KR20220133925A - Atn1의 안티센스 올리고뉴클레오티드 - Google Patents

Atn1의 안티센스 올리고뉴클레오티드 Download PDF

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Publication number
KR20220133925A
KR20220133925A KR1020227029055A KR20227029055A KR20220133925A KR 20220133925 A KR20220133925 A KR 20220133925A KR 1020227029055 A KR1020227029055 A KR 1020227029055A KR 20227029055 A KR20227029055 A KR 20227029055A KR 20220133925 A KR20220133925 A KR 20220133925A
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South Korea
Prior art keywords
dna
nucleic acid
artificial sequence
synthetic oligonucleotide
compound
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Korean (ko)
Inventor
신지 하시모토
이즈미 고토
유스케 이리야마
Original Assignee
가부시키가이샤산와카가쿠켄큐쇼
닛산 가가쿠 가부시키가이샤
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Publication of KR20220133925A publication Critical patent/KR20220133925A/ko
Pending legal-status Critical Current

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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
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
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    • 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
    • A—HUMAN NECESSITIES
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
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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 ===---===

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Veterinary Medicine (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
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  • Psychology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pain & Pain Management (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020227029055A 2020-01-31 2021-01-29 Atn1의 안티센스 올리고뉴클레오티드 Pending KR20220133925A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JPJP-P-2020-015521 2020-01-31
JP2020015521 2020-01-31
JP2020183718 2020-11-02
JPJP-P-2020-183718 2020-11-02
PCT/JP2021/003293 WO2021153747A1 (ja) 2020-01-31 2021-01-29 Ａｔｎ１のアンチセンスオリゴヌクレオチド

Publications (1)

Publication Number Publication Date
KR20220133925A true KR20220133925A (ko) 2022-10-05

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KR1020227029055A Pending KR20220133925A (ko) 2020-01-31 2021-01-29 Atn1의 안티센스 올리고뉴클레오티드

Country Status (8)

Country Link
US (1) US20230127437A1 (https=)
EP (1) EP4098747A4 (https=)
JP (2) JP7753106B2 (https=)
KR (1) KR20220133925A (https=)
CN (1) CN114929876A (https=)
AU (1) AU2021212949A1 (https=)
CA (1) CA3169474A1 (https=)
WO (1) WO2021153747A1 (https=)

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WO2023112931A1 (ja) * 2021-12-13 2023-06-22 日本新薬株式会社 ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド
WO2023164656A2 (en) * 2022-02-25 2023-08-31 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atn1 expression
WO2023201281A2 (en) * 2022-04-14 2023-10-19 University Of Massachusetts Oligonucleotides for atn1 modulation
WO2025043202A1 (en) * 2023-08-23 2025-02-27 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atn1 expression

Citations (1)

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WO2015017675A2 (en) 2013-07-31 2015-02-05 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion

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JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
JP2009536222A (ja) 2006-05-05 2009-10-08 アイシス ファーマシューティカルズ， インコーポレーテッド Ｐｃｓｋ９の発現を調節するための化合物および方法
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Publication number Publication date
CA3169474A1 (en) 2021-08-05
WO2021153747A1 (ja) 2021-08-05
EP4098747A4 (en) 2025-03-26
CN114929876A (zh) 2022-08-19
AU2021212949A1 (en) 2022-09-01
EP4098747A1 (en) 2022-12-07
US20230127437A1 (en) 2023-04-27
JP2026004436A (ja) 2026-01-14
JP7753106B2 (ja) 2025-10-14
JPWO2021153747A1 (https=) 2021-08-05

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