ECSP23056133A - 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
ECSP23056133A
ECSP23056133A ECSENADI202356133A ECDI202356133A ECSP23056133A EC SP23056133 A ECSP23056133 A EC SP23056133A EC SENADI202356133 A ECSENADI202356133 A EC SENADI202356133A EC DI202356133 A ECDI202356133 A EC DI202356133A EC SP23056133 A ECSP23056133 A EC SP23056133A
Authority
EC
Ecuador
Prior art keywords
nucleic acid
smn1
mir
recombinant virus
treatment
Prior art date
Application number
ECSENADI202356133A
Other languages
English (en)
Inventor
Dmitriy Aleksandrovich Madera
Aleksei Sergeevich Siutkin
Pavel Mikhailovich Gershovich
Anna Sergeevna Veselova
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
Priority claimed from RU2021102051A external-priority patent/RU2839898C2/ru
Application filed by Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of ECSP23056133A publication Critical patent/ECSP23056133A/es

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Classifications

    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • 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)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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.
ECSENADI202356133A 2021-01-29 2023-07-24 Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal ECSP23056133A (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
ECSP23056133A true ECSP23056133A (es) 2023-08-31

Family

ID=82653732

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI202356133A ECSP23056133A (es) 2021-01-29 2023-07-24 Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal

Country Status (19)

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)
JO (1) JOP20230168A1 (es)
MA (1) MA62178A1 (es)
MX (1) MX2023008825A (es)
PE (1) PE20250601A1 (es)
PY (1) PY2205136A (es)
TW (1) TW202246501A (es)
UY (1) UY39621A (es)
WO (1) WO2022164351A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20211081T1 (hr) 2015-09-15 2021-10-15 Scholar Rock, Inc. Anti-pro/latentna miostatinska protutijela i njihove uporabe
DK4518970T3 (da) 2022-05-04 2026-04-07 Scholar Rock Inc Anvendelse af myostatinhæmmer til behandling af spinal muskelatrofi
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
WO2026076383A1 (en) 2024-10-06 2026-04-09 Scholar Rock, Inc. Use of myostatin inhibitor for treating spinal muscular atrophy

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA201100225A1 (ru) * 2008-07-23 2011-08-30 Бёрингер Ингельхайм Фарма Гмбх Унд Ко. Кг Новые регуляторные элементы
HRP20250897T1 (hr) * 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
WO2012057363A1 (ja) * 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
MX2018007234A (es) * 2015-12-14 2018-11-09 Univ Pennsylvania Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.
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
US20240091383A1 (en) 2024-03-21
MX2023008825A (es) 2023-08-10
UY39621A (es) 2022-08-31
CA3206671A1 (en) 2022-08-04
CL2023002218A1 (es) 2024-02-02
AU2022213262A9 (en) 2024-10-17
PY2205136A (es) 2022-08-16
BR112023015177A2 (pt) 2023-11-14
TW202246501A (zh) 2022-12-01
IL304612A (en) 2023-09-01
PE20250601A1 (es) 2025-02-26
AR124736A1 (es) 2023-04-26
CN117545842A (zh) 2024-02-09
WO2022164351A1 (en) 2022-08-04
CO2023009633A2 (es) 2023-12-20
CR20230363A (es) 2024-02-20
MA62178A1 (fr) 2023-12-29
AU2022213262A1 (en) 2023-08-24
JOP20230168A1 (ar) 2023-07-27

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