CN119403927A - 用于抑制跨膜丝氨酸蛋白酶6（tmprss6）表达的组合物和方法 - Google Patents

用于抑制跨膜丝氨酸蛋白酶6（tmprss6）表达的组合物和方法 Download PDF

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Publication number
CN119403927A
CN119403927A CN202380048101.3A CN202380048101A CN119403927A CN 119403927 A CN119403927 A CN 119403927A CN 202380048101 A CN202380048101 A CN 202380048101A CN 119403927 A CN119403927 A CN 119403927A
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CN
China
Prior art keywords
nucleotides
oligonucleotide
antisense strand
seq
sense strand
Prior art date
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Withdrawn
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CN202380048101.3A
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English (en)
Chinese (zh)
Inventor
A·L·布卢瓦
C·M·普利斯特
朴智慧
H·杜德克
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Novo Nordisk AS
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Novo Nordisk AS
Dicerna Pharmaceuticals Inc
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Publication of CN119403927A publication Critical patent/CN119403927A/zh
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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
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  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
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  • Hematology (AREA)
  • Diabetes (AREA)
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  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202380048101.3A 2022-06-24 2023-06-23 用于抑制跨膜丝氨酸蛋白酶6（tmprss6）表达的组合物和方法 Withdrawn CN119403927A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263355210P 2022-06-24 2022-06-24
US63/355210 2022-06-24
EP22209113 2022-11-23
EP22209113.4 2022-11-23
PCT/EP2023/067070 WO2023247738A1 (en) 2022-06-24 2023-06-23 Compositions and methods for inhibiting transmembrane serine protease 6 (tmprss6) expression

Publications (1)

Publication Number Publication Date
CN119403927A true CN119403927A (zh) 2025-02-07

Family

ID=87036742

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380048101.3A Withdrawn CN119403927A (zh) 2022-06-24 2023-06-23 用于抑制跨膜丝氨酸蛋白酶6（tmprss6）表达的组合物和方法

Country Status (14)

Country Link
US (2) US11952574B2 (https=)
EP (1) EP4544049A1 (https=)
JP (1) JP7560616B2 (https=)
KR (1) KR102759742B1 (https=)
CN (1) CN119403927A (https=)
AR (1) AR129710A1 (https=)
AU (1) AU2023288906A1 (https=)
CA (1) CA3257962A1 (https=)
CO (1) CO2024017422A2 (https=)
IL (1) IL317012A (https=)
MA (1) MA71238A (https=)
MX (1) MX2024015763A (https=)
TW (1) TWI868755B (https=)
WO (1) WO2023247738A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI868755B (zh) 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
WO2025148896A1 (en) * 2024-01-08 2025-07-17 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of transmembrane serine protease 6 (tmprss6)
WO2026003163A1 (en) * 2024-06-26 2026-01-02 Silence Therapeutics Gmbh Sirna against tmprss6 for treating polycythemia vera

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WO1995006731A2 (en) 1993-09-02 1995-03-09 Ribozyme Pharmaceuticals, Inc. Non-nucleotide containing enzymatic nucleic acid
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
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US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
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Publication number Publication date
EP4544049A1 (en) 2025-04-30
US12503701B2 (en) 2025-12-23
KR20240002200A (ko) 2024-01-04
MX2024015763A (es) 2025-02-10
US20240309388A1 (en) 2024-09-19
US11952574B2 (en) 2024-04-09
WO2023247738A1 (en) 2023-12-28
US20240002858A1 (en) 2024-01-04
JP2024002985A (ja) 2024-01-11
TW202400193A (zh) 2024-01-01
CA3257962A1 (en) 2023-12-28
CO2024017422A2 (es) 2025-05-08
AR129710A1 (es) 2024-09-18
IL317012A (en) 2025-01-01
AU2023288906A1 (en) 2024-12-12
MA71238A (fr) 2025-04-30
KR102759742B1 (ko) 2025-02-04
JP7560616B2 (ja) 2024-10-02
TWI868755B (zh) 2025-01-01

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Applicant before: DICERNA PHARMACEUTICALS, Inc.

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Application publication date: 20250207