EA202191418A1 - Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения - Google Patents

Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения

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Publication number
EA202191418A1
EA202191418A1 EA202191418A EA202191418A EA202191418A1 EA 202191418 A1 EA202191418 A1 EA 202191418A1 EA 202191418 A EA202191418 A EA 202191418A EA 202191418 A EA202191418 A EA 202191418A EA 202191418 A1 EA202191418 A1 EA 202191418A1
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EA
Eurasian Patent Office
Prior art keywords
nucleic acids
viral vectors
preparation
recombinant viral
recombinant adeno
Prior art date
Application number
EA202191418A
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English (en)
Inventor
Дарби Томас
Девид Дисмук
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Страйдбайо, Инк.
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Publication date
Application filed by Страйдбайо, Инк. filed Critical Страйдбайо, Инк.
Publication of EA202191418A1 publication Critical patent/EA202191418A1/ru

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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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

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

Abstract

В данном документе описаны нуклеиновые кислоты, кассеты переноса AAV и плазмиды, применяемые при получении рекомбинантных аденоассоциированных вирусных векторов (rAAV - англ.: recombinant adeno-associated virus). Раскрытые нуклеиновые кислоты, кассеты и плазмиды содержат последовательности, которые экспрессируют один или несколько трансгенов, обладающих терапевтической эффективностью в облегчении, лечении и/или профилактике одного или нескольких заболеваний или расстройств.
EA202191418A 2018-11-21 2019-11-21 Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения EA202191418A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862770202P 2018-11-21 2018-11-21
PCT/US2019/062531 WO2020106916A1 (en) 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same

Publications (1)

Publication Number Publication Date
EA202191418A1 true EA202191418A1 (ru) 2021-08-05

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Family Applications (1)

Application Number Title Priority Date Filing Date
EA202191418A EA202191418A1 (ru) 2018-11-21 2019-11-21 Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения

Country Status (20)

Country Link
US (1) US20210324418A1 (ru)
EP (1) EP3883954A4 (ru)
JP (1) JP2022508182A (ru)
KR (1) KR20210103469A (ru)
CN (1) CN113302201A (ru)
AR (1) AR117145A1 (ru)
AU (1) AU2019385506A1 (ru)
BR (1) BR112021009733A2 (ru)
CA (1) CA3120289A1 (ru)
CL (1) CL2021001327A1 (ru)
CO (1) CO2021008120A2 (ru)
EA (1) EA202191418A1 (ru)
EC (1) ECSP21044840A (ru)
IL (1) IL283344A (ru)
MX (1) MX2021005997A (ru)
PE (1) PE20211419A1 (ru)
PH (1) PH12021551155A1 (ru)
SG (1) SG11202105326WA (ru)
TW (1) TW202039533A (ru)
WO (1) WO2020106916A1 (ru)

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Publication number Priority date Publication date Assignee Title
CN107686842A (zh) * 2016-08-03 2018-02-13 南京大学 一种靶多核苷酸编辑方法及其应用
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
US20220010332A1 (en) * 2020-07-08 2022-01-13 Neuracle Genetics Inc. Intron fragments
WO2022026410A2 (en) * 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
WO2022072325A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 combination vector
WO2022072309A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Ascl1 vector
AU2021355362A1 (en) * 2020-09-29 2023-05-11 NeuExcell Therapeutics Inc. Neurod1 vector
WO2023221942A1 (en) * 2022-05-16 2023-11-23 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating glutaric aciduria type i
WO2023240162A1 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2024074143A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090221620A1 (en) * 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
WO2015044704A1 (en) * 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease
EP3245221A4 (en) * 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
WO2016150964A1 (en) * 2015-03-23 2016-09-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
US11896651B2 (en) * 2015-05-16 2024-02-13 Genzyme Corporation Gene editing of deep intronic mutations
RU2021102893A (ru) * 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
RU2742612C2 (ru) * 2015-12-15 2021-02-09 Джензим Корпорейшн Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks

Also Published As

Publication number Publication date
CL2021001327A1 (es) 2021-12-31
MX2021005997A (es) 2021-08-11
AR117145A1 (es) 2021-07-14
US20210324418A1 (en) 2021-10-21
PE20211419A1 (es) 2021-08-03
TW202039533A (zh) 2020-11-01
IL283344A (en) 2021-07-29
EP3883954A1 (en) 2021-09-29
CO2021008120A2 (es) 2021-08-09
KR20210103469A (ko) 2021-08-23
BR112021009733A2 (pt) 2022-01-04
EP3883954A4 (en) 2022-08-10
CN113302201A (zh) 2021-08-24
JP2022508182A (ja) 2022-01-19
PH12021551155A1 (en) 2021-11-03
SG11202105326WA (en) 2021-06-29
AU2019385506A1 (en) 2021-06-03
WO2020106916A1 (en) 2020-05-28
CA3120289A1 (en) 2020-05-28
ECSP21044840A (es) 2021-09-30

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