MX2015009963A - Metodos y composición farmacéutica para el tratamiento y la prevención de cardiomiopatía debido a falta de energía. - Google Patents

Metodos y composición farmacéutica para el tratamiento y la prevención de cardiomiopatía debido a falta de energía.

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Publication number
MX2015009963A
MX2015009963A MX2015009963A MX2015009963A MX2015009963A MX 2015009963 A MX2015009963 A MX 2015009963A MX 2015009963 A MX2015009963 A MX 2015009963A MX 2015009963 A MX2015009963 A MX 2015009963A MX 2015009963 A MX2015009963 A MX 2015009963A
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MX
Mexico
Prior art keywords
energy failure
subject
prevention
treatment
methods
Prior art date
Application number
MX2015009963A
Other languages
English (en)
Inventor
Hélène Moniique Puccio
Patrick Aubourg
Ronald G Crystal
Pierre Bougneres
Original Assignee
Inserm Inst Nat De La Santé Et De La Rech Médicale
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Filing date
Publication date
Application filed by Inserm Inst Nat De La Santé Et De La Rech Médicale filed Critical Inserm Inst Nat De La Santé Et De La Rech Médicale
Publication of MX2015009963A publication Critical patent/MX2015009963A/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • 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
    • A61K48/0016—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 nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2750/14032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • 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
    • C12N2750/14071—Demonstrated in vivo effect
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    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y116/00—Oxidoreductases oxidizing metal ions (1.16)
    • C12Y116/03—Oxidoreductases oxidizing metal ions (1.16) with oxygen as acceptor (1.16.3)
    • C12Y116/03001—Ferroxidase (1.16.3.1), i.e. ceruloplasmin

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

Abstract

La presente invención se refiere a un método para prevenir o tratar cardiomiopatía debido a falta de energía en un paciente que lo necesita, dicho método comprende administrar a dicho paciente una cantidad efectiva terapéuticamente de un vector que contiene una secuencia de ácido nucleico de un gen que puede restaurar la falta de energía. Mas particularmente, la invención se refiere a un método para prevenir o tratar una cardiomiopatía asociada con ataxia de Friedreich en un paciente que lo necesita, dicho método comprende administrar a dicho paciente una cantidad efectiva terapéuticamente de un vector que contiene un ácido nucleico que codifica una frataxina (FXN).
MX2015009963A 2013-02-01 2014-01-31 Metodos y composición farmacéutica para el tratamiento y la prevención de cardiomiopatía debido a falta de energía. MX2015009963A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US13/756,651 US9066966B2 (en) 2013-02-01 2013-02-01 Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
PCT/EP2014/051966 WO2014118346A1 (en) 2013-02-01 2014-01-31 Methods and pharmaceutical composition for the treatment and the prevention of cardiomyopathy due to energy failure

Publications (1)

Publication Number Publication Date
MX2015009963A true MX2015009963A (es) 2016-03-04

Family

ID=50033540

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2015009963A MX2015009963A (es) 2013-02-01 2014-01-31 Metodos y composición farmacéutica para el tratamiento y la prevención de cardiomiopatía debido a falta de energía.
MX2021000533A MX2021000533A (es) 2013-02-01 2015-07-31 Composiciones farmaceuticas y uso de las mismas para la prevencion y el tratamiento de cardiomiopatia asociada a la ataxia de friedreich.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2021000533A MX2021000533A (es) 2013-02-01 2015-07-31 Composiciones farmaceuticas y uso de las mismas para la prevencion y el tratamiento de cardiomiopatia asociada a la ataxia de friedreich.

Country Status (10)

Country Link
US (7) US9066966B2 (es)
EP (2) EP4324845A3 (es)
AU (5) AU2014211315A1 (es)
BR (1) BR112015018255A2 (es)
CA (1) CA2900135A1 (es)
EA (1) EA201500802A1 (es)
IL (2) IL240311A0 (es)
MX (2) MX2015009963A (es)
WO (1) WO2014118346A1 (es)
ZA (2) ZA201506093B (es)

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JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7128491B2 (ja) * 2017-10-17 2022-08-31 フンダシオ インスティテュート ディンベスティガシオ エン シエンシエス デ ラ サリュ ジャーマンス トライアス アイ プジョル フリードライヒ運動失調症の治療用のベクター
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SG11202012044QA (en) * 2018-06-29 2021-01-28 Wuhan Neurophth Biological Tech Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
MX2022004352A (es) 2019-10-17 2022-07-19 Stridebio Inc Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.
WO2023150563A1 (en) * 2022-02-01 2023-08-10 Cornell University Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy associated with friedreich ataxia

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Also Published As

Publication number Publication date
CA2900135A1 (en) 2014-08-07
US20220090128A1 (en) 2022-03-24
US10337027B2 (en) 2019-07-02
US20160024526A1 (en) 2016-01-28
US20180334687A1 (en) 2018-11-22
US20200157564A1 (en) 2020-05-21
AU2018202783A1 (en) 2018-05-24
NZ710497A (en) 2021-01-29
US20150313969A1 (en) 2015-11-05
AU2020200408A1 (en) 2020-02-13
ZA201703041B (en) 2023-12-20
ZA201506093B (en) 2023-12-20
MX2021000533A (es) 2021-04-19
AU2024201048A1 (en) 2024-03-14
EP4324845A3 (en) 2024-04-17
IL289016A (en) 2022-02-01
US9066966B2 (en) 2015-06-30
US20210340565A1 (en) 2021-11-04
AU2014211315A1 (en) 2015-08-13
EP2950824A1 (en) 2015-12-09
EA201500802A1 (ru) 2016-01-29
US20140221462A1 (en) 2014-08-07
WO2014118346A1 (en) 2014-08-07
AU2021282480A1 (en) 2022-01-06
BR112015018255A2 (pt) 2017-08-22
IL240311A0 (en) 2015-09-24
EP4324845A2 (en) 2024-02-21

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