CN115279903A - 用于降低htt表达的方法 - Google Patents

用于降低htt表达的方法 Download PDF

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Publication number
CN115279903A
CN115279903A CN202180016151.4A CN202180016151A CN115279903A CN 115279903 A CN115279903 A CN 115279903A CN 202180016151 A CN202180016151 A CN 202180016151A CN 115279903 A CN115279903 A CN 115279903A
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weeks
administering
once
modified oligonucleotide
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Chinese (zh)
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F·C·贝内特
D·A·诺里斯
A·V·史密斯
R·莱恩
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—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
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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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Steroid Compounds (AREA)
  • Saccharide Compounds (AREA)
CN202180016151.4A 2020-02-21 2021-02-19 用于降低htt表达的方法 Pending CN115279903A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062980010P 2020-02-21 2020-02-21
US62/980,010 2020-02-21
PCT/US2021/018681 WO2021168183A1 (en) 2020-02-21 2021-02-19 Methods for reducing htt expression

Publications (1)

Publication Number Publication Date
CN115279903A true CN115279903A (zh) 2022-11-01

Family

ID=77391689

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180016151.4A Pending CN115279903A (zh) 2020-02-21 2021-02-19 用于降低htt表达的方法

Country Status (12)

Country Link
US (1) US20230108340A1 (enExample)
EP (1) EP4107269A4 (enExample)
JP (1) JP2023515466A (enExample)
KR (1) KR20220144837A (enExample)
CN (1) CN115279903A (enExample)
AU (1) AU2021224729A1 (enExample)
BR (1) BR112022016370A2 (enExample)
CA (1) CA3172336A1 (enExample)
IL (1) IL295625A (enExample)
MX (1) MX2022010204A (enExample)
WO (1) WO2021168183A1 (enExample)
ZA (1) ZA202209112B (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025265013A2 (en) 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1991677A2 (en) * 2006-01-26 2008-11-19 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
NZ714887A (en) * 2009-09-11 2019-07-26 Ionis Pharmaceuticals Inc Modulation of huntingtin expression
LT3052628T (lt) * 2013-10-04 2020-09-10 Alnylam Pharmaceuticals, Inc. Kompozicijos ir būdai alas1 geno raiškai nuslopinti
EP3797780B1 (en) * 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
IL320434A (en) * 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
US11268093B2 (en) * 2017-01-03 2022-03-08 Rula Zain-Luqman Therapeutic method for huntington's disease
CN114286710A (zh) * 2019-06-28 2022-04-05 宾州研究基金会 治疗亨廷顿病的方法和材料

Also Published As

Publication number Publication date
WO2021168183A1 (en) 2021-08-26
IL295625A (en) 2022-10-01
CA3172336A1 (en) 2021-08-26
BR112022016370A2 (pt) 2022-10-04
AU2021224729A1 (en) 2022-09-08
EP4107269A1 (en) 2022-12-28
MX2022010204A (es) 2022-09-19
JP2023515466A (ja) 2023-04-13
US20230108340A1 (en) 2023-04-06
KR20220144837A (ko) 2022-10-27
EP4107269A4 (en) 2024-11-20
ZA202209112B (en) 2024-10-30

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