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AR124736A1 - EFECTO SINÉRGICO DE SMN1 Y miR-23a EN EL TRATAMIENTO DE LA ATROFIA MUSCULAR ESPINAL - Google Patents

EFECTO SINÉRGICO DE SMN1 Y miR-23a EN EL TRATAMIENTO DE LA ATROFIA MUSCULAR ESPINAL

Info

Publication number
AR124736A1
AR124736A1 ARP220100193A ARP220100193A AR124736A1 AR 124736 A1 AR124736 A1 AR 124736A1 AR P220100193 A ARP220100193 A AR P220100193A AR P220100193 A ARP220100193 A AR P220100193A AR 124736 A1 AR124736 A1 AR 124736A1
Authority
AR
Argentina
Prior art keywords
nucleic acid
smn1
recombinant virus
aav9
mir
Prior art date
Application number
ARP220100193A
Other languages
English (en)
Inventor
Dmitriy Aleksandrovich Madera
Anna Sergeevna Veselova
Aleksei Sergeevich Siutkin
Pavel Mikhailovich Gershovich
Dmitry Valentinovich Morozov
Original Assignee
Llc «Anabion»
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
Priority claimed from RU2021102051A external-priority patent/RU2839898C2/ru
Application filed by Llc «Anabion» filed Critical Llc «Anabion»
Publication of AR124736A1 publication Critical patent/AR124736A1/es

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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
    • 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
    • C12N15/11—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • 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

La presente solicitud está relacionada con los campos de la biotecnología, la virología, la genética y la biología molecular. Más específicamente, la presente invención se refiere a un ácido nucleico aislado para producir un producto viral para terapia génica; dicho ácido nucleico aislado comprende un ácido nucleico que codifica la proteína SMN1 que tiene la secuencia de aminoácidos de SEQ ID Nº 1, y un ácido nucleico que codifica el microARN miR-23a, un casete de expresión y un vector basado en el mismo, así como un virus recombinante basado en AAV9 para expresar el gen SMN1 en células diana, una composición farmacéutica que incluye dicho virus recombinante, y diversos usos del virus recombinante anterior y de la composición anterior. Reivindicación 1: Un ácido nucleico aislado para producir un producto viral para terapia génica; dicho ácido nucleico aislado comprende un ácido nucleico que codifica la proteína SMN1 (proteína de supervivencia de motoneuronas) que tiene la secuencia de aminoácidos de SEQ ID Nº 1, y un ácido nucleico que codifica el microARN miR-23a. Reivindicación 5: Un casete de expresión que comprende el ácido nucleico según cualquiera de las reivindicaciones 1 a 4. Reivindicación 8: Un vector de expresión que comprende el ácido nucleico según una cualquiera de las reivindicaciones 1 a 4 o el casete según una cualquiera de las reivindicaciones 5 a 7. Reivindicación 9: Un virus recombinante basado en AAV9 (virus adeno-asociado de serotipo 9) para la expresión del gen SMN1 en células diana; dicho virus recombinante basado en AAV9 comprende una cápside y el casete de expresión según cualquiera de las reivindicaciones 5 a 7. Reivindicación 15: Una composición farmacéutica para entregar el gen SMN1 a las células diana; dicha composición farmacéutica comprende el virus recombinante basado en AAV9 según las reivindicaciones 9 a 14 en combinación con uno o más excipientes farmacéuticamente aceptables.
ARP220100193A 2021-01-29 2022-01-31 EFECTO SINÉRGICO DE SMN1 Y miR-23a EN EL TRATAMIENTO DE LA ATROFIA MUSCULAR ESPINAL AR124736A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
RU2021102051A RU2839898C2 (ru) 2021-01-29 Синергетическое действие SMN1 и miR-23a при лечении спинальной мышечной атрофии

Publications (1)

Publication Number Publication Date
AR124736A1 true AR124736A1 (es) 2023-04-26

Family

ID=82653732

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220100193A AR124736A1 (es) 2021-01-29 2022-01-31 EFECTO SINÉRGICO DE SMN1 Y miR-23a EN EL TRATAMIENTO DE LA ATROFIA MUSCULAR ESPINAL

Country Status (17)

Country Link
US (1) US20240091383A1 (es)
CN (1) CN117545842A (es)
AR (1) AR124736A1 (es)
AU (1) AU2022213262A1 (es)
BR (1) BR112023015177A2 (es)
CA (1) CA3206671A1 (es)
CL (1) CL2023002218A1 (es)
CO (1) CO2023009633A2 (es)
CR (1) CR20230363A (es)
EC (1) ECSP23056133A (es)
IL (1) IL304612A (es)
MA (1) MA62178A1 (es)
MX (1) MX2023008825A (es)
PE (1) PE20250601A1 (es)
TW (1) TW202246501A (es)
UY (1) UY39621A (es)
WO (1) WO2022164351A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023265846A1 (en) 2022-05-04 2024-12-12 Scholar Rock, Inc. Use of myostatin inhibitor for treating spinal muscular atrophy
EP4638496A1 (en) 2022-12-22 2025-10-29 Scholar Rock, Inc. Selective and potent inhibitory antibodies of myostatin activation
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
WO2025124614A1 (en) * 2023-12-12 2025-06-19 Sichuan Real & Best Biotech Co., Ltd. A method for treating spinal muscular atrophy
CN118685413B (zh) * 2024-08-28 2025-02-07 杭州嘉因生物科技有限公司 下调内源性smn的诱导型稳定细胞株的构建及其应用

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20130888T1 (hr) * 2008-07-23 2013-10-25 Boehringer Ingelheim Pharma Gmbh & Co.Kg. Novi regulacijski elementi
CN102459611B (zh) * 2009-05-02 2016-11-09 建新公司 神经退行性疾病的基因治疗
EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
WO2017106354A1 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
CA3011939A1 (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
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение

Also Published As

Publication number Publication date
AU2022213262A1 (en) 2023-08-24
MX2023008825A (es) 2023-08-10
BR112023015177A2 (pt) 2023-11-14
CO2023009633A2 (es) 2023-12-20
MA62178A1 (fr) 2023-12-29
AU2022213262A9 (en) 2024-10-17
UY39621A (es) 2022-08-31
TW202246501A (zh) 2022-12-01
US20240091383A1 (en) 2024-03-21
PE20250601A1 (es) 2025-02-26
CR20230363A (es) 2024-02-20
CN117545842A (zh) 2024-02-09
CA3206671A1 (en) 2022-08-04
IL304612A (en) 2023-09-01
WO2022164351A1 (en) 2022-08-04
ECSP23056133A (es) 2023-08-31
CL2023002218A1 (es) 2024-02-02

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