AU2023266069A1 - Nucleic acid constructs and vectors for podocyte specific expression - Google Patents

Nucleic acid constructs and vectors for podocyte specific expression Download PDF

Info

Publication number
AU2023266069A1
AU2023266069A1 AU2023266069A AU2023266069A AU2023266069A1 AU 2023266069 A1 AU2023266069 A1 AU 2023266069A1 AU 2023266069 A AU2023266069 A AU 2023266069A AU 2023266069 A AU2023266069 A AU 2023266069A AU 2023266069 A1 AU2023266069 A1 AU 2023266069A1
Authority
AU
Australia
Prior art keywords
gene
sequence
promoter
seq
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
AU2023266069A
Other languages
English (en)
Inventor
Jazmine Paola Arévalo Bautista
Miguel Chillon Rodriguez
Gonzalo FERNÁNDEZ-MIRANDA
Anna MESEGUER NAVARRO
Marc Ramis Castelltort
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.)
Ninevah Therapeutics Sl
Universitat Autonòma de Barcelona
Institució Catalana de Recerca i Estudis Avançats
Fundació Institut de Recerca Hospital Universitari Vall d'Hebron
Original Assignee
Fundacio Hospital Univ Vall D'hebron Institut De Recerca
Ninevah Therapeutics Sl
Universitat Autonòma de Barcelona
Institució Catalana de Recerca i Estudis Avançats
Fundació Institut de Recerca Hospital Universitari Vall d'Hebron
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 Fundacio Hospital Univ Vall D'hebron Institut De Recerca, Ninevah Therapeutics Sl, Universitat Autonòma de Barcelona, Institució Catalana de Recerca i Estudis Avançats, Fundació Institut de Recerca Hospital Universitari Vall d'Hebron filed Critical Fundacio Hospital Univ Vall D'hebron Institut De Recerca
Publication of AU2023266069A1 publication Critical patent/AU2023266069A1/en
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)
AU2023266069A 2022-05-02 2023-04-28 Nucleic acid constructs and vectors for podocyte specific expression Pending AU2023266069A1 (en)

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
AU2023266069A1 true AU2023266069A1 (en) 2024-12-19

Family

ID=82218428

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2023266069A Pending AU2023266069A1 (en) 2022-05-02 2023-04-28 Nucleic acid constructs and vectors for podocyte specific expression

Country Status (8)

Country Link
US (1) US20250288696A1 (cg-RX-API-DMAC7.html)
EP (2) EP4273243A1 (cg-RX-API-DMAC7.html)
JP (1) JP2025515979A (cg-RX-API-DMAC7.html)
KR (1) KR20250006979A (cg-RX-API-DMAC7.html)
CN (1) CN119677849A (cg-RX-API-DMAC7.html)
AU (1) AU2023266069A1 (cg-RX-API-DMAC7.html)
CA (1) CA3256851A1 (cg-RX-API-DMAC7.html)
WO (1) WO2023213738A1 (cg-RX-API-DMAC7.html)

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
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
AU2025218305A1 (en) 2024-02-07 2026-07-30 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
WO2020150832A1 (en) * 2019-01-24 2020-07-30 Valorisation-Hsj, Limited Partnership Cell-specific transcriptional regulatory sequences and uses thereof
GB202003618D0 (en) 2020-03-12 2020-04-29 Univ Bristol Gene Therapy

Also Published As

Publication number Publication date
EP4273243A1 (en) 2023-11-08
EP4519433A1 (en) 2025-03-12
KR20250006979A (ko) 2025-01-13
US20250288696A1 (en) 2025-09-18
JP2025515979A (ja) 2025-05-22
CA3256851A1 (en) 2023-11-09
CN119677849A (zh) 2025-03-21
WO2023213738A1 (en) 2023-11-09

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
KR102599909B1 (ko) 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
KR102527259B1 (ko) 스터퍼/필러 폴리누클레오티드 서열을 포함하는 벡터 및 사용 방법
JP6373763B2 (ja) Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法
US12559757B2 (en) Regulation of gene expression through aptamer-modulated polyadenylation
CA3103963A1 (en) Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
CN120641126A (zh) 合成单链dna分子及其产生和使用方法
US20240398990A1 (en) Liver-specific expression cassettes, vectors and uses thereof for expressing therapeutic proteins
JP2024519733A (ja) Ｄｋｃ１を使用するｒｎａ編集の組成物、系、及び方法
US20250049961A1 (en) Scalable and high-purity cell-free synthesis of closed-ended dna vectors
WO2025119204A1 (en) Methods and compositions for improved readthrough of pseudouridine-modified codons
JP2022548320A (ja) アポリポタンパク質ｂ（ａｐｏｂ）遺伝子発現をモジュレートするための組成物および方法
WO2025019820A1 (en) All in one vectors for the treatment of facioscapulohumeral muscular dystrophy
WO2025059540A1 (en) Systems and methods of gene-editing to treat spinal muscular atrophy
WO2023168000A1 (en) Compositions and methods for treatment of angelman syndrome
HK40059892A (en) Regulation of gene expression by aptamer-mediated modulation of alternative splicing
EP3678709A1 (en) Method and composition for treating neuropathic pain
HK1248760B (en) Regulation of gene expression by aptamer-mediated modulation of alternative splicing
HK40015272B (en) Compositions and methods for treating alpha-1 antitrypsin deficiency