JPWO2022019325A1 - - Google Patents

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Publication number
JPWO2022019325A1
JPWO2022019325A1 JP2022538037A JP2022538037A JPWO2022019325A1 JP WO2022019325 A1 JPWO2022019325 A1 JP WO2022019325A1 JP 2022538037 A JP2022538037 A JP 2022538037A JP 2022538037 A JP2022538037 A JP 2022538037A JP WO2022019325 A1 JPWO2022019325 A1 JP WO2022019325A1
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JP
Japan
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JP2022538037A
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Japanese (ja)
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JP7774807B2 (ja
JPWO2022019325A5 (https=
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Priority to JP2025185750A priority Critical patent/JP2026027332A/ja
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • 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
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/78Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0652Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662Stem cells
    • C12N5/0668Mesenchymal stem cells from other natural sources
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Rheumatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022538037A 2020-07-22 2021-07-21 栄養障害型表皮水疱症の治療薬 Active JP7774807B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025185750A JP2026027332A (ja) 2020-07-22 2025-11-04 栄養障害型表皮水疱症の治療薬

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2020125620 2020-07-22
JP2020125620 2020-07-22
PCT/JP2021/027279 WO2022019325A1 (ja) 2020-07-22 2021-07-21 栄養障害型表皮水疱症の治療薬

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025185750A Division JP2026027332A (ja) 2020-07-22 2025-11-04 栄養障害型表皮水疱症の治療薬

Publications (3)

Publication Number Publication Date
JPWO2022019325A1 true JPWO2022019325A1 (https=) 2022-01-27
JPWO2022019325A5 JPWO2022019325A5 (https=) 2024-07-29
JP7774807B2 JP7774807B2 (ja) 2025-11-25

Family

ID=79729198

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022538037A Active JP7774807B2 (ja) 2020-07-22 2021-07-21 栄養障害型表皮水疱症の治療薬
JP2025185750A Pending JP2026027332A (ja) 2020-07-22 2025-11-04 栄養障害型表皮水疱症の治療薬

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025185750A Pending JP2026027332A (ja) 2020-07-22 2025-11-04 栄養障害型表皮水疱症の治療薬

Country Status (5)

Country Link
US (1) US20240050481A1 (https=)
JP (2) JP7774807B2 (https=)
KR (1) KR20230043906A (https=)
AU (1) AU2020459825A1 (https=)
WO (2) WO2022018884A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025094280A (ja) * 2022-01-21 2025-06-25 国立大学法人大阪大学 細胞シート
CN118001404B (zh) * 2024-02-06 2025-02-25 四川大学华西医院 抑制C17orf67基因表达试剂在制备治疗获得性大疱表皮松解症药物中的用途
KR102736437B1 (ko) * 2024-06-20 2024-12-02 주식회사 에이바이오테크 콜라겐 합성 촉진 활성을 갖는 콜라겐 타입 7 저분자 펩타이드 및 이를 이용한 방법

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018235834A1 (ja) * 2017-06-19 2018-12-27 国立大学法人北海道大学 表皮水疱症の治療剤
JP2019506925A (ja) * 2016-01-04 2019-03-14 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 遺伝子修正自己ケラチノサイトを使用する劣性栄養障害型表皮水疱症のための遺伝子療法
JP2019508454A (ja) * 2016-03-18 2019-03-28 イントレキソン コーポレーション Vii型コラーゲン欠損症の治療のための組成物及び方法
WO2019060719A1 (en) * 2017-09-22 2019-03-28 University Of Miami METHODS AND COMPOSITIONS FOR THE TREATMENT OF BULLETIN EPIDERMOLYSIS
JP2019142819A (ja) * 2018-02-22 2019-08-29 学校法人順天堂 栄養障害型表皮水疱症治療剤
WO2020149395A1 (ja) * 2019-01-18 2020-07-23 国立大学法人大阪大学 栄養障害型表皮水疱症治療薬

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018154413A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of dystrophic epidermolysis bullosa (deb) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019506925A (ja) * 2016-01-04 2019-03-14 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 遺伝子修正自己ケラチノサイトを使用する劣性栄養障害型表皮水疱症のための遺伝子療法
JP2019508454A (ja) * 2016-03-18 2019-03-28 イントレキソン コーポレーション Vii型コラーゲン欠損症の治療のための組成物及び方法
WO2018235834A1 (ja) * 2017-06-19 2018-12-27 国立大学法人北海道大学 表皮水疱症の治療剤
WO2019060719A1 (en) * 2017-09-22 2019-03-28 University Of Miami METHODS AND COMPOSITIONS FOR THE TREATMENT OF BULLETIN EPIDERMOLYSIS
JP2019142819A (ja) * 2018-02-22 2019-08-29 学校法人順天堂 栄養障害型表皮水疱症治療剤
WO2020149395A1 (ja) * 2019-01-18 2020-07-23 国立大学法人大阪大学 栄養障害型表皮水疱症治療薬

Also Published As

Publication number Publication date
WO2022018884A1 (ja) 2022-01-27
JP7774807B2 (ja) 2025-11-25
JP2026027332A (ja) 2026-02-18
WO2022019325A1 (ja) 2022-01-27
KR20230043906A (ko) 2023-03-31
US20240050481A1 (en) 2024-02-15
AU2020459825A1 (en) 2023-03-16

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