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SG11202101698WA - Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use - Google Patents

Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use

Info

Publication number
SG11202101698WA
SG11202101698WA SG11202101698WA SG11202101698WA SG11202101698WA SG 11202101698W A SG11202101698W A SG 11202101698WA SG 11202101698W A SG11202101698W A SG 11202101698WA SG 11202101698W A SG11202101698W A SG 11202101698WA SG 11202101698W A SG11202101698W A SG 11202101698WA
Authority
SG
Singapore
Prior art keywords
hsd17b13
17beta
compositions
methods
rnai agents
Prior art date
Application number
SG11202101698WA
Inventor
Zhen Li
Rui Zhu
Shawn A Morales
Original Assignee
Arrowhead Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of SG11202101698WA publication Critical patent/SG11202101698WA/en

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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
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    • C12Y—ENZYMES
    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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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
    • C12N2310/31—Chemical structure of the backbone
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    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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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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    • C12N2310/33—Chemical structure of the base
    • C12N2310/331—Universal or degenerate 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
    • C12N2310/343—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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Biotechnology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
SG11202101698WA 2018-09-19 2019-09-18 Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use SG11202101698WA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862733320P 2018-09-19 2018-09-19
US201862773707P 2018-11-30 2018-11-30
US201962890220P 2019-08-22 2019-08-22
PCT/US2019/051707 WO2020061177A1 (en) 2018-09-19 2019-09-18 Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use

Publications (1)

Publication Number Publication Date
SG11202101698WA true SG11202101698WA (en) 2021-04-29

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SG11202101698WA SG11202101698WA (en) 2018-09-19 2019-09-18 Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use

Country Status (21)

Country Link
US (1) US20220056454A1 (en)
EP (1) EP3852769A4 (en)
JP (2) JP7488254B2 (en)
KR (1) KR102769565B1 (en)
CN (1) CN113164509A (en)
AU (1) AU2019342117A1 (en)
BR (1) BR112021005137A2 (en)
CA (1) CA3109553A1 (en)
CL (1) CL2021000669A1 (en)
CO (1) CO2021003446A2 (en)
CR (1) CR20210186A (en)
EC (1) ECSP21027262A (en)
IL (1) IL281597A (en)
MX (1) MX2021003077A (en)
MY (1) MY209138A (en)
PE (1) PE20211212A1 (en)
PH (1) PH12021550423A1 (en)
SG (1) SG11202101698WA (en)
TW (1) TWI870358B (en)
UY (1) UY38382A (en)
WO (1) WO2020061177A1 (en)

