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AR129710A1 - Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6) - Google Patents

Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6)

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Publication number
AR129710A1
AR129710A1 ARP230101626A ARP230101626A AR129710A1 AR 129710 A1 AR129710 A1 AR 129710A1 AR P230101626 A ARP230101626 A AR P230101626A AR P230101626 A ARP230101626 A AR P230101626A AR 129710 A1 AR129710 A1 AR 129710A1
Authority
AR
Argentina
Prior art keywords
seq
antisense strand
tmprss6
strand comprises
oligonucleotide
Prior art date
Application number
ARP230101626A
Other languages
English (en)
Inventor
Anna Linda Blois
Christina Marie Priest
Jihye Park
Henryk Dudek
Original Assignee
Novo Nordisk As
Dicerna 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 Novo Nordisk As, Dicerna Pharmaceuticals Inc filed Critical Novo Nordisk As
Publication of AR129710A1 publication Critical patent/AR129710A1/es

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    • 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
    • C12N15/1137—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 N.A.
    • 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
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    • 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
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    • 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
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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

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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)
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  • General Engineering & Computer Science (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Hematology (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

En la presente descripción se proporcionan oligonucleótidos que inhiben la expresión de TMPRSS6. También, se proporcionan composiciones que incluyen los mismos y usos de estos, particularmente usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociadas con la deficiencia o supresión de hepcidina. Reivindicación 1: Un oligonucleótido de ARNi para reducir la expresión de serina proteasa transmembrana 6 (TMPRSS6), el oligonucleótido comprende una hebra sentido y una hebra antisentido, en donde la hebra sentido y la hebra antisentido forman una región híbrida, en donde la hebra antisentido comprende al menos 20 nucleótidos contiguos de una secuencia de nucleótidos como se establece en la SEQ ID Nº 600, y en donde la hebra antisentido comprende una región de complementariedad con una secuencia diana de ARNm de TMPRSS6, la región de complementariedad es de al menos 18 nucleótidos contiguos. Reivindicación 2: El oligonucleótido de ARNi de acuerdo con la reivindicación 1, en donde la región de complementariedad es de al menos 19 nucleótidos contiguos. Reivindicación 3: El oligonucleótido de ARNi de acuerdo con cualquiera de las reivindicaciones 1 - 2, en donde la hebra sentido comprende una secuencia de nucleótidos como se establece en la SEQ ID Nº 844, opcionalmente en donde la hebra sentido comprende una secuencia de nucleótidos como se establece en la SEQ ID Nº 579. Reivindicación 4: El oligonucleótido de ARNi de acuerdo con cualquiera de las reivindicaciones 1 - 3, en donde la hebra antisentido comprende al menos 21 nucleótidos contiguos de una secuencia de nucleótidos como se establece en la SEQ ID Nº 600, en donde opcionalmente la hebra antisentido comprende una secuencia de nucleótidos como se establece en la SEQ ID Nº 600. Reivindicación 5: El oligonucleótido de ARNi de acuerdo con cualquiera de las reivindicaciones 1 - 4, en donde el oligonucleótido comprende al menos un nucleótido modificado, opcionalmente en donde todos los nucleótidos del oligonucleótido son nucleótidos modificados. Reivindicación 12: Un oligonucleótido de ARNi para reducir la expresión de TMPRSS6, el oligonucleótido comprende una hebra sentido y una hebra antisentido, en donde la hebra sentido comprende la secuencia y todas las modificaciones de 5’ - [mGs] [mG] [mU] [mG] [mC] [mU] [mA] [fC] [fU] [fC] [fU] [mG] [mG] [mU] [mA] [mU] [mU] [mU] [mC] [mA] [mG] [mC] [mA] [mG] [mC] [mC] [mG] [ademA-GalNAc] [ademA-GalNAc] [ademA-GalNAc] [mG] [mG] [mC] [mU] [mG] [mC] - 3’ (SEQ ID Nº 621), y en donde la hebra antisentido comprende la secuencia y todas las modificaciones de 5’ - [MeFosfonato-4O-mUs] [fGs] [fAs] [fA] [fA] [mU] [fA] [mC] [mC] [fA] [mG] [mA] [mG] [fU] [mA] [mG] [mC] [mA] [mC] [mCs] [mGs] [mG] - 3’ (SEQ ID Nº 642), en donde mC, mA, mG, mU = ribonucleósidos 2’-OMe; fA, fC, fG, fU = ribonucleósidos 2’-F; s = fosforotioato, y en donde ademA-GalNAc = al compuesto de fórmula (1). Reivindicación 15: El oligonucleótido de ARNi de acuerdo con cualquiera de las reivindicaciones 1 - 12, o la composición farmacéutica de acuerdo con la reivindicación 13, para su uso en el tratamiento de la hemocromatosis tal como la hemocromatosis hereditaria o betatalasemia.
ARP230101626A 2022-06-24 2023-06-23 Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6) AR129710A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263355210P 2022-06-24 2022-06-24
EP22209113 2022-11-23

Publications (1)

Publication Number Publication Date
AR129710A1 true AR129710A1 (es) 2024-09-18

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ID=87036742

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ARP230101626A AR129710A1 (es) 2022-06-24 2023-06-23 Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6)

Country Status (8)

Country Link
US (2) US11952574B2 (es)
JP (1) JP7560616B2 (es)
KR (1) KR102759742B1 (es)
CN (1) CN119403927A (es)
AR (1) AR129710A1 (es)
AU (1) AU2023288906A1 (es)
IL (1) IL317012A (es)
WO (1) WO2023247738A1 (es)

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Also Published As

Publication number Publication date
WO2023247738A1 (en) 2023-12-28
TW202400193A (zh) 2024-01-01
CN119403927A (zh) 2025-02-07
US20240309388A1 (en) 2024-09-19
AU2023288906A1 (en) 2024-12-12
IL317012A (en) 2025-01-01
KR20240002200A (ko) 2024-01-04
US20240002858A1 (en) 2024-01-04
JP7560616B2 (ja) 2024-10-02
KR102759742B1 (ko) 2025-02-04
JP2024002985A (ja) 2024-01-11
US11952574B2 (en) 2024-04-09

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