BR112022006530A2 - Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c - Google Patents

Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c

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Publication number
BR112022006530A2
BR112022006530A2 BR112022006530A BR112022006530A BR112022006530A2 BR 112022006530 A2 BR112022006530 A2 BR 112022006530A2 BR 112022006530 A BR112022006530 A BR 112022006530A BR 112022006530 A BR112022006530 A BR 112022006530A BR 112022006530 A2 BR112022006530 A2 BR 112022006530A2
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Prior art keywords
adeno
treatment
associated viral
niemann
viral vectors
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BR112022006530A
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English (en)
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Mccoy Daniel
Berry Garrett
DISMUKE David
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Stridebio Inc
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Publication date
Application filed by Stridebio Inc filed Critical Stridebio Inc
Publication of BR112022006530A2 publication Critical patent/BR112022006530A2/pt

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    • 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
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    • 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
    • A61K48/0041—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 the non-active part being polymeric
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C—CHEMISTRY; METALLURGY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14044—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
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  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

VETORES VIRAIS ADENOASSOCIADOS PARA O TRATAMENTO DA DOENÇA DE NIEMANN-PICK TIPO C. A presente invenção refere-se a composições e métodos de terapia de genes para o tratamento, a prevenção e/ou a cura de NPC1. Mais especificamente, a invenção refere-se a vetores virais adenoassociados (AAV) para a aplicação de ácidos nucleicos, e ácidos nucleicos (incluindo cassetes de transferência de AAV) para o tratamento, a prevenção e/ou a cura de NPC1.
BR112022006530A 2019-10-17 2020-10-16 Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c BR112022006530A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962916749P 2019-10-17 2019-10-17
US201962923253P 2019-10-18 2019-10-18
US202063082425P 2020-09-23 2020-09-23
US202063082899P 2020-09-24 2020-09-24
PCT/US2020/056031 WO2021076925A1 (en) 2019-10-17 2020-10-16 Adeno-associated viral vectors for treatment of niemann-pick disease type c

Publications (1)

Publication Number Publication Date
BR112022006530A2 true BR112022006530A2 (pt) 2022-07-05

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BR112022006530A BR112022006530A2 (pt) 2019-10-17 2020-10-16 Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c

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US (2) US20230392163A1 (pt)
EP (1) EP4045525A1 (pt)
JP (1) JP2022551739A (pt)
KR (1) KR20220083714A (pt)
CN (1) CN114787180A (pt)
AU (1) AU2020367532A1 (pt)
BR (1) BR112022006530A2 (pt)
CA (1) CA3157700A1 (pt)
CL (1) CL2022000939A1 (pt)
CO (1) CO2022004649A2 (pt)
IL (1) IL292297A (pt)
MX (1) MX2022004352A (pt)
TW (1) TW202128736A (pt)
WO (1) WO2021076925A1 (pt)

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WO2023230579A2 (en) * 2022-05-25 2023-11-30 Metagenomi, Inc. Supplementation of liver enzyme expression

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