KR20230161459A - 최소 네프린 프로모터 - Google Patents

최소 네프린 프로모터 Download PDF

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Publication number
KR20230161459A
KR20230161459A KR1020237034581A KR20237034581A KR20230161459A KR 20230161459 A KR20230161459 A KR 20230161459A KR 1020237034581 A KR1020237034581 A KR 1020237034581A KR 20237034581 A KR20237034581 A KR 20237034581A KR 20230161459 A KR20230161459 A KR 20230161459A
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KR
South Korea
Prior art keywords
seq
promoter
nucleotide sequence
vector
identity
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
KR1020237034581A
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English (en)
Korean (ko)
Inventor
모인 살렘-우딘
개빈 웰시
발렐이아 쿠즈묵
알란 윌리엄 그리피스
Original Assignee
더 유니버시티 오브 브리스톨
신코나 아이피 홀드코 (3) 리미티드
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
Priority claimed from PCT/GB2021/050633 external-priority patent/WO2021181118A1/en
Priority claimed from PCT/GB2021/051668 external-priority patent/WO2022003357A1/en
Application filed by 더 유니버시티 오브 브리스톨, 신코나 아이피 홀드코 (3) 리미티드 filed Critical 더 유니버시티 오브 브리스톨
Publication of KR20230161459A publication Critical patent/KR20230161459A/ko
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • 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
    • C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • 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
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020237034581A 2021-03-12 2022-03-14 최소 네프린 프로모터 Pending KR20230161459A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
GB2103470.7 2021-03-12
GBPCT/GB2021/050633 2021-03-12
PCT/GB2021/050633 WO2021181118A1 (en) 2020-03-12 2021-03-12 Gene therapy
GBGB2103470.7A GB202103470D0 (en) 2021-03-12 2021-03-12 Promoter
GBPCT/GB2021/051668 2021-06-30
PCT/GB2021/051668 WO2022003357A1 (en) 2020-06-30 2021-06-30 Vector
PCT/GB2022/050649 WO2022189811A1 (en) 2021-03-12 2022-03-14 Minimal nephrin promoter

Publications (1)

Publication Number Publication Date
KR20230161459A true KR20230161459A (ko) 2023-11-27

Family

ID=75623235

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237034581A Pending KR20230161459A (ko) 2021-03-12 2022-03-14 최소 네프린 프로모터

Country Status (18)

Country Link
US (1) US20250283072A1 (enExample)
EP (2) EP4305181B1 (enExample)
JP (1) JP2024509316A (enExample)
KR (1) KR20230161459A (enExample)
CN (1) CN117377770A (enExample)
AU (1) AU2022235209A1 (enExample)
BR (1) BR112023018362A2 (enExample)
CA (1) CA3211566A1 (enExample)
DK (1) DK4305181T3 (enExample)
ES (1) ES3056304T3 (enExample)
FI (1) FI4305181T3 (enExample)
GB (1) GB202103470D0 (enExample)
HR (1) HRP20251561T1 (enExample)
IL (1) IL305714A (enExample)
MX (1) MX2023010573A (enExample)
PL (1) PL4305181T3 (enExample)
PT (1) PT4305181T (enExample)
WO (1) WO2022189811A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202003109D0 (en) * 2020-03-04 2020-04-15 Univ Bristol Gene therapy
GB202003618D0 (en) 2020-03-12 2020-04-29 Univ Bristol Gene Therapy
GB202217332D0 (en) * 2022-11-18 2023-01-04 Univ Bristol Methods
EP4397976A1 (en) * 2023-01-09 2024-07-10 x-kidney diagnostics GmbH Biomarkers for the pre-clinical and/or early-stage detection and/or diagnosis of kidney diseases
GB202309908D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases
GB202309913D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Polynucleotides, avv vectors, and pharmaceutical compositions for treating nephrotic syndrome
GB202309914D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Promoters
GB202309909D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Regulatory elements

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
US8852936B2 (en) 2012-09-21 2014-10-07 Laurantis Pharma Oy Autologous lymph node transfer in combination with VEGF-C or VEGF-D growth factor therapy to treat lymphedema and to improve reconstructive surgery
TW201506036A (zh) 2013-08-14 2015-02-16 Laurantis Pharma Oy Vegf-c及ccbe1之治療用途
GB201900702D0 (en) * 2019-01-18 2019-03-06 Univ Bristol Therapy

Also Published As

Publication number Publication date
JP2024509316A (ja) 2024-02-29
WO2022189811A1 (en) 2022-09-15
DK4305181T3 (en) 2025-12-15
IL305714A (en) 2023-11-01
AU2022235209A1 (en) 2023-10-05
EP4305181B1 (en) 2025-11-05
EP4722371A2 (en) 2026-04-08
FI4305181T3 (fi) 2025-12-12
ES3056304T3 (en) 2026-02-19
BR112023018362A2 (pt) 2023-12-05
HRP20251561T1 (hr) 2026-02-13
PT4305181T (pt) 2025-12-04
PL4305181T3 (pl) 2026-01-26
US20250283072A1 (en) 2025-09-11
EP4305181A1 (en) 2024-01-17
CA3211566A1 (en) 2022-09-15
EP4722371A3 (en) 2026-06-17
CN117377770A (zh) 2024-01-09
GB202103470D0 (en) 2021-04-28
MX2023010573A (es) 2023-11-03

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PA0105 International application

Patent event date: 20231010

Patent event code: PA01051R01D

Comment text: International Patent Application

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A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20250312

Comment text: Request for Examination of Application