BR112022025586A2 - Liberação de vetor de vírus adenoassociado para distrofias musculares - Google Patents

Liberação de vetor de vírus adenoassociado para distrofias musculares

Info

Publication number
BR112022025586A2
BR112022025586A2 BR112022025586A BR112022025586A BR112022025586A2 BR 112022025586 A2 BR112022025586 A2 BR 112022025586A2 BR 112022025586 A BR112022025586 A BR 112022025586A BR 112022025586 A BR112022025586 A BR 112022025586A BR 112022025586 A2 BR112022025586 A2 BR 112022025586A2
Authority
BR
Brazil
Prior art keywords
adeno
associated virus
muscular dystrophies
virus vector
release
Prior art date
Application number
BR112022025586A
Other languages
English (en)
Inventor
Rodino-Klapac Louise
R Mendell Jerry
Peterson Ellyn
Potter Rachael
Griffin Danielle
Original Assignee
Res Inst Nationwide Childrens Hospital
Sarepta Therapeutics 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 Res Inst Nationwide Childrens Hospital, Sarepta Therapeutics Inc filed Critical Res Inst Nationwide Childrens Hospital
Publication of BR112022025586A2 publication Critical patent/BR112022025586A2/pt

Links

Classifications

    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2887—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD20
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
  • Ultra Sonic Daignosis Equipment (AREA)

Abstract

LIBERAÇÃO DE VETOR DE VÍRUS ADENOASSOCIADO PARA DISTROFIAS MUSCULARES. A presente invenção refere-se a um método de tratamento de distrofia muscular em um indivíduo em necessidade, compreendendo a administração de um vetor de terapia gênica, tal como vetor do vírus adenoassociado (AAV), expressando um gene de microdistrofina humana miniaturizada em combinação com uma etapa de supressão do sistema imunológico do indivíduo.
BR112022025586A 2020-06-15 2021-06-15 Liberação de vetor de vírus adenoassociado para distrofias musculares BR112022025586A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063039252P 2020-06-15 2020-06-15
US202063083953P 2020-09-27 2020-09-27
US202163160376P 2021-03-12 2021-03-12
US202163188266P 2021-05-13 2021-05-13
PCT/US2021/037470 WO2021257595A1 (en) 2020-06-15 2021-06-15 Adeno-associated virus vector delivery for muscular dystrophies

Publications (1)

Publication Number Publication Date
BR112022025586A2 true BR112022025586A2 (pt) 2023-03-07

Family

ID=77051123

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022025586A BR112022025586A2 (pt) 2020-06-15 2021-06-15 Liberação de vetor de vírus adenoassociado para distrofias musculares

Country Status (13)

Country Link
US (1) US20210393801A1 (pt)
EP (1) EP4164668A1 (pt)
JP (1) JP2023530974A (pt)
KR (1) KR20230035043A (pt)
CN (1) CN116348149A (pt)
AU (1) AU2021293197A1 (pt)
BR (1) BR112022025586A2 (pt)
CA (1) CA3187353A1 (pt)
CO (1) CO2023000156A2 (pt)
IL (1) IL299094A (pt)
MX (1) MX2022016332A (pt)
TW (1) TW202208630A (pt)
WO (1) WO2021257595A1 (pt)

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Publication number Priority date Publication date Assignee Title
CN109121395B (zh) * 2016-04-15 2022-08-09 全国儿童医院研究所 腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗
WO2024006855A1 (en) * 2022-06-30 2024-01-04 Sarepta Therapeutics, Inc. Methods of treating muscular dystrophy
WO2024138158A2 (en) 2022-12-23 2024-06-27 Ultragenyx Pharmaceutical Inc. Method of producing soluble microdystrophin protein and uses of the same

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Also Published As

Publication number Publication date
KR20230035043A (ko) 2023-03-10
CO2023000156A2 (es) 2023-03-27
JP2023530974A (ja) 2023-07-20
US20210393801A1 (en) 2021-12-23
MX2022016332A (es) 2023-04-03
EP4164668A1 (en) 2023-04-19
IL299094A (en) 2023-02-01
CA3187353A1 (en) 2021-12-23
WO2021257595A1 (en) 2021-12-23
AU2021293197A1 (en) 2023-02-09
CN116348149A (zh) 2023-06-27
TW202208630A (zh) 2022-03-01

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