IL303536A - Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors - Google Patents

Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors

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Publication number
IL303536A
IL303536A IL303536A IL30353623A IL303536A IL 303536 A IL303536 A IL 303536A IL 303536 A IL303536 A IL 303536A IL 30353623 A IL30353623 A IL 30353623A IL 303536 A IL303536 A IL 303536A
Authority
IL
Israel
Prior art keywords
nucleic acid
acid molecule
cideb
subject
inhibitor
Prior art date
Application number
IL303536A
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English (en)
Hebrew (he)
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of IL303536A publication Critical patent/IL303536A/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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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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    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6844—Nucleic acid amplification reactions
    • C12Q1/6858—Allele-specific amplification
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6869—Methods for sequencing
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
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    • C12Q2600/156—Polymorphic or mutational markers
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Lubricants (AREA)
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IL303536A 2020-12-23 2021-12-22 Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors IL303536A (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063129725P 2020-12-23 2020-12-23
US202163149258P 2021-02-13 2021-02-13
US202163246101P 2021-09-20 2021-09-20
US202163257137P 2021-10-19 2021-10-19
PCT/US2021/064987 WO2022140624A1 (en) 2020-12-23 2021-12-22 Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors

Publications (1)

Publication Number Publication Date
IL303536A true IL303536A (en) 2023-08-01

Family

ID=80001294

Family Applications (1)

Application Number Title Priority Date Filing Date
IL303536A IL303536A (en) 2020-12-23 2021-12-22 Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors

Country Status (8)

Country Link
US (3) US12018259B2 (https=)
EP (1) EP4267739A1 (https=)
JP (1) JP2024502282A (https=)
KR (1) KR20230124915A (https=)
CA (1) CA3201624A1 (https=)
IL (1) IL303536A (https=)
MX (1) MX2023007587A (https=)
WO (1) WO2022140624A1 (https=)

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JP2024538859A (ja) * 2021-08-31 2024-10-24 アルナイラム ファーマシューティカルズ， インコーポレイテッド 細胞死誘導ＤＦＦＡ様エフェクターＢ（ＣＩＤＥＢ）ｉＲＮＡ組成物およびその使用方法
US20230193392A1 (en) 2021-12-20 2023-06-22 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
CN119278205A (zh) * 2022-04-07 2025-01-07 得克萨斯大学体系董事会 用靶向CIDEB的siRNA治疗肝病的组合物和方法
US20240071625A1 (en) * 2022-08-29 2024-02-29 Cedars-Sinai Medical Center Nafld identification and prediction systems and methods
WO2024109722A1 (zh) * 2022-11-21 2024-05-30 广州必贝特医药股份有限公司 用于抑制CIDEB基因表达的siRNA、药物及其应用
WO2024188164A1 (en) * 2023-03-10 2024-09-19 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of cell death inducing dffa like effector b (cideb)
AU2024243110A1 (en) * 2023-04-07 2025-11-06 Rona Bioscience, Limited Dsrna molecule for regulating expression of cideb mrna
CN118063533B (zh) * 2023-09-18 2024-08-27 广州必贝特医药股份有限公司 修饰的核苷酸化合物、其寡聚核苷酸及其应用
WO2025162435A1 (en) * 2024-02-02 2025-08-07 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of liver disease
WO2025178854A2 (en) * 2024-02-19 2025-08-28 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting cideb and related methods
WO2025245188A2 (en) * 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease

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WO2022140624A1 (en) 2022-06-30
MX2023007587A (es) 2023-09-21
AU2021410768A1 (en) 2023-06-22
US11773393B2 (en) 2023-10-03
US12018259B2 (en) 2024-06-25
US20220220474A1 (en) 2022-07-14
AU2021410768A9 (en) 2025-01-09
US20220282253A1 (en) 2022-09-08
KR20230124915A (ko) 2023-08-28
EP4267739A1 (en) 2023-11-01
US20240376471A1 (en) 2024-11-14
JP2024502282A (ja) 2024-01-18
CA3201624A1 (en) 2022-06-30

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