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BR112021011143A2 - Composições para redução específica de drg da expressão de transgene - Google Patents

Composições para redução específica de drg da expressão de transgene

Info

Publication number
BR112021011143A2
BR112021011143A2 BR112021011143A BR112021011143A BR112021011143A2 BR 112021011143 A2 BR112021011143 A2 BR 112021011143A2 BR 112021011143 A BR112021011143 A BR 112021011143A BR 112021011143 A BR112021011143 A BR 112021011143A BR 112021011143 A2 BR112021011143 A2 BR 112021011143A2
Authority
BR
Brazil
Prior art keywords
drg
expression
cells
compositions
aav
Prior art date
Application number
BR112021011143A
Other languages
English (en)
Inventor
M Wilson James
Juliette Hordeaux
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of BR112021011143A2 publication Critical patent/BR112021011143A2/pt

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

composições para redução específica de drg da expressão de transgene. a presente invenção refere-se a um aav recombinante (ra-av) compreendendo um capsídeo de aav e um genoma de vetor empacotado nele, em que o genoma do vetor compreende uma repetição terminal invertida (itr) 5' de aav, uma sequência de ácido nucleico modificada que codifica um produto genético para expressão em células-alvo e sequências-alvo de mirna que reprimem seletivamente a expressão em células do gânglio da raiz dorsal (drg). também é fornecida uma composição farmacêutica compreendendo um raav como descrito aqui em um tampão de formulação e um método de tratamento de um sujeito humano com terapia genética direcionada a cns, enquanto evita seletivamente a expressão em células drg.
BR112021011143A 2018-12-21 2019-12-20 Composições para redução específica de drg da expressão de transgene BR112021011143A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862783956P 2018-12-21 2018-12-21
US201962924970P 2019-10-23 2019-10-23
US201962934915P 2019-11-13 2019-11-13
PCT/US2019/067872 WO2020132455A1 (en) 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression

Publications (1)

Publication Number Publication Date
BR112021011143A2 true BR112021011143A2 (pt) 2022-01-25

Family

ID=71100588

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021011143A BR112021011143A2 (pt) 2018-12-21 2019-12-20 Composições para redução específica de drg da expressão de transgene

Country Status (17)

Country Link
US (1) US20210077553A1 (pt)
EP (1) EP3908326A4 (pt)
JP (1) JP7660062B2 (pt)
KR (1) KR20210107037A (pt)
CN (1) CN113646005A (pt)
AU (1) AU2019401314A1 (pt)
BR (1) BR112021011143A2 (pt)
CA (1) CA3123600A1 (pt)
CL (1) CL2021001624A1 (pt)
CO (1) CO2021008538A2 (pt)
IL (1) IL284185B2 (pt)
JO (1) JOP20210160A1 (pt)
MX (1) MX2021007600A (pt)
PE (1) PE20211581A1 (pt)
PH (1) PH12021551341A1 (pt)
SG (1) SG11202105907QA (pt)
WO (1) WO2020132455A1 (pt)

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Publication number Publication date
IL284185B2 (en) 2025-04-01
EP3908326A1 (en) 2021-11-17
US20210077553A1 (en) 2021-03-18
PE20211581A1 (es) 2021-08-17
JP7660062B2 (ja) 2025-04-10
CL2021001624A1 (es) 2021-11-26
IL284185B1 (en) 2024-12-01
MX2021007600A (es) 2021-08-11
EP3908326A4 (en) 2022-10-26
KR20210107037A (ko) 2021-08-31
CO2021008538A2 (es) 2021-07-19
CN113646005A (zh) 2021-11-12
CA3123600A1 (en) 2020-06-25
JOP20210160A1 (ar) 2023-01-30
IL284185A (en) 2021-08-31
AU2019401314A1 (en) 2021-06-24
JP2022517174A (ja) 2022-03-07
WO2020132455A1 (en) 2020-06-25
PH12021551341A1 (en) 2021-12-13
SG11202105907QA (en) 2021-07-29

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