WO2023006890A1 - Amélioration de l'efficacité d'une thérapie génique ciblant les muscles - Google Patents

Amélioration de l'efficacité d'une thérapie génique ciblant les muscles Download PDF

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Publication number
WO2023006890A1
WO2023006890A1 PCT/EP2022/071225 EP2022071225W WO2023006890A1 WO 2023006890 A1 WO2023006890 A1 WO 2023006890A1 EP 2022071225 W EP2022071225 W EP 2022071225W WO 2023006890 A1 WO2023006890 A1 WO 2023006890A1
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dph
cre
nucleic acid
vector
acid expression
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PCT/EP2022/071225
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English (en)
Inventor
Thierry Vandendriessche
Lay Khim Chuah
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Vrije Universiteit Brussel
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Publication of WO2023006890A1 publication Critical patent/WO2023006890A1/fr

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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
    • C12N15/63—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
    • 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
    • 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
    • C07K14/4707—Muscular dystrophy
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La présente invention concerne des éléments régulateurs d'acide nucléique qui sont capables d'améliorer l'expression de gènes spécifiquement à une membrane, en particulier l'expression dans un diaphragme seule, ou en combinaison avec l'expression dans le muscle cardiaque et/ou le muscle squelettique, des procédés faisant appel à ces éléments régulateurs et des utilisations de ces éléments. L'invention concerne également des cassettes d'expression et des vecteurs contenant ces éléments régulateurs d'acide nucléique. La présente invention est particulièrement utile pour des applications faisant appel à une thérapie génique, plus particulièrement une thérapie génique ciblant le tissu musculaire et/ou cardiaque, et à des fins de vaccination.
PCT/EP2022/071225 2021-07-28 2022-07-28 Amélioration de l'efficacité d'une thérapie génique ciblant les muscles WO2023006890A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP21188306.1 2021-07-28
EP21188306 2021-07-28

Publications (1)

Publication Number Publication Date
WO2023006890A1 true WO2023006890A1 (fr) 2023-02-02

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ID=77398387

Family Applications (1)

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PCT/EP2022/071225 WO2023006890A1 (fr) 2021-07-28 2022-07-28 Amélioration de l'efficacité d'une thérapie génique ciblant les muscles

Country Status (3)

Country Link
AR (1) AR126660A1 (fr)
TW (1) TW202310880A (fr)
WO (1) WO2023006890A1 (fr)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023108129A1 (fr) * 2021-12-10 2023-06-15 Spacecraft Seven, Llc Thérapie génique pour la troponine c (tnnc1) à l'aide d'un vecteur aav
CN116926125A (zh) * 2023-09-07 2023-10-24 昆明理工大学 一种抑制炎症与基因编辑同时进行的基因载体

Citations (10)

* Cited by examiner, † Cited by third party
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WO2011051450A1 (fr) 2009-10-29 2011-05-05 Vib Vzw Éléments régulateurs de l'acide nucléique spécifique du coeur et procédés et utilisation de ceux-ci
WO2014063753A1 (fr) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vecteurs du facteur ix hyperactif pour thérapie génique ciblée sur le foie de l'hémophilie 'b' et procédés d'utilisation associés
WO2014064277A1 (fr) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vecteur pour thérapie génique ciblée sur le foie de l'hémophilie et procédés associés
WO2015110449A1 (fr) 2014-01-21 2015-07-30 Vrije Universiteit Brussel Éléments régulateurs d'acide nucléique exprimé dans un muscle, méthodes et utilisation associées
WO2016146757A1 (fr) 2015-03-17 2016-09-22 Vrije Universiteit Brussel Systèmes d'expression spécifiques du foie optimisés pour fviii et fix
WO2018178067A1 (fr) 2017-03-27 2018-10-04 Vrije Universiteit Brussel Éléments régulateurs d'acides nucléiques spécifiques au diaphragme et méthodes et utilisation associées
WO2019153009A1 (fr) * 2018-02-05 2019-08-08 Audentes Therapeutics, Inc. Éléments de régulation de la transcription et utilisations associées
WO2020091471A1 (fr) * 2018-10-31 2020-05-07 서울대학교산학협력단 Production de chien transgénique surexprimant la pck1 spécifique du muscle

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009071679A1 (fr) 2007-12-05 2009-06-11 Vib Vzw Nouveau vecteur aav et ses utilisations
WO2009130208A1 (fr) 2008-04-22 2009-10-29 Vib Vzw Eléments régulateurs d’acide nucléique à spécificité hépatique, procédés et utilisations
WO2011051450A1 (fr) 2009-10-29 2011-05-05 Vib Vzw Éléments régulateurs de l'acide nucléique spécifique du coeur et procédés et utilisation de ceux-ci
WO2014063753A1 (fr) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vecteurs du facteur ix hyperactif pour thérapie génique ciblée sur le foie de l'hémophilie 'b' et procédés d'utilisation associés
WO2014064277A1 (fr) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vecteur pour thérapie génique ciblée sur le foie de l'hémophilie et procédés associés
WO2015110449A1 (fr) 2014-01-21 2015-07-30 Vrije Universiteit Brussel Éléments régulateurs d'acide nucléique exprimé dans un muscle, méthodes et utilisation associées
WO2016146757A1 (fr) 2015-03-17 2016-09-22 Vrije Universiteit Brussel Systèmes d'expression spécifiques du foie optimisés pour fviii et fix
WO2018178067A1 (fr) 2017-03-27 2018-10-04 Vrije Universiteit Brussel Éléments régulateurs d'acides nucléiques spécifiques au diaphragme et méthodes et utilisation associées
WO2019153009A1 (fr) * 2018-02-05 2019-08-08 Audentes Therapeutics, Inc. Éléments de régulation de la transcription et utilisations associées
WO2020091471A1 (fr) * 2018-10-31 2020-05-07 서울대학교산학협력단 Production de chien transgénique surexprimant la pck1 spécifique du muscle

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023108129A1 (fr) * 2021-12-10 2023-06-15 Spacecraft Seven, Llc Thérapie génique pour la troponine c (tnnc1) à l'aide d'un vecteur aav
CN116926125A (zh) * 2023-09-07 2023-10-24 昆明理工大学 一种抑制炎症与基因编辑同时进行的基因载体

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TW202310880A (zh) 2023-03-16
AR126660A1 (es) 2023-11-01

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