BR112023015177A2 - Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados - Google Patents

Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados

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Publication number
BR112023015177A2
BR112023015177A2 BR112023015177A BR112023015177A BR112023015177A2 BR 112023015177 A2 BR112023015177 A2 BR 112023015177A2 BR 112023015177 A BR112023015177 A BR 112023015177A BR 112023015177 A BR112023015177 A BR 112023015177A BR 112023015177 A2 BR112023015177 A2 BR 112023015177A2
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BR
Brazil
Prior art keywords
nucleic acid
recombinant virus
smn1
mir
pharmaceutical composition
Prior art date
Application number
BR112023015177A
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English (en)
Inventor
Pavel Mikhailovich Gershovich
Dmitriy Aleksandrovich Madera
Dmitry Valentinovich Morozov
Aleksei Sergeevich Siutkin
Anna Sergeevna Veselova
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.)
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Publication date
Priority claimed from RU2021102051A external-priority patent/RU2021102051A/ru
Application filed by Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of BR112023015177A2 publication Critical patent/BR112023015177A2/pt

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    • 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
    • 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
    • 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
    • C12N15/63—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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
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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
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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
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    • C12N2840/00—Vectors comprising a special translation-regulating system

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Epidemiology (AREA)
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  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados. o presente pedido refere-se aos campos de biotecnologia, virologia, genética e biologia molecular. mais especificamente, a presente invenção refere-se a um ácido nucleico isolado para produzir um produto viral de terapia gênica, o referido ácido nucleico isolado compreendendo um ácido nucleico que codifica a proteína smn1 possuindo a sequência de aminoácidos de seq id no: 1 e um ácido nucleico que codifica o microrna mir-23a, um cassete de expressão e um vetor baseado no mesmo, bem como um vírus recombinante baseado em aav9 para expressar o gene smn1 em células alvo, uma composição farmacêutica que inclui o referido vírus recombinante e vários usos do vírus recombinante acima e dos anteriores composição.
BR112023015177A 2021-01-29 2022-01-28 Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados BR112023015177A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2021102051A RU2021102051A (ru) 2021-01-29 Синергетическое действие SMN1 и miR-23a при лечении спинальной мышечной атрофии
PCT/RU2022/000025 WO2022164351A1 (en) 2021-01-29 2022-01-28 Synergistic effect of smn1 and mir-23a in treating spinal muscular atrophy

Publications (1)

Publication Number Publication Date
BR112023015177A2 true BR112023015177A2 (pt) 2023-11-14

Family

ID=82653732

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023015177A BR112023015177A2 (pt) 2021-01-29 2022-01-28 Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados

Country Status (16)

Country Link
US (1) US20240091383A1 (pt)
CN (1) CN117545842A (pt)
AR (1) AR124736A1 (pt)
AU (1) AU2022213262A1 (pt)
BR (1) BR112023015177A2 (pt)
CA (1) CA3206671A1 (pt)
CL (1) CL2023002218A1 (pt)
CO (1) CO2023009633A2 (pt)
CR (1) CR20230363A (pt)
EC (1) ECSP23056133A (pt)
IL (1) IL304612A (pt)
MA (1) MA62178A1 (pt)
MX (1) MX2023008825A (pt)
TW (1) TW202246501A (pt)
UY (1) UY39621A (pt)
WO (1) WO2022164351A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023215384A2 (en) 2022-05-04 2023-11-09 Scholar Rock, Inc. Use of myostatin inhibitor for treating spinal muscular atrophy
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3421603T (pt) * 2009-05-02 2022-01-10 Genzyme Corp Terapia génica para distúrbios neurodegenerativos
WO2017136536A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system

Also Published As

Publication number Publication date
CA3206671A1 (en) 2022-08-04
UY39621A (es) 2022-08-31
IL304612A (en) 2023-09-01
ECSP23056133A (es) 2023-08-31
AR124736A1 (es) 2023-04-26
TW202246501A (zh) 2022-12-01
MX2023008825A (es) 2023-08-10
CO2023009633A2 (es) 2023-12-20
MA62178A1 (fr) 2023-12-29
AU2022213262A1 (en) 2023-08-24
US20240091383A1 (en) 2024-03-21
CL2023002218A1 (es) 2024-02-02
CN117545842A (zh) 2024-02-09
CR20230363A (es) 2024-02-20
WO2022164351A1 (en) 2022-08-04

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