CL2023001876A1 - Proteínas de la cápside viral con especificidad para las células del tejido cardiaco - Google Patents

Proteínas de la cápside viral con especificidad para las células del tejido cardiaco

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Publication number
CL2023001876A1
CL2023001876A1 CL2023001876A CL2023001876A CL2023001876A1 CL 2023001876 A1 CL2023001876 A1 CL 2023001876A1 CL 2023001876 A CL2023001876 A CL 2023001876A CL 2023001876 A CL2023001876 A CL 2023001876A CL 2023001876 A1 CL2023001876 A1 CL 2023001876A1
Authority
CL
Chile
Prior art keywords
primate
viral
cardiac
cells
cardiac tissue
Prior art date
Application number
CL2023001876A
Other languages
English (en)
Inventor
Lamla Thorsten
BLAZEVIC Dragica
Michelfelder Stefan
Düchs Matthias
Kreuz Sebastian
Sauer Achim
Meier Florian
Stierstorfer Birgit
Christoph Wollert Kai
Korf-Klingebiel Mortimer
Original Assignee
Boehringer Ingelheim Int
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 Boehringer Ingelheim Int filed Critical Boehringer Ingelheim Int
Publication of CL2023001876A1 publication Critical patent/CL2023001876A1/es

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • 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
    • A01K2207/00—Modified animals
    • A01K2207/30—Animals modified by surgical methods
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0375—Animal model for cardiovascular diseases
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14145—Special targeting system for 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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)
  • Peptides Or Proteins (AREA)

Abstract

La presente invención se refiere en general al campo de la terapia génica somática mediante el uso de vectores virales, y en particular vectores de virus adenoasociados (AAV), para el tratamiento de enfermedades hereditarias o adquiridas. Más específicamente, la invención se refiere a una proteína de la cápside viral que proporciona una transducción específica de células endoteliales murinas para tratar o prevenir una enfermedad cardiaca en un primate. Se comprobó que la proteína de la cápside viral se une específicamente a las células del tejido cardíaco de primates, y en particular a las células del músculo cardíaco de primates, y puede utilizarse para proporcionar una transducción eficiente y selectiva de los cardiomiocitos de primates y garantizar la expresión específica del tejido cardíaco de uno o más transgenes en el primate. La invención se refiere además a un vector viral recombinante, preferentemente un vector AAV, que comprende una cápside con al menos un transgén empaquetado en la cápside. El vector viral es adecuado para el tratamiento terapéutico de un trastorno o enfermedad cardíaca en un primate. La invención se refiere además a células y composiciones farmacéuticas que comprenden el vector viral según la invención.
CL2023001876A 2020-12-23 2023-06-22 Proteínas de la cápside viral con especificidad para las células del tejido cardiaco CL2023001876A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP20217171 2020-12-23
EP21171861 2021-05-03

Publications (1)

Publication Number Publication Date
CL2023001876A1 true CL2023001876A1 (es) 2024-01-26

Family

ID=80685029

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2023001876A CL2023001876A1 (es) 2020-12-23 2023-06-22 Proteínas de la cápside viral con especificidad para las células del tejido cardiaco

Country Status (9)

Country Link
EP (1) EP4267594A1 (es)
JP (1) JP2024501821A (es)
KR (1) KR20230129245A (es)
AU (1) AU2021405790A1 (es)
CA (1) CA3202675A1 (es)
CL (1) CL2023001876A1 (es)
IL (1) IL303891A (es)
MX (1) MX2023007474A (es)
WO (1) WO2022136655A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2021218411A1 (en) 2020-02-13 2022-10-06 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
WO2024130070A2 (en) * 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3747457A3 (en) 2013-01-17 2021-03-03 Medizinische Hochschule Hannover Factor 1 protein, factor 2 protein and inhibitors thereof for use in treating or preventing diseases
DE102013215817A1 (de) 2013-08-09 2015-02-12 Universitätsklinikum Hamburg-Eppendorf Neue peptide mit spezifität für die lunge
DE102014207498A1 (de) 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
EP3662060A2 (en) * 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2019253700A1 (en) 2018-04-09 2020-11-26 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
CN115698048A (zh) 2020-01-21 2023-02-03 勃林格殷格翰国际有限公司 用于治疗或预防纤维化、肥大或心力衰竭的髓源性生长因子

Also Published As

Publication number Publication date
EP4267594A1 (en) 2023-11-01
CA3202675A1 (en) 2022-06-30
WO2022136655A1 (en) 2022-06-30
KR20230129245A (ko) 2023-09-07
IL303891A (en) 2023-08-01
AU2021405790A1 (en) 2023-07-06
MX2023007474A (es) 2023-09-13
JP2024501821A (ja) 2024-01-16

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