BR112022024708A2 - Ácido nucleico otimizado por códon que codifica a proteína smn1, cassete de expressão, vetor de expressão, vírus recombinante baseado em aav9 e seu uso e composição farmacêutica para entregar o gene smn1 a células alvo - Google Patents

Ácido nucleico otimizado por códon que codifica a proteína smn1, cassete de expressão, vetor de expressão, vírus recombinante baseado em aav9 e seu uso e composição farmacêutica para entregar o gene smn1 a células alvo

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Publication number
BR112022024708A2
BR112022024708A2 BR112022024708A BR112022024708A BR112022024708A2 BR 112022024708 A2 BR112022024708 A2 BR 112022024708A2 BR 112022024708 A BR112022024708 A BR 112022024708A BR 112022024708 A BR112022024708 A BR 112022024708A BR 112022024708 A2 BR112022024708 A2 BR 112022024708A2
Authority
BR
Brazil
Prior art keywords
smn1
aav9
nucleic acid
codon
acid encoding
Prior art date
Application number
BR112022024708A
Other languages
English (en)
Inventor
Dmitriy Aleksandrovich Madera
Pavel Mikhailovich Gershovich
Anna Sergeevna Veselova
Tatiana Evgenievna Shugaeva
Maria Andreevna Lomunova
Margarita Aleksandrovna Shkliaeva
Dmitry Valentinovich Morozov
Original Assignee
Biocad Joint Stock Co
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 Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of BR112022024708A2 publication Critical patent/BR112022024708A2/pt

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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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

Abstract

ÁCIDO NUCLEICO OTIMIZADO POR CÓDON QUE CODIFICA A PROTEÍNA SMN1, CASSETE DE EXPRESSÃO, VETOR DE EXPRESSÃO, VÍRUS RECOMBINANTE BASEADO EM AAV9 E SEU USO E COMPOSIÇÃO FARMACÊUTICA PARA ENTREGAR O GENE SMN1 A CÉLULAS ALVO. O presente pedido refere-se aos campos da genética, terapia gênica e biologia molecular. Mais especificamente, a presente invenção refere-se a um ácido nucleico otimizado por códon isolado que codifica a proteína SMN (proteína de sobrevivência do neurônio motor), um cassete de expressão e um vetor baseado nele, bem como um vírus recombinante baseado em AAV9 (vírus adeno-associado sorotipo 9) para aumentar a expressão do gene SMN1 em células alvo e seu uso.
BR112022024708A 2020-06-02 2021-06-02 Ácido nucleico otimizado por códon que codifica a proteína smn1, cassete de expressão, vetor de expressão, vírus recombinante baseado em aav9 e seu uso e composição farmacêutica para entregar o gene smn1 a células alvo BR112022024708A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2020118148A RU2742837C1 (ru) 2020-06-02 2020-06-02 Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
PCT/RU2021/000238 WO2021246909A1 (ru) 2020-06-02 2021-06-02 Кодон-оптимизированная нуклеиновая кислота, кодирующая белок smn1

Publications (1)

Publication Number Publication Date
BR112022024708A2 true BR112022024708A2 (pt) 2023-04-11

Family

ID=74665909

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022024708A BR112022024708A2 (pt) 2020-06-02 2021-06-02 Ácido nucleico otimizado por códon que codifica a proteína smn1, cassete de expressão, vetor de expressão, vírus recombinante baseado em aav9 e seu uso e composição farmacêutica para entregar o gene smn1 a células alvo

Country Status (21)

Country Link
US (1) US20230212609A1 (pt)
EP (1) EP4159863A1 (pt)
JP (1) JP2023529855A (pt)
KR (1) KR20230019162A (pt)
CN (1) CN116234914A (pt)
AR (1) AR122500A1 (pt)
AU (1) AU2021282898A1 (pt)
BR (1) BR112022024708A2 (pt)
CA (1) CA3181288A1 (pt)
CL (1) CL2022003428A1 (pt)
CO (1) CO2022017334A2 (pt)
CR (1) CR20220619A (pt)
EC (1) ECSP22092206A (pt)
IL (1) IL298772A (pt)
MA (1) MA58655B1 (pt)
MX (1) MX2022015231A (pt)
PE (1) PE20240079A1 (pt)
RU (1) RU2742837C1 (pt)
TW (1) TW202214862A (pt)
UY (1) UY39245A (pt)
WO (1) WO2021246909A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117210457A (zh) * 2022-06-10 2023-12-12 拜奥卡德联合股份公司 具有启动子活性的核酸及其用途
WO2023246734A1 (en) * 2022-06-21 2023-12-28 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of sma disease

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4399216A (en) 1980-02-25 1983-08-16 The Trustees Of Columbia University Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
US5179017A (en) 1980-02-25 1993-01-12 The Trustees Of Columbia University In The City Of New York Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
US4634665A (en) 1980-02-25 1987-01-06 The Trustees Of Columbia University In The City Of New York Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
DE3431140A1 (de) 1984-08-24 1986-03-06 Behringwerke Ag, 3550 Marburg Enhancer fuer eukaryotische expressionssysteme
GB8717430D0 (en) 1987-07-23 1987-08-26 Celltech Ltd Recombinant dna product
RU2312896C2 (ru) * 2001-09-20 2007-12-20 Глаксо Груп Лимитед Последовательность нуклеиновой кислоты, кодирующая белок gag вич-1, способ получения указанной последовательности, вектор, содержащий ее, белок, кодируемый ею, фармацевтическая композиция и их применение для профилактики и/или лечения вич-инфекции и спида
BR112018011975A2 (pt) * 2015-12-14 2018-12-11 Univ Pennsylvania composições úteis no tratamento de atrofia muscular espinhal

Also Published As

Publication number Publication date
CL2022003428A1 (es) 2023-06-09
MA58655A1 (fr) 2023-06-28
MA58655B1 (fr) 2023-12-29
PE20240079A1 (es) 2024-01-16
US20230212609A1 (en) 2023-07-06
MX2022015231A (es) 2023-02-09
CN116234914A (zh) 2023-06-06
CA3181288A1 (en) 2021-12-09
AU2021282898A1 (en) 2023-02-02
AR122500A1 (es) 2022-09-14
CR20220619A (es) 2023-06-14
WO2021246909A1 (ru) 2021-12-09
RU2742837C1 (ru) 2021-02-11
UY39245A (es) 2021-12-31
ECSP22092206A (es) 2023-01-31
TW202214862A (zh) 2022-04-16
JP2023529855A (ja) 2023-07-12
IL298772A (en) 2023-02-01
KR20230019162A (ko) 2023-02-07
CO2022017334A2 (es) 2023-04-17
EP4159863A1 (en) 2023-04-05

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