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CA3091146A1 (en) 2018-03-21 2019-09-26 Regeneron Pharmaceuticals, Inc. 17.beta.-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
KR102769565B1 (en) * 2018-09-19 2025-02-20 애로우헤드 파마슈티컬스 인코포레이티드 RNAi agent for inhibiting expression of 17beta-HSD type 13- (HSD17B13), composition thereof, and method of use
JOP20210158A1 (en) 2018-12-21 2023-01-30 Ionis Pharmaceuticals Inc Modulators of hsd17b13 expression
US20230279399A1 (en) * 2020-06-01 2023-09-07 Amgen Inc. Rnai constructs for inhibiting hsd17b13 expression and methods of use thereof
WO2022098748A1 (en) * 2020-11-06 2022-05-12 Inipharm, Inc. Uses for hsd17b13 inhibitors
WO2022140624A1 (en) 2020-12-23 2022-06-30 Regeneron Pharmaceuticals, Inc. Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors
EP4328310A4 (en) * 2021-04-22 2025-10-15 Tuojie Biotech Shanghai Co Ltd 17B-HYDROXYSTEROID DEHYDROGENASE TYPE 13 TARGETTING SIRNA AND SIRNA CONJUGATE
JP2024534799A (en) 2021-08-20 2024-09-26 エナンタ ファーマシューティカルズ インコーポレイテッド 17-beta-hydroxysteroid dehydrogenase type 13 inhibitors and methods of use thereof
EP4399301A1 (en) * 2021-09-08 2024-07-17 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
WO2023091985A1 (en) * 2021-11-18 2023-05-25 Dtx Pharma, Inc. Compounds targeting pmp22 for the treatment of charcot-marie-tooth disease
CN118355121A (en) * 2021-12-16 2024-07-16 上海拓界生物医药科技有限公司 DsRNA, preparation method and application thereof
EP4453257A2 (en) 2021-12-20 2024-10-30 Regeneron Pharmaceuticals, Inc. Methods of identifying and evaluating liver inflammation and liver fibrosis in a subject by determining a stratified score based on gene expression
WO2023138650A1 (en) * 2022-01-20 2023-07-27 上海拓界生物医药科技有限公司 Dsrna, use thereof and preparation method therefor
EP4504337A1 (en) 2022-04-08 2025-02-12 Arrowhead Pharmaceuticals, Inc. Treatment of a non-alcoholic fatty liver disease
EP4514805A1 (en) * 2022-04-28 2025-03-05 Enanta Pharmaceuticals, Inc. Pyrimidinone-containing 17-beta-hydroxysteroid dehydrogenase type 13 inhibitors
WO2023208109A1 (en) * 2022-04-29 2023-11-02 北京福元医药股份有限公司 Sirna for inhibiting hsd17b13 expression, conjugate and pharmaceutical composition thereof, and use thereof
WO2023213284A1 (en) * 2022-05-06 2023-11-09 苏州瑞博生物技术股份有限公司 Nucleic acid, composition and conjugate comprising same, preparation method therefor, and use thereof
TW202440924A (en) * 2022-12-22 2024-10-16 大陸商上海舶望製藥有限公司 Compositions and methods for inhibiting the expression of 17β-hydroxysteroid dehydrogenase type 13 (HSD17B13)
WO2024255748A1 (en) * 2023-06-16 2024-12-19 施能康医药科技(苏州)有限公司 NUCLEIC ACID TARGETING HYDROXYSTEROID 17-β DEHYDROGENASE 13 AND USE THEREOF
US20250075214A1 (en) * 2023-08-30 2025-03-06 Arrowhead Pharmaceuticals, Inc. RNAi Agents for Inhibiting Expression of Inhibin Subunit Beta E (INHBE), Pharmaceutical Compositions Thereof, and Methods of Use
WO2025076225A1 (en) * 2023-10-04 2025-04-10 Enanta Pharmaceuticals, Inc. 17-beta-hydroxysteroid dehydrogenase type 13 inhibitors and methods of use thereof
WO2025074157A1 (en) 2023-10-06 2025-04-10 Glaxosmithkline Intellectual Property (No.3) Limited Treatment of a non-alcoholic fatty liver disease

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KR102769565B1 (en) * 2018-09-19 2025-02-20 애로우헤드 파마슈티컬스 인코포레이티드 RNAi agent for inhibiting expression of 17beta-HSD type 13- (HSD17B13), composition thereof, and method of use
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AU2019342117A1 (en) 2021-03-04
BR112021005137A2 (en) 2021-06-15
US20220056454A1 (en) 2022-02-24
JP2022501040A (en) 2022-01-06
WO2020061177A1 (en) 2020-03-26
CO2021003446A2 (en) 2021-04-08
KR102769565B1 (en) 2025-02-20
CL2021000669A1 (en) 2021-10-29
JP7741927B2 (en) 2025-09-18
EP3852769A4 (en) 2023-12-13
JP2024112838A (en) 2024-08-21
PH12021550423A1 (en) 2021-09-20
EP3852769A1 (en) 2021-07-28
KR20210061380A (en) 2021-05-27
AU2019342117A2 (en) 2021-03-11
IL281597A (en) 2021-05-31
CN113164509A (en) 2021-07-23
TWI870358B (en) 2025-01-21
TW202024324A (en) 2020-07-01
CR20210186A (en) 2021-06-14
JP7488254B2 (en) 2024-05-21
MY209138A (en) 2025-06-24
ECSP21027262A (en) 2021-05-31
UY38382A (en) 2020-03-31
MX2021003077A (en) 2021-05-27
CA3109553A1 (en) 2020-03-26
PE20211212A1 (en) 2021-07-05

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