BR112022007674A2 - Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav - Google Patents

Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav

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Publication number
BR112022007674A2
BR112022007674A2 BR112022007674A BR112022007674A BR112022007674A2 BR 112022007674 A2 BR112022007674 A2 BR 112022007674A2 BR 112022007674 A BR112022007674 A BR 112022007674A BR 112022007674 A BR112022007674 A BR 112022007674A BR 112022007674 A2 BR112022007674 A2 BR 112022007674A2
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BR
Brazil
Prior art keywords
methods
improve
kits
function
aav vector
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BR112022007674A
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English (en)
Inventor
T Gray John
Cunningham Justine
Rico Salvador
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Audentes Therapeutics Inc
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Publication date
Application filed by Audentes Therapeutics Inc filed Critical Audentes Therapeutics Inc
Publication of BR112022007674A2 publication Critical patent/BR112022007674A2/pt

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
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    • 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
    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • 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
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • 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

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

Abstract

MÉTODOS PARA TRATAR DOENÇA DE POMPE, PARA MELHORAR A FUNÇÃO MUSCULAR, PARA REDUZIR O ACÚMULO DE GLICOGÊNIO, PARA MELHORAR A FUNÇÃO PULMONAR E PARA AUMENTAR A EXPRESSÃO DE GAA, KITS E USOS DE UM VETOR AAV. A presente divulgação se refere a composições e métodos úteis para tratar distúrbios de armazenamento de glicogênio, como distúrbio de armazenamento de glicogênio tipo II, também denominado, no presente documento, doença de Pompe. Usando-se as composições e os métodos da divulgação, um paciente (por exemplo, um paciente mamífero, como um paciente humano) que tem a doença de Pompe pode ser administrado com um vetor viral, como um vetor viral adenoassociado (AAV), que contém um transgene que codifica alfa-glicosidase ácida.
BR112022007674A 2019-10-25 2020-10-23 Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav BR112022007674A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962926282P 2019-10-25 2019-10-25
US202063083349P 2020-09-25 2020-09-25
PCT/US2020/057081 WO2021081338A1 (en) 2019-10-25 2020-10-23 Compositions and methods for treating glycogen storage disorders

Publications (1)

Publication Number Publication Date
BR112022007674A2 true BR112022007674A2 (pt) 2022-08-09

Family

ID=75620302

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022007674A BR112022007674A2 (pt) 2019-10-25 2020-10-23 Métodos para tratar doença de pompe, para melhorar a função muscular, para reduzir o acúmulo de glicogênio, para melhorar a função pulmonar e para aumentar a expressão de gaa, kits e usos de um vetor aav

Country Status (13)

Country Link
US (1) US20220387562A1 (pt)
EP (1) EP4048286A4 (pt)
JP (1) JP2022554141A (pt)
KR (1) KR20220105643A (pt)
CN (1) CN114828858A (pt)
AU (1) AU2020372429A1 (pt)
BR (1) BR112022007674A2 (pt)
CA (1) CA3158281A1 (pt)
CO (1) CO2022006772A2 (pt)
IL (1) IL292401A (pt)
MX (1) MX2022004799A (pt)
TW (1) TW202116359A (pt)
WO (1) WO2021081338A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202342092A (zh) * 2022-02-03 2023-11-01 美商安斯泰來基因治療股份有限公司 用於改良龐貝病治療之組合物及方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3292875T3 (en) * 2012-06-19 2020-08-03 Univ Florida Compositions and methods for treating diseases
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
CN111902539A (zh) * 2018-02-07 2020-11-06 吉尼松公司 杂合调控元件

Also Published As

Publication number Publication date
US20220387562A1 (en) 2022-12-08
IL292401A (en) 2022-06-01
WO2021081338A1 (en) 2021-04-29
AU2020372429A1 (en) 2022-04-28
EP4048286A1 (en) 2022-08-31
TW202116359A (zh) 2021-05-01
CO2022006772A2 (es) 2022-08-09
CN114828858A (zh) 2022-07-29
JP2022554141A (ja) 2022-12-28
MX2022004799A (es) 2022-07-19
CA3158281A1 (en) 2021-04-29
KR20220105643A (ko) 2022-07-27
EP4048286A4 (en) 2023-12-06

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