JP7461687B2 - 筋強直性ジストロフィー１型治療薬 - Google Patents

筋強直性ジストロフィー１型治療薬 Download PDF

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Publication number
JP7461687B2
JP7461687B2 JP2023517588A JP2023517588A JP7461687B2 JP 7461687 B2 JP7461687 B2 JP 7461687B2 JP 2023517588 A JP2023517588 A JP 2023517588A JP 2023517588 A JP2023517588 A JP 2023517588A JP 7461687 B2 JP7461687 B2 JP 7461687B2
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ppr
vector
amino acids
protein
cug
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JPWO2022230924A1 (https=
Inventor
雅之 中森
祐介 八木
崇喜 今井
英里香 沖井
喬之 玉井
理紗 二宮
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EDITFORCE, INC.
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EDITFORCE, INC.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/168—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from plants
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/415—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from plants
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Botany (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2023517588A 2021-04-30 2022-04-27 筋強直性ジストロフィー１型治療薬 Active JP7461687B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024038955A JP7854207B2 (ja) 2021-04-30 2024-03-13 筋強直性ジストロフィー１型治療薬

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JP2021077262 2021-04-30
JP2021077262 2021-04-30
PCT/JP2022/019038 WO2022230924A1 (ja) 2021-04-30 2022-04-27 筋強直性ジストロフィー１型治療薬

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JPWO2022230924A1 JPWO2022230924A1 (https=) 2022-11-03
JP7461687B2 true JP7461687B2 (ja) 2024-04-04

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JP2024038955A Active JP7854207B2 (ja) 2021-04-30 2024-03-13 筋強直性ジストロフィー１型治療薬

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US (1) US20250281571A1 (https=)
EP (1) EP4331619A4 (https=)
JP (2) JP7461687B2 (https=)
KR (1) KR20240004715A (https=)
CN (1) CN117597151A (https=)
AU (1) AU2022265246A1 (https=)
BR (1) BR112023022129A2 (https=)
CA (1) CA3216313A1 (https=)
IL (1) IL308029A (https=)
TW (1) TW202304948A (https=)
WO (1) WO2022230924A1 (https=)
ZA (1) ZA202309721B (https=)

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013058404A1 (ja) 2011-10-21 2013-04-25 国立大学法人九州大学 Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用
WO2018030488A1 (ja) 2016-08-10 2018-02-15 和光純薬工業株式会社 Ｐｐｒモチーフを利用したｄｎａ結合性タンパク質およびその利用
JP2020196751A (ja) 2012-09-25 2020-12-10 ジェンザイム・コーポレーション 筋強直性ジストロフィーを治療するためのペプチド結合モルホリノアンチセンスオリゴヌクレオチド
WO2021201198A1 (ja) 2020-03-31 2021-10-07 エディットフォース株式会社 標的ｒｎａを編集する方法
WO2021235293A1 (ja) 2020-05-20 2021-11-25 国立大学法人大阪大学 Ｃｕｇリピート配列の結合剤

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12503495B2 (en) * 2014-04-14 2025-12-23 Association Institut De Myologie Treatment of myotonic dystrophy

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013058404A1 (ja) 2011-10-21 2013-04-25 国立大学法人九州大学 Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用
JP2020196751A (ja) 2012-09-25 2020-12-10 ジェンザイム・コーポレーション 筋強直性ジストロフィーを治療するためのペプチド結合モルホリノアンチセンスオリゴヌクレオチド
WO2018030488A1 (ja) 2016-08-10 2018-02-15 和光純薬工業株式会社 Ｐｐｒモチーフを利用したｄｎａ結合性タンパク質およびその利用
WO2021201198A1 (ja) 2020-03-31 2021-10-07 エディットフォース株式会社 標的ｒｎａを編集する方法
WO2021235293A1 (ja) 2020-05-20 2021-11-25 国立大学法人大阪大学 Ｃｕｇリピート配列の結合剤

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
YAGI, Y., et al.，Elucidation of the RNA Recognition Code for Pentatricopeptide Repeat Proteins Involved in Organelle，PLOS ONE，2013年，Vol.8, No.3, Article No.e57286，pp.1-8
中森 雅之 ほか，筋強直性ジストロフィー ー異常ＲＮＡによる病態機序と新たな治療法の探索，BRAIN and NERVE，医学書院，2011年，Vol.63, No.11，pp.1161-1168

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Publication number Publication date
JP2024069430A (ja) 2024-05-21
CN117597151A (zh) 2024-02-23
JPWO2022230924A1 (https=) 2022-11-03
BR112023022129A2 (pt) 2024-01-09
EP4331619A1 (en) 2024-03-06
JP7854207B2 (ja) 2026-05-01
CA3216313A1 (en) 2022-11-03
AU2022265246A1 (en) 2023-11-02
EP4331619A4 (en) 2024-12-25
IL308029A (en) 2023-12-01
WO2022230924A1 (ja) 2022-11-03
TW202304948A (zh) 2023-02-01
US20250281571A1 (en) 2025-09-11
KR20240004715A (ko) 2024-01-11
ZA202309721B (en) 2025-03-26

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