JP7774807B2 - 栄養障害型表皮水疱症の治療薬 - Google Patents

栄養障害型表皮水疱症の治療薬

Info

Publication number
JP7774807B2
JP7774807B2 JP2022538037A JP2022538037A JP7774807B2 JP 7774807 B2 JP7774807 B2 JP 7774807B2 JP 2022538037 A JP2022538037 A JP 2022538037A JP 2022538037 A JP2022538037 A JP 2022538037A JP 7774807 B2 JP7774807 B2 JP 7774807B2
Authority
JP
Japan
Prior art keywords
cells
blister
derived
acid sequence
type vii
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.)
Active
Application number
JP2022538037A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2022019325A5 (https=
JPWO2022019325A1 (https=
Inventor
克人 玉井
康 菊池
智樹 玉越
尊彦 山崎
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.)
StemRIM Inc
University of Osaka NUC
Original Assignee
Osaka University NUC
StemRIM Inc
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 Osaka University NUC, StemRIM Inc filed Critical Osaka University NUC
Publication of JPWO2022019325A1 publication Critical patent/JPWO2022019325A1/ja
Publication of JPWO2022019325A5 publication Critical patent/JPWO2022019325A5/ja
Priority to JP2025185750A priority Critical patent/JP2026027332A/ja
Application granted granted Critical
Publication of JP7774807B2 publication Critical patent/JP7774807B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662—Stem cells
    • C12N5/0668—Mesenchymal stem cells from other natural sources
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use 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 JPWO2022019325A1 (https=) 2022-01-27
JPWO2022019325A5 JPWO2022019325A5 (https=) 2024-07-29
JP7774807B2 true 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
JP2026027332A (ja) 2026-02-18
WO2022019325A1 (ja) 2022-01-27
JPWO2022019325A1 (https=) 2022-01-27
KR20230043906A (ko) 2023-03-31
US20240050481A1 (en) 2024-02-15
AU2020459825A1 (en) 2023-03-16

Similar Documents

Publication Publication Date Title
JP7815327B2 (ja) フソソーム組成物およびその使用
JP2026067851A (ja) 標的化脂質粒子及び組成物ならびにその使用
JP2026027332A (ja) 栄養障害型表皮水疱症の治療薬
JP2021530985A (ja) フソソームの組成物及びその使用
US11371059B2 (en) Viral particle for the transfer of RNAs, especially into cells involved in immune response
JP2024521811A (ja) 短縮型ヒヒ内在性レトロウイルス（ＢａＥＶ）エンベロープ糖タンパク質を含む脂質粒子、ならびに関連する方法及び使用
KR20160075676A (ko) 방법
JP7228523B2 (ja) ヘモグロビン異常症を治療するためのベクターおよび組成物
JP2025188163A (ja) ウイルスフソソームを産生するための方法及び組成物
WO2019040655A1 (en) LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES
JP7536296B2 (ja) 栄養障害型表皮水疱症治療薬
JP2018519827A (ja) 逆配向のヒトユビキチンｃプロモーターを含むレトロウイルスベクター
US20260055146A1 (en) Delivery of heterologous proteins
Goyvaerts et al. Targeted lentiviral vectors: current applications and future potential
JPWO2007020873A1 (ja) 免疫不全ウイルス感染症の治療または予防のための核酸
KR20230131816A (ko) 저면역원성 줄기세포, 줄기세포로부터 분화되거나 유래된 저면역원성 세포및 이의 제조방법
WO2023140349A1 (ja) 細胞シート
US20190099451A1 (en) Retroviral construct harboring a let-7 insensitive nucleic acid encoding hmga2 and methods of use thereof
KR20260016952A (ko) 탠덤 퓨소젠 및 관련 지질 입자
EP4649162A1 (en) Regulatory sequences and uses thereof
WO2025184529A1 (en) Viral particles with fusogen display and related compositions and methods

Legal Events

Date Code Title Description
A80 Written request to apply exceptions to lack of novelty of invention

Free format text: JAPANESE INTERMEDIATE CODE: A801

Effective date: 20230105

A80 Written request to apply exceptions to lack of novelty of invention

Free format text: JAPANESE INTERMEDIATE CODE: A80

Effective date: 20230105

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240719

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240719

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250805

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250925

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20251007

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20251104

R150 Certificate of patent or registration of utility model

Ref document number: 7774807

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150