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AR130561A1 - Composiciones y métodos para el tratamiento de trastornos neuromusculares - Google Patents

Composiciones y métodos para el tratamiento de trastornos neuromusculares

Info

Publication number
AR130561A1
AR130561A1 ARP230102536A ARP230102536A AR130561A1 AR 130561 A1 AR130561 A1 AR 130561A1 AR P230102536 A ARP230102536 A AR P230102536A AR P230102536 A ARP230102536 A AR P230102536A AR 130561 A1 AR130561 A1 AR 130561A1
Authority
AR
Argentina
Prior art keywords
promoter
aav vector
aav
vector
liver tissue
Prior art date
Application number
ARP230102536A
Other languages
English (en)
Inventor
Carlos Fonck
Clarice Weili Chen
Dwaipayan Sen
Original Assignee
Astellas Gene Therapies Inc
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 Astellas Gene Therapies Inc filed Critical Astellas Gene Therapies Inc
Publication of AR130561A1 publication Critical patent/AR130561A1/es

Links

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    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • 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
    • C07K14/775—Apolipopeptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • C07K14/8125—Alpha-1-antitrypsin
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03064—Phosphatidylinositol-3-phosphatase (3.1.3.64)
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente divulgación se refiere a la administración de ácidos nucleicos mediada por virus adenoasociado (AAV) para el tratamiento de trastornos neuromusculares, por ejemplo, la miopatía miotubular ligada al cromosoma X (XLMTM), en pacientes que lo necesitan. Los vectores AAV de la divulgación pueden incluir, por ejemplo, un transgén que codifica una proteína miotubularina unida operablemente a uno o más elementos reguladores de la transcripción. En algunas modalidades, el uno o más elementos reguladores de la transcripción son específicamente activos en el tejido muscular y/o hepático. Reivindicación 1: Un vector viral adenoasociado (AAV) recombinante que comprende un transgén que codifica miotubularina 1 (MTM1), en donde el transgén está enlazado de forma funcional a un promotor que es activo en el tejido hepático. Reivindicación 2: El vector AAV de la reivindicación 1, en donde el promotor es selectivamente activo en el tejido hepático. Reivindicación 8: El vector AAV de cualquiera de las reivindicaciones 3 a 6, en donde las células no hepáticas son células musculares o neuronales. Reivindicación 10: El vector AAV de cualquiera de las reivindicaciones 1 a 9, en donde el promotor comprende un promotor LP1. Reivindicación 14: El vector AAV de cualquiera de las reivindicaciones 1 a 13, en donde el promotor comprende un promotor de apolipoproteína E (ApoE). Reivindicación 18: El vector AAV de cualquiera de las reivindicaciones 1 a 17, en donde el promotor comprende un promotor de a-1-antitrisina (A1AT). Reivindicación 26: El vector AAV de cualquiera de las reivindicaciones 1 a 25, en donde el promotor comprende un promotor constitutivo. Reivindicación 43: Un método para tratar la miopatía miotubular ligada al cromosoma X (XLMTM) en un paciente humano que la necesite, comprendiendo el método administrar al paciente una cantidad terapéuticamente eficaz del vector AAV de cualquiera de las reivindicaciones 1 a 42.
ARP230102536A 2022-09-23 2023-09-22 Composiciones y métodos para el tratamiento de trastornos neuromusculares AR130561A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202263409423P 2022-09-23 2022-09-23

Publications (1)

Publication Number Publication Date
AR130561A1 true AR130561A1 (es) 2024-12-18

Family

ID=90455301

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP230102536A AR130561A1 (es) 2022-09-23 2023-09-22 Composiciones y métodos para el tratamiento de trastornos neuromusculares

Country Status (5)

Country Link
EP (1) EP4590314A2 (es)
JP (1) JP2025531998A (es)
AR (1) AR130561A1 (es)
TW (1) TW202424194A (es)
WO (1) WO2024064895A2 (es)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11072801B2 (en) * 2014-01-21 2021-07-27 Vrije Universiteit Brussel Muscle-specific nucleic acid regulatory elements and methods and use thereof
IL322212A (en) * 2017-08-09 2025-09-01 Bioverativ Therapeutics Inc Nucleic acid molecules and their uses
CA3120014A1 (en) * 2018-11-19 2020-05-28 Uniqure Ip B.V. Liver-specific viral promoters and methods of using the same
CA3210955A1 (en) * 2021-02-09 2022-08-18 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity

Also Published As

Publication number Publication date
EP4590314A2 (en) 2025-07-30
TW202424194A (zh) 2024-06-16
WO2024064895A2 (en) 2024-03-28
WO2024064895A3 (en) 2024-05-10
JP2025531998A (ja) 2025-09-29

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