KR20250006979A - 족세포 특이적 발현을 위한 핵산 작제물 및 벡터 - Google Patents

족세포 특이적 발현을 위한 핵산 작제물 및 벡터 Download PDF

Info

Publication number
KR20250006979A
KR20250006979A KR1020247040008A KR20247040008A KR20250006979A KR 20250006979 A KR20250006979 A KR 20250006979A KR 1020247040008 A KR1020247040008 A KR 1020247040008A KR 20247040008 A KR20247040008 A KR 20247040008A KR 20250006979 A KR20250006979 A KR 20250006979A
Authority
KR
South Korea
Prior art keywords
sequence
gene
promoter
podocyte
nucleotide sequence
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
KR1020247040008A
Other languages
English (en)
Korean (ko)
Inventor
나바로 안나 메세게르
로드리게스 미구엘 칠론
바우티스타 재즈민 파올라 아레발로
곤잘로 페르난데스-미란다
카스텔토르트 마르크 라미스
Original Assignee
펀다시오 호스피탈 유니버시타리 발 드헤브론-인스티튜트 디 레세르카
인스티투시오 카타라나 데 레세르카 아이 에스투디스 아반카츠 (아이크리아)
니네바 테라퓨틱스 에스엘
우니버시타트 아우토노마 데 바르셀로나
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 펀다시오 호스피탈 유니버시타리 발 드헤브론-인스티튜트 디 레세르카, 인스티투시오 카타라나 데 레세르카 아이 에스투디스 아반카츠 (아이크리아), 니네바 테라퓨틱스 에스엘, 우니버시타트 아우토노마 데 바르셀로나 filed Critical 펀다시오 호스피탈 유니버시타리 발 드헤브론-인스티튜트 디 레세르카
Publication of KR20250006979A publication Critical patent/KR20250006979A/ko
Pending legal-status Critical Current

Links

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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • 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
    • 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
    • C12N2830/36—Vector systems having a special element relevant for transcription being a transcription termination element
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020247040008A 2022-05-02 2023-04-28 족세포 특이적 발현을 위한 핵산 작제물 및 벡터 Pending KR20250006979A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP22382421.0A EP4273243A1 (en) 2022-05-02 2022-05-02 Nucleic acid constructs and vectors for podocyte specific expression
EP22382421.0 2022-05-02
PCT/EP2023/061388 WO2023213738A1 (en) 2022-05-02 2023-04-28 Nucleic acid constructs and vectors for podocyte specific expression

Publications (1)

Publication Number Publication Date
KR20250006979A true KR20250006979A (ko) 2025-01-13

Family

ID=82218428

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247040008A Pending KR20250006979A (ko) 2022-05-02 2023-04-28 족세포 특이적 발현을 위한 핵산 작제물 및 벡터

Country Status (8)

Country Link
US (1) US20250288696A1 (enExample)
EP (2) EP4273243A1 (enExample)
JP (1) JP2025515979A (enExample)
KR (1) KR20250006979A (enExample)
CN (1) CN119677849A (enExample)
AU (1) AU2023266069A1 (enExample)
CA (1) CA3256851A1 (enExample)
WO (1) WO2023213738A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201900702D0 (en) * 2019-01-18 2019-03-06 Univ Bristol Therapy
GB202009039D0 (en) 2020-06-15 2020-07-29 Univ Bristol Therapy
GB202304841D0 (en) * 2023-03-31 2023-05-17 Purespring Therapeutics Ltd Promoter
GB202309909D0 (en) 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Regulatory elements
GB202309913D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Polynucleotides, avv vectors, and pharmaceutical compositions for treating nephrotic syndrome
GB202309908D0 (en) * 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases
GB202309914D0 (en) 2023-06-29 2023-08-16 Purespring Therapeutics Ltd Promoters
WO2025168940A1 (en) 2024-02-07 2025-08-14 Purespring Therapeutics Limited Vectors
GB202407863D0 (en) 2024-06-03 2024-07-17 Purespring Therapeutics Ltd Methods
WO2026024176A1 (en) * 2024-07-22 2026-01-29 The Trustees Of The Columbia University In The City Of New York Synthetic reporters for multiplexed detection of transcription factor activity
GB202414499D0 (en) 2024-10-02 2024-11-13 Purespring Therapeutics Ltd Methods

