BR112022019729A2 - Repetição terminal invertida (itr), vetor de ácido nucleico, partícula de vírus adenoassociado recombinante, composição farmacêutica e métodos de entrega de um ácido nucleico e de prevenção ou tratamento de uma doença - Google Patents

Repetição terminal invertida (itr), vetor de ácido nucleico, partícula de vírus adenoassociado recombinante, composição farmacêutica e métodos de entrega de um ácido nucleico e de prevenção ou tratamento de uma doença

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Publication number
BR112022019729A2
BR112022019729A2 BR112022019729A BR112022019729A BR112022019729A2 BR 112022019729 A2 BR112022019729 A2 BR 112022019729A2 BR 112022019729 A BR112022019729 A BR 112022019729A BR 112022019729 A BR112022019729 A BR 112022019729A BR 112022019729 A2 BR112022019729 A2 BR 112022019729A2
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BR
Brazil
Prior art keywords
nucleic acid
methods
associated virus
inverted terminal
recombinant adeno
Prior art date
Application number
BR112022019729A
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English (en)
Inventor
Duan Dongsheng
Pan Xiufang
Yue Yongping
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Univ Missouri
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.)
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Publication date
Application filed by Univ Missouri filed Critical Univ Missouri
Publication of BR112022019729A2 publication Critical patent/BR112022019729A2/pt

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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/117—Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

REPETIÇÃO TERMINAL INVERTIDA (ITR), VETOR DE ÁCIDO NUCLEICO, PARTÍCULA DE VÍRUS ADENOASSOCIADO RECOMBINANTE, COMPOSIÇÃO FARMACÊUTICA E MÉTODOS DE ENTREGA DE UM ÁCIDO NUCLEICO E DE PREVENÇÃO OU TRATAMENTO DE UMA DOENÇA. São divulgados vetores de ácido nucleico de vírus adenoassociado recombinante (rAAV) compreendendo repetições terminais invertidas (ITRs) livres de motivos 5-citosina-fosfato-guanina-3 (CpG). Também são divulgadas partículas de rAAV compreendendo os vetores de rAAV e composições e métodos para distribuir ácidos nucleicos e/ou para terapia gênica. São divulgadas ainda composições e métodos para o tratamento de doenças com terapia gênica de AAV usando o vetor de rAAV.
BR112022019729A 2020-04-07 2021-04-07 Repetição terminal invertida (itr), vetor de ácido nucleico, partícula de vírus adenoassociado recombinante, composição farmacêutica e métodos de entrega de um ácido nucleico e de prevenção ou tratamento de uma doença BR112022019729A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063006148P 2020-04-07 2020-04-07
PCT/US2021/026262 WO2021207415A1 (en) 2020-04-07 2021-04-07 CpG-FREE ITRs FOR AAV GENE THERAPY

Publications (1)

Publication Number Publication Date
BR112022019729A2 true BR112022019729A2 (pt) 2022-11-22

Family

ID=78023894

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022019729A BR112022019729A2 (pt) 2020-04-07 2021-04-07 Repetição terminal invertida (itr), vetor de ácido nucleico, partícula de vírus adenoassociado recombinante, composição farmacêutica e métodos de entrega de um ácido nucleico e de prevenção ou tratamento de uma doença

Country Status (11)

Country Link
US (1) US20230250422A1 (pt)
EP (1) EP4133092A4 (pt)
JP (1) JP2023521090A (pt)
KR (1) KR20220164708A (pt)
CN (1) CN115461465A (pt)
AU (1) AU2021251175A1 (pt)
BR (1) BR112022019729A2 (pt)
CA (1) CA3169945A1 (pt)
CO (1) CO2022014515A2 (pt)
MX (1) MX2022011777A (pt)
WO (1) WO2021207415A1 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023235888A2 (en) * 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
CN117802161A (zh) * 2022-06-30 2024-04-02 苏州吉恒基因科技有限公司 精准重组腺相关病毒载体及其用途
US11993783B1 (en) * 2023-03-27 2024-05-28 Genecraft Inc. Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
WO2014143932A1 (en) * 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
CN108138159A (zh) * 2015-06-23 2018-06-08 费城儿童医院 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途
CN109311932B (zh) * 2016-04-16 2022-05-03 佛罗里达大学研究基金会有限公司 提高杆状病毒系统产生的重组腺相关病毒的生物学效力的方法
KR20210008501A (ko) * 2018-05-09 2021-01-22 바이오마린 파머수티컬 인크. 페닐케톤뇨증을 치료하는 방법
AU2021394878A1 (en) * 2020-12-09 2023-06-22 Scribe Therapeutics Inc. Aav vectors for gene editing

Also Published As

Publication number Publication date
AU2021251175A1 (en) 2022-09-22
CO2022014515A2 (es) 2022-10-21
EP4133092A1 (en) 2023-02-15
US20230250422A1 (en) 2023-08-10
KR20220164708A (ko) 2022-12-13
EP4133092A4 (en) 2024-05-22
CA3169945A1 (en) 2021-10-14
WO2021207415A1 (en) 2021-10-14
CN115461465A (zh) 2022-12-09
JP2023521090A (ja) 2023-05-23
MX2022011777A (es) 2022-10-18

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