021382621A1 (https=)
WO (1) WO2022109398A1 (https=)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
CN116600817A (zh) * 2020-07-16 2023-08-15 马萨诸塞大学 用于组织特异性递送的缀合寡核苷酸
AU2022299169A1 (en) 2021-06-23 2024-02-08 Beth Israel Deaconess Medical Center, Inc. Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
WO2024228030A2 (en) * 2023-05-04 2024-11-07 Argonaute RNA Limited Dual silencing
EP4705458A2 (en) * 2023-05-04 2026-03-11 Argonaute RNA Limited Dual silencing
EP4712982A1 (en) * 2023-05-17 2026-03-25 University of Massachusetts Oligonucleotides for dgat2, fasn, opn, and nox4 modulations
WO2024245151A1 (zh) * 2023-05-26 2024-12-05 维亚臻生物技术（苏州）有限公司 一种用于代谢疾病的双链核苷酸化合物及其应用
AU2024321098A1 (en) * 2023-08-09 2026-03-19 Beijing Foyou Pharma Co., Ltd Sirna for inhibiting dgat2 gene expression, sirna conjugate or prodrug and pharmaceutical composition thereof, and use thereof
WO2025040139A1 (zh) * 2023-08-23 2025-02-27 云合智药（苏州）生物科技有限公司 用于抑制二酰甘油-O-酰基转移酶2表达的RNAi剂及其应用
CN117257764A (zh) * 2023-09-26 2023-12-22 中国药科大学 一种脂滴衍生的核酸药物递送系统及其制备方法和应用
CN121825964A (zh) * 2023-12-26 2026-04-10 苏州吉玛基因股份有限公司 用于抑制DGAT2的siRNA及其修饰物与应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1455815A4 (en) * 2001-12-19 2006-11-02 Millennium Pharm Inc MEMBERS OF THE DIACYLGLYCEROL-ACYLTRANSFERASES FAMILY 2 (DGAT2) AND USES THEREOF
WO2006006948A2 (en) * 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US20190270995A1 (en) * 2002-11-14 2019-09-05 Thermo Fisher Scientific Inc. Methods and Compositions for Selecting siRNA of Improved Functionality
US7825235B2 (en) * 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
KR102486617B1 (ko) * 2013-05-22 2023-01-12 알닐람 파마슈티칼스 인코포레이티드 Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법
PT3277815T (pt) * 2015-04-03 2021-11-11 Beth Israel Deaconess Medical Ct Inc Compostos de oligonucleótidos para o tratamento de pré-eclâmpsia e outros distúrbios angiogénicos
CA2991894A1 (en) * 2015-07-10 2017-01-19 Ionis Pharmaceuticals, Inc. Modulators of diacyglycerol acyltransferase 2 (dgat2)
CN115175685B (zh) * 2019-12-09 2024-12-13 艾姆皮瑞克公司 用于治疗血管生成素样4(angptl4)相关疾病的寡核苷酸
TW202221120A (zh) * 2020-08-04 2022-06-01 美商黛瑟納製藥公司 用於治療代謝症候群之組成物及方法

Also Published As

Publication number Publication date
WO2022109398A9 (en) 2022-09-29
EP4247391A4 (en) 2025-11-19
WO2022109398A1 (en) 2022-05-27
JP2023550485A (ja) 2023-12-01
AU2021382621A9 (en) 2024-02-08
EP4247391A1 (en) 2023-09-27
US20220228141A1 (en) 2022-07-21

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