TWI868755B - 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法 - Google Patents

抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法 Download PDF

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TWI868755B
TWI868755B TW112123278A TW112123278A TWI868755B TW I868755 B TWI868755 B TW I868755B TW 112123278 A TW112123278 A TW 112123278A TW 112123278 A TW112123278 A TW 112123278A TW I868755 B TWI868755 B TW I868755B
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nucleotides
oligonucleotide
seq
antisense strand
tmprss6
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TW112123278A
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Chinese (zh)
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TW202400193A (zh
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安娜 琳達 布洛伊斯
克里斯蒂娜 馬利 普萊斯特
朴智惠
亨利克 杜德克
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丹麥商諾佛 儂迪克股份有限公司
美商戴瑟納製藥股份有限公司
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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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    • 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
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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]
    • 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
    • C12N2310/31—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
    • C12N2310/32—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
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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
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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)
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  • Diabetes (AREA)
  • Biochemistry (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)
TW112123278A 2022-06-24 2023-06-21 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法 TWI868755B (zh)

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US202263355210P 2022-06-24 2022-06-24
US63/355,210 2022-06-24
EP22209113 2022-11-23
EP22209113.4 2022-11-23

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TW202400193A TW202400193A (zh) 2024-01-01
TWI868755B true TWI868755B (zh) 2025-01-01

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US (2) US11952574B2 (cg-RX-API-DMAC7.html)
EP (1) EP4544049A1 (cg-RX-API-DMAC7.html)
JP (1) JP7560616B2 (cg-RX-API-DMAC7.html)
KR (1) KR102759742B1 (cg-RX-API-DMAC7.html)
CN (1) CN119403927A (cg-RX-API-DMAC7.html)
AR (1) AR129710A1 (cg-RX-API-DMAC7.html)
AU (1) AU2023288906A1 (cg-RX-API-DMAC7.html)
CA (1) CA3257962A1 (cg-RX-API-DMAC7.html)
CO (1) CO2024017422A2 (cg-RX-API-DMAC7.html)
IL (1) IL317012A (cg-RX-API-DMAC7.html)
MA (1) MA71238A (cg-RX-API-DMAC7.html)
MX (1) MX2024015763A (cg-RX-API-DMAC7.html)
TW (1) TWI868755B (cg-RX-API-DMAC7.html)
WO (1) WO2023247738A1 (cg-RX-API-DMAC7.html)

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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
CN122477285A (zh) * 2024-01-08 2026-07-28 上海舶望制药有限公司 用于抑制跨膜丝氨酸蛋白酶6（tmprss6）表达的组合物和方法
WO2026003163A1 (en) * 2024-06-26 2026-01-02 Silence Therapeutics Gmbh Sirna against tmprss6 for treating polycythemia vera

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US20240002858A1 (en) 2024-01-04
TW202400193A (zh) 2024-01-01
CN119403927A (zh) 2025-02-07
AR129710A1 (es) 2024-09-18
EP4544049A1 (en) 2025-04-30
JP2024002985A (ja) 2024-01-11
MX2024015763A (es) 2025-02-10
AU2023288906A1 (en) 2024-12-12
MA71238A (fr) 2025-04-30
CO2024017422A2 (es) 2025-05-08
US12503701B2 (en) 2025-12-23
WO2023247738A1 (en) 2023-12-28
KR102759742B1 (ko) 2025-02-04
CA3257962A1 (en) 2023-12-28
KR20240002200A (ko) 2024-01-04
US20240309388A1 (en) 2024-09-19
US11952574B2 (en) 2024-04-09
IL317012A (en) 2025-01-01
JP7560616B2 (ja) 2024-10-02

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