CN114929876A - Atn1的反义寡核苷酸 - Google Patents

Atn1的反义寡核苷酸 Download PDF

Info

Publication number
CN114929876A
CN114929876A CN202180008680.XA CN202180008680A CN114929876A CN 114929876 A CN114929876 A CN 114929876A CN 202180008680 A CN202180008680 A CN 202180008680A CN 114929876 A CN114929876 A CN 114929876A
Authority
CN
China
Prior art keywords
compound
pharmaceutically acceptable
acceptable salt
group
nucleoside
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202180008680.XA
Other languages
English (en)
Chinese (zh)
Inventor
桥本信嗣
后藤和泉
入山友辅
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nissan Chemical Corp
Sanwa Kagaku Kenkyusho Co Ltd
Original Assignee
Nissan Chemical Corp
Sanwa Kagaku Kenkyusho Co Ltd
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 Nissan Chemical Corp, Sanwa Kagaku Kenkyusho Co Ltd filed Critical Nissan Chemical Corp
Publication of CN114929876A publication Critical patent/CN114929876A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • A61P25/28—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • C12N2310/11—Antisense
    • 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
    • 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
    • C12N2310/32—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
    • 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
    • C12N2310/32—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
    • 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
    • C12N2310/33—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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Psychiatry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • 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)
CN202180008680.XA 2020-01-31 2021-01-29 Atn1的反义寡核苷酸 Pending CN114929876A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JP2020-015521 2020-01-31
JP2020015521 2020-01-31
JP2020-183718 2020-11-02
JP2020183718 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
CN114929876A true CN114929876A (zh) 2022-08-19

Family

ID=77079145

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180008680.XA Pending CN114929876A (zh) 2020-01-31 2021-01-29 Atn1的反义寡核苷酸

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=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130198877A1 (en) * 2010-08-05 2013-08-01 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
WO2019182037A1 (ja) * 2018-03-20 2019-09-26 国立大学法人東京工業大学 毒性が低減されたアンチセンスオリゴヌクレオチド

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3781879B2 (ja) 1996-11-18 2006-05-31 武 今西 新規ヌクレオチド類縁体
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
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
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
CA2666191C (en) 2006-10-09 2017-07-11 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9
ES2526295T5 (es) 2006-10-18 2021-05-04 Ionis Pharmaceuticals Inc Compuestos antisentido
CA2693742A1 (en) * 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
CN103562215B (zh) * 2011-04-01 2017-04-26 埃西斯药品公司 信号转导及转录激活蛋白3(stat3)表达的调节
JP6112569B2 (ja) 2011-12-16 2017-04-12 国立大学法人 東京医科歯科大学 キメラ２重鎖核酸
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
US10435430B2 (en) 2013-07-31 2019-10-08 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
WO2016127002A1 (en) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
JPWO2017068791A1 (ja) 2015-10-23 2018-08-09 レナセラピューティクス株式会社 少なくとも１つのバルジ構造を有する核酸複合体
JPWO2017068790A1 (ja) 2015-10-23 2018-08-09 レナセラピューティクス株式会社 核酸複合体
AU2016344609B2 (en) * 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
WO2017131124A1 (ja) 2016-01-26 2017-08-03 日産化学工業株式会社 一本鎖オリゴヌクレオチド
EP3418289B1 (en) 2016-02-17 2026-04-08 Institute Of Science Tokyo Artificial nucleoside and artificial nucleotide, and artificial oligonucleotide
JPWO2018003739A1 (ja) 2016-06-30 2019-04-18 レナセラピューティクス株式会社 機能的リガンドを含む核酸複合体
AU2018215440B2 (en) 2017-02-06 2024-10-24 Nissan Chemical Corporation Single-stranded oligonucleotide
EP3587576A4 (en) 2017-02-21 2021-01-06 Osaka University ANTISENSE OLIGONUCLEIC ACID
EP3585900B1 (en) * 2017-02-22 2022-12-21 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
BR112020001430A2 (pt) 2017-07-26 2020-07-28 Nissan Chemical Corporation oligonucleotídeo de fita simples

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130198877A1 (en) * 2010-08-05 2013-08-01 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
WO2019182037A1 (ja) * 2018-03-20 2019-09-26 国立大学法人東京工業大学 毒性が低減されたアンチセンスオリゴヌクレオチド

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JIAXIN HU: ""Allele-Selective Inhibition of Mutant Atrophin-1 Expression by Duplex and Single-Stranded RNAs"", 《BIOCHEMISTRY》, 1 July 2014 (2014-07-01), pages 4510 - 4518, XP055166764, DOI: 10.1021/bi500610r *
MELVIN M EVERS: ""Targeting several CAG expansion diseases by a single antisense oligonucleotide"", 《PLOS ONE》, 30 September 2011 (2011-09-30), pages 1 - 11 *

Also Published As

Publication number Publication date
CA3169474A1 (en) 2021-08-05
WO2021153747A1 (ja) 2021-08-05
EP4098747A4 (en) 2025-03-26
KR20220133925A (ko) 2022-10-05
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

Similar Documents

Publication Publication Date Title
JP7554488B2 (ja) 血液脳関門通過型ヘテロ２本鎖核酸
CN114929876A (zh) Atn1的反义寡核苷酸
JP7690979B2 (ja) 一本鎖オリゴヌクレオチド
JP7394392B2 (ja) 核酸複合体
EP3769769A1 (en) Nucleic acid with reduced toxicity
JP2026052054A (ja) Ｃａｌｍ２のアンチセンスオリゴヌクレオチド
JP7737667B2 (ja) 核酸複合体
WO2022211129A1 (ja) Ａｔｎ１のアンチセンスオリゴヌクレオチド誘導体
US20240182889A1 (en) Microrna-27b inhibitors
WO2022211095A1 (ja) Ｆｇｆｒ３のアンチセンスオリゴヌクレオチド
US20240336920A1 (en) Threose nucleic acid antisense oligonucleotides and methods thereof
HK40074825A (en) Antisense oligonucleotide of atn1
WO2021187540A1 (ja) Ｓｃｎ１ａ遺伝子の発現及び／又は機能調節剤
WO2025047953A1 (ja) ＲａｓＧＲＰ４のアンチセンスオリゴヌクレオチド
WO2026018824A1 (ja) 核酸医薬とその使用
WO2024257848A1 (ja) 遅発性中枢毒性が低減されているアンチセンスオリゴヌクレオチドの設計方法、及びその製造方法
HK40022804A (en) Single-stranded oligonucleotide

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40074825

Country of ref document: HK

SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination