CN116209478A - 肌病的预防或治疗剂 - Google Patents

肌病的预防或治疗剂 Download PDF

Info

Publication number
CN116209478A
CN116209478A CN202180061076.3A CN202180061076A CN116209478A CN 116209478 A CN116209478 A CN 116209478A CN 202180061076 A CN202180061076 A CN 202180061076A CN 116209478 A CN116209478 A CN 116209478A
Authority
CN
China
Prior art keywords
mrln
nucleic acid
muscular dystrophy
myopathy
preventive
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
CN202180061076.3A
Other languages
English (en)
Chinese (zh)
Inventor
石川洁
村田俊平
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.)
Tanabe Pharma Corp
Original Assignee
Mitsubishi Tanabe Pharma Corp
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 Mitsubishi Tanabe Pharma Corp filed Critical Mitsubishi Tanabe Pharma Corp
Publication of CN116209478A publication Critical patent/CN116209478A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202180061076.3A 2020-07-17 2021-07-16 肌病的预防或治疗剂 Pending CN116209478A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JP2020-122975 2020-07-17
JP2020122975 2020-07-17
JP2021-025158 2021-02-19
JP2021025158 2021-02-19
PCT/JP2021/026798 WO2022014703A1 (ja) 2020-07-17 2021-07-16 筋疾患の予防または治療剤

Publications (1)

Publication Number Publication Date
CN116209478A true CN116209478A (zh) 2023-06-02

Family

ID=79555651

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180061076.3A Pending CN116209478A (zh) 2020-07-17 2021-07-16 肌病的预防或治疗剂

Country Status (5)

Country Link
US (1) US20230272386A1 (https=)
EP (1) EP4183416A4 (https=)
JP (1) JPWO2022014703A1 (https=)
CN (1) CN116209478A (https=)
WO (1) WO2022014703A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025206092A1 (ja) * 2024-03-27 2025-10-02 田辺三菱製薬株式会社 Ｍｙｏｒｅｇｕｌｉｎ発現を調節するための化合物及び医薬組成物

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103079597A (zh) * 2010-05-13 2013-05-01 夏洛特-梅克伦堡医院管理局D/B/A卡罗莱纳医学中心 包含反义寡核苷酸的药物组合物及其使用方法

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS6147500A (ja) 1984-08-15 1986-03-07 Res Dev Corp Of Japan キメラモノクロ−ナル抗体及びその製造法
GB8607679D0 (en) 1986-03-27 1986-04-30 Winter G P Recombinant dna product
GB8928874D0 (en) 1989-12-21 1990-02-28 Celltech Ltd Humanised antibodies
EP1690934A3 (en) 1990-01-12 2008-07-30 Abgenix, Inc. Generation of xenogeneic antibodies
ATE300615T1 (de) 1990-08-29 2005-08-15 Genpharm Int Transgene mäuse fähig zur produktion heterologer antikörper
CA2161351C (en) 1993-04-26 2010-12-21 Nils Lonberg Transgenic non-human animals capable of producing heterologous antibodies
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
JP5342881B2 (ja) 2006-01-27 2013-11-13 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 ６−修飾された二環式核酸類似体
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
US10543252B2 (en) * 2015-02-05 2020-01-28 University Of Maryland, Baltimore Regulating calcium in excitable cells with a novel protein
US10570183B2 (en) * 2016-04-19 2020-02-25 The Board Of Regents Of The University Of Texas System Use of a small native peptide activator of SERCA pump for treatment of heart failure and other disorders characterized by cytosolic calcium overload
JP2017214337A (ja) * 2016-06-01 2017-12-07 国立研究開発法人国立精神・神経医療研究センター 筋ジストロフィー治療剤
JP7356448B2 (ja) 2018-11-12 2023-10-04 田辺三菱製薬株式会社 架橋型人工核酸ａｌｎａ

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103079597A (zh) * 2010-05-13 2013-05-01 夏洛特-梅克伦堡医院管理局D/B/A卡罗莱纳医学中心 包含反义寡核苷酸的药物组合物及其使用方法

Also Published As

Publication number Publication date
US20230272386A1 (en) 2023-08-31
WO2022014703A1 (ja) 2022-01-20
EP4183416A4 (en) 2025-08-27
EP4183416A1 (en) 2023-05-24
JPWO2022014703A1 (https=) 2022-01-20

Similar Documents

Publication Publication Date Title
JP7089763B2 (ja) トリヌクレオチドリピート伸長が関与する遺伝的疾患の予防または治療における一本鎖アンチセンスオリゴヌクレオチドの使用
US20180155414A1 (en) Targeting Apolipoprotein E (APOE) in Neurologic Disease
EP2978446B1 (en) Anti-cd33 antibody for use in treating alzheimer's disease
AU2016201644B2 (en) Detection and treatment of fibrosis
US8877188B2 (en) Detection and treatment of non-dermal fibrosis
JPWO2015064768A1 (ja) 角膜内皮の小胞体細胞死関連疾患治療薬
US20150132321A1 (en) Methods of modulating angiogenesis via trpv4
US20150297679A1 (en) Methods and assays relating to macrophage differentiation
US20220372489A1 (en) Ppm1a inhibitors and methods of using same
WO2012047706A2 (en) Methods for promotiing reinnervation of auditory hair cells
CN116209478A (zh) 肌病的预防或治疗剂
WO2009021295A2 (en) Inhibition of alpha synuclein toxicity
JP7033072B2 (ja) Ｓｍｏｃ２を標的化する線維症のための治療方法
CN120152741A (zh) 异常蛋白的聚集体蓄积所参与的疾病的预防或治疗剂
WO2025102119A1 (en) Improvement of blood brain barrier integrity
WO2026068572A1 (en) Treatment/prevention of disease by lrg1 inhibition
WO2009026705A1 (en) Method of diagnosing and treating osteoarthritis
JP2007537167A (ja) ホスホリパーゼｃガンマの調節、並びにそれによる疼痛及び侵害受容の制御
HK1183322B (en) Cadherin-11 antagonist for the treatment of fibrosis

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination