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HUE063026T2 - Eljárások és készítmények LDHA expressziójának gátlására - Google Patents

Eljárások és készítmények LDHA expressziójának gátlására

Info

Publication number
HUE063026T2
HUE063026T2 HUE18867097A HUE18867097A HUE063026T2 HU E063026 T2 HUE063026 T2 HU E063026T2 HU E18867097 A HUE18867097 A HU E18867097A HU E18867097 A HUE18867097 A HU E18867097A HU E063026 T2 HUE063026 T2 HU E063026T2
Authority
HU
Hungary
Prior art keywords
ldha
compositions
methods
inhibiting expression
inhibiting
Prior art date
Application number
HUE18867097A
Other languages
English (en)
Inventor
Bob Brown
Henryk Dudek
Utsav Saxena
Natalie Pursell
Cheng Lai
Weimin Wang
Rachel Storr
Naim Nazef
Boyoung Kim
Original Assignee
Novo Nordisk Healthcare Ag
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 Novo Nordisk Healthcare Ag filed Critical Novo Nordisk Healthcare Ag
Publication of HUE063026T2 publication Critical patent/HUE063026T2/hu

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/04Drugs for disorders of the urinary system for urolithiasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y101/00Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/01Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01027L-Lactate dehydrogenase (1.1.1.27)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Urology & Nephrology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
HUE18867097A 2017-10-13 2018-10-12 Eljárások és készítmények LDHA expressziójának gátlására HUE063026T2 (hu)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762572403P 2017-10-13 2017-10-13
US201762572398P 2017-10-13 2017-10-13
US201862726950P 2018-09-04 2018-09-04

Publications (1)

Publication Number Publication Date
HUE063026T2 true HUE063026T2 (hu) 2023-12-28

Family

ID=66101691

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE18867097A HUE063026T2 (hu) 2017-10-13 2018-10-12 Eljárások és készítmények LDHA expressziójának gátlására

Country Status (15)

Country Link
US (3) US11286488B2 (hu)
EP (2) EP4265261A3 (hu)
JP (2) JP7308213B2 (hu)
KR (2) KR102609396B1 (hu)
CN (3) CN111448319B (hu)
AU (1) AU2018346971A1 (hu)
CA (1) CA3078933A1 (hu)
ES (1) ES2955045T3 (hu)
HR (1) HRP20231063T1 (hu)
HU (1) HUE063026T2 (hu)
IL (1) IL273875A (hu)
MX (1) MX2020003836A (hu)
PL (1) PL3679141T3 (hu)
RS (1) RS64483B1 (hu)
WO (1) WO2019075419A1 (hu)

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WO2016057932A1 (en) 2014-10-10 2016-04-14 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
EP3334499A4 (en) 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
HUE063026T2 (hu) 2017-10-13 2023-12-28 Novo Nordisk Healthcare Ag Eljárások és készítmények LDHA expressziójának gátlására
EP3684377B1 (en) * 2017-10-20 2022-12-07 Dicerna Pharmaceuticals, Inc. Methods for treating hepatitis b infection
KR20200109311A (ko) 2018-01-16 2020-09-22 다이서나 파마수이티컬, 인크. Aldh2 발현을 억제하기 위한 조성물 및 방법
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
JP2022545118A (ja) * 2019-08-23 2022-10-25 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
IL291841A (en) * 2019-10-02 2022-06-01 Dicerna Pharmaceuticals Inc Chemical modifications of tiny interfering rna with minimal fluoride content
EP4121534A1 (en) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
WO2021234543A1 (en) * 2020-05-18 2021-11-25 Chinook Therapeutics Canada, Inc. Substituted pyrazolyl compounds and methods of use thereof
MX2023005627A (es) * 2020-11-13 2023-05-24 Dicerna Pharmaceuticals Inc Modificaciones quimicas para inhibir la expresion de aldh2.
CN117836304A (zh) 2021-03-19 2024-04-05 巴克姆股份公司 改进的寡核苷酸合成
WO2022214692A1 (en) 2021-04-09 2022-10-13 Bachem Holding Ag Pseudo solid phase protecting group and methods for the synthesis of oligonucleotides and oligonucleotide conjugates
AU2022261359A1 (en) * 2021-04-19 2023-10-12 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
US11649260B2 (en) 2021-06-18 2023-05-16 Hongene Biotech Corporation Functionalized N-acetylgalactosamine nucleosides
TW202333748A (zh) 2021-07-19 2023-09-01 美商艾拉倫製藥股份有限公司 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物
US20230107967A1 (en) * 2021-08-25 2023-04-06 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting alpha-1 antitrypsin expression
US11692001B2 (en) 2021-08-30 2023-07-04 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
AR127260A1 (es) * 2021-10-05 2024-01-03 Dicerna Pharmaceuticals Inc Composiciones y métodos para inhibir el receptor de melanocortina 2 y la expresión del citocromo p450 11b
WO2023114746A1 (en) 2021-12-15 2023-06-22 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
WO2024061842A1 (en) 2022-09-19 2024-03-28 Bachem Holding Ag Improved oligonucleotide synthesis
WO2024061157A1 (en) * 2022-09-19 2024-03-28 Kylonova (Xiamen) Biopharma Co., Ltd. Carbohydrate-oligonucleotide conjugates, pharmaceutical compositions, and therapeutic applications
WO2024083746A1 (en) 2022-10-17 2024-04-25 Bachem Holding Ag Method and composition for oligonucleotide synthesis
WO2024118503A1 (en) 2022-11-28 2024-06-06 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs

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US10920226B2 (en) 2002-11-14 2021-02-16 Thermo Fisher Scientific Inc. siRNA targeting LDHA
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US20050014692A1 (en) 2003-01-21 2005-01-20 Newgard Christopher B. Lactate dehydrogenase as a novel target and reagent for diabetes therapy
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
CA2559955C (en) 2004-03-15 2016-02-16 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna
KR20070085113A (ko) 2004-05-11 2007-08-27 가부시키가이샤 알파젠 Rna간섭을 생기게 하는 폴리뉴클레오티드, 및 이를 이용한유전자발현억제 방법
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TWI595885B (zh) 2012-05-02 2017-08-21 喜納製藥公司 包含四galnac之新穎結合物及傳送寡核苷酸之方法
WO2016057932A1 (en) 2014-10-10 2016-04-14 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
CN109517878A (zh) 2014-12-18 2019-03-26 加利福尼亚大学董事会 使用纳米管的核酸聚合酶构象变化的检测
AR104754A1 (es) * 2015-05-29 2017-08-09 Arrowhead Pharmaceuticals Inc Agentes de unión de tetrapéptido escindibles biológicamente
HUE063026T2 (hu) 2017-10-13 2023-12-28 Novo Nordisk Healthcare Ag Eljárások és készítmények LDHA expressziójának gátlására

Also Published As

Publication number Publication date
WO2019075419A1 (en) 2019-04-18
KR102609396B1 (ko) 2023-12-01
US20210062199A1 (en) 2021-03-04
EP4265261A3 (en) 2024-01-24
PL3679141T3 (pl) 2023-10-02
KR20230169413A (ko) 2023-12-15
EP3679141B1 (en) 2023-06-07
EP3679141A1 (en) 2020-07-15
MX2020003836A (es) 2020-11-06
CN111448319B (zh) 2024-05-17
IL273875A (en) 2020-05-31
CA3078933A1 (en) 2019-04-18
CN118530989A (zh) 2024-08-23
CN111448319A (zh) 2020-07-24
US20200283775A1 (en) 2020-09-10
KR20200078530A (ko) 2020-07-01
US20240182901A1 (en) 2024-06-06
EP3679141A4 (en) 2021-06-16
EP3679141C0 (en) 2023-06-07
JP2023139014A (ja) 2023-10-03
JP2020536587A (ja) 2020-12-17
EP4265261A2 (en) 2023-10-25
CN118440940A (zh) 2024-08-06
US11661604B2 (en) 2023-05-30
AU2018346971A1 (en) 2020-04-23
HRP20231063T1 (hr) 2023-12-22
ES2955045T3 (es) 2023-11-28
US11286488B2 (en) 2022-03-29
JP7308213B2 (ja) 2023-07-13
RS64483B1 (sr) 2023-09-29

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