KR20220144837A - Htt 발현을 감소시키기 위한 방법 - Google Patents

Htt 발현을 감소시키기 위한 방법 Download PDF

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Publication number
KR20220144837A
KR20220144837A KR1020227032214A KR20227032214A KR20220144837A KR 20220144837 A KR20220144837 A KR 20220144837A KR 1020227032214 A KR1020227032214 A KR 1020227032214A KR 20227032214 A KR20227032214 A KR 20227032214A KR 20220144837 A KR20220144837 A KR 20220144837A
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weeks
once
modified oligonucleotide
administering
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씨. 프랭크 베넷
대니얼 에이. 노리스
앤 브이. 스미스
로저 레인
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아이오니스 파마수티컬즈, 인코포레이티드
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Publication of KR20220144837A publication Critical patent/KR20220144837A/ko
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • 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
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special 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)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
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  • 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)
KR1020227032214A 2020-02-21 2021-02-19 Htt 발현을 감소시키기 위한 방법 Pending KR20220144837A (ko)

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
KR20220144837A true KR20220144837A (ko) 2022-10-27

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KR1020227032214A Pending KR20220144837A (ko) 2020-02-21 2021-02-19 Htt 발현을 감소시키기 위한 방법

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

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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
EP4107269A4 (en) 2024-11-20
CN115279903A (zh) 2022-11-01
ZA202209112B (en) 2024-10-30

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Patent event date: 20220916

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Patent event code: PA02012R01D

Patent event date: 20240219

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