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017153606A1 (en) 2016-03-11 2017-09-14 Fundació Institut De Recerca Biomèdica (Irb Barcelona) Nucleic acid constructs and vectors for oncoselective expression of a transgene
GB201900702D0 (en) 2019-01-18 2019-03-06 Univ Bristol Therapy
EP3914719A4 (en) * 2019-01-24 2023-04-05 Valorisation-HSJ, Limited Partnership Cell-specific transcriptional regulatory sequences and uses thereof
GB202003618D0 (en) 2020-03-12 2020-04-29 Univ Bristol Gene Therapy

Non-Patent Citations (10)

* Cited by examiner, † Cited by third party
Title
Altschul et al., "Basic local alignment search tool", 1990, J. Mol. Biol, v. 215, pages 403-410.
EMBOSS: The European Molecular Biology Open Software Suite, Rice et al., 2000, Trends Genet. 16: 276-277
Harendza et al. 2009
Juven-Gershon et al., "rational design of super core promoter that enhances gene expression" Nature Methods-2006, vol. no. 3(11), pp. 917-922.
Moeller et al. 2003
Needleman and Wunsch, 1970, J. Mol. Biol. 48: 443-453
Pique et al in "A Combinatorial Code for CPE-Mediated Translational Control" Cell 2008, 132(3): 434-48.
WO2017153606 (Fundacio Institut de Recerca Biomedica (IRB Barcelona), et al.)
WO2020148548A1 (The University of Bristol)
WO2021181118A1 (The University of Bristol et al.)

Also Published As

Publication number Publication date
CN119677849A (zh) 2025-03-21
WO2023213738A1 (en) 2023-11-09
AU2023266069A1 (en) 2024-12-19
CA3256851A1 (en) 2023-11-09
EP4519433A1 (en) 2025-03-12
US20250288696A1 (en) 2025-09-18
EP4273243A1 (en) 2023-11-08
JP2025515979A (ja) 2025-05-22

Similar Documents

Publication Publication Date Title
US20250288696A1 (en) Nucleic acid constructs and vectors for podocyte specific expression
US12416018B2 (en) Regulation of gene expression by aptamer-mediated modulation of alternative splicing
US12559757B2 (en) Regulation of gene expression through aptamer-modulated polyadenylation
US20240398990A1 (en) Liver-specific expression cassettes, vectors and uses thereof for expressing therapeutic proteins
HK40059892A (en) Regulation of gene expression by aptamer-mediated modulation of alternative splicing
HK40079456A (en) Regulation of gene expression by aptamer-mediated modulation of alternative splicing
WO2025059540A1 (en) Systems and methods of gene-editing to treat spinal muscular atrophy
WO2024050548A2 (en) Compact promoters for targeting hypoxia induced genes
WO2025019820A1 (en) All in one vectors for the treatment of facioscapulohumeral muscular dystrophy
WO2024182444A9 (en) Compositions and methods for the modification and regulation of liver gene expression
HK40108167A (zh) 用於治疗α-1抗胰蛋白酶缺乏症的组合物及方法
HK1248760B (en) Regulation of gene expression by aptamer-mediated modulation of alternative splicing
HK40015272B (zh) 用於治疗α-1抗胰蛋白酶缺乏症的组合物及方法
HK40001465B (en) Regulation of gene expression through aptamer-modulated polyadenylation
HK40001465A (en) Regulation of gene expression through aptamer-modulated polyadenylation
WO2025194082A1 (en) Compositions and methods for delivering nucleic acids to podocytes

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-EXM-PA0201 (AS PROVIDED BY THE NATIONAL OFFICE)

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201