MA62178A1 - Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale - Google Patents

Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale

Info

Publication number
MA62178A1
MA62178A1 MA62178A MA62178A MA62178A1 MA 62178 A1 MA62178 A1 MA 62178A1 MA 62178 A MA62178 A MA 62178A MA 62178 A MA62178 A MA 62178A MA 62178 A1 MA62178 A1 MA 62178A1
Authority
MA
Morocco
Prior art keywords
nucleic acid
smn1
recombinant virus
mir
treatment
Prior art date
Application number
MA62178A
Other languages
English (en)
Inventor
Dmitry Valentinovich Morozov
Dmitriy Aleksandrovich Madera
Pavel Mikhailovich Gershovich
Anna Sergeevna Veselova
Aleksei Sergeevich Siutkin
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 MA62178A1 publication Critical patent/MA62178A1/fr

Links

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

Abstract

La présente invention se rapporte aux domaines de la biotechnologie, de la virologie, de la génétique et de la biologie moléculaire. Plus particulièrement, la présente invention concerne un acide nucléique isolé servant à produire un produit viral de thérapie génique, ledit acide nucléique isolé comprenant un acide nucléique qui code la protéine smn1 ayant la séquence d'acides aminés de seq id no : 1, et un acide nucléique qui code le microarn mir-23a, une cassette d'expression et un vecteur basé sur cette dernière, ainsi qu'un virus recombinant à base d'aav9 pour exprimer le gène smn1 dans des cellules cibles, une composition pharmaceutique qui comprend ledit virus recombinant, et diverses utilisations du virus recombinant susmentionné et de la composition susmentionnée.
MA62178A 2021-01-29 2022-01-28 Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale MA62178A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2021102051A RU2839898C2 (ru) 2021-01-29 Синергетическое действие SMN1 и miR-23a при лечении спинальной мышечной атрофии
PCT/RU2022/000025 WO2022164351A1 (fr) 2021-01-29 2022-01-28 Effet synergique <p> de smn1 et mir-23a dans le traitement de l'amyotrophie spinale </p>

Publications (1)

Publication Number Publication Date
MA62178A1 true MA62178A1 (fr) 2023-12-29

Family

ID=82653732

Family Applications (1)

Application Number Title Priority Date Filing Date
MA62178A MA62178A1 (fr) 2021-01-29 2022-01-28 Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale

Country Status (19)

Country Link
US (1) US20240091383A1 (fr)
CN (1) CN117545842A (fr)
AR (1) AR124736A1 (fr)
AU (1) AU2022213262A1 (fr)
BR (1) BR112023015177A2 (fr)
CA (1) CA3206671A1 (fr)
CL (1) CL2023002218A1 (fr)
CO (1) CO2023009633A2 (fr)
CR (1) CR20230363A (fr)
EC (1) ECSP23056133A (fr)
IL (1) IL304612A (fr)
JO (1) JOP20230168A1 (fr)
MA (1) MA62178A1 (fr)
MX (1) MX2023008825A (fr)
PE (1) PE20250601A1 (fr)
PY (1) PY2205136A (fr)
TW (1) TW202246501A (fr)
UY (1) UY39621A (fr)
WO (1) WO2022164351A1 (fr)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4518970B1 (fr) 2022-05-04 2026-03-25 Scholar Rock, Inc. Utilisation d'un inhibiteur de la myostatine pour le traitement de l'amyotrophie spinale
CN120677175A (zh) 2022-12-22 2025-09-19 供石公司 肌生成抑制蛋白活化的选择性和强效抑制剂
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 (fr) * 2023-12-12 2025-06-19 Sichuan Real & Best Biotech Co., Ltd. Méthode de traitement de l'amyotrophie spinale
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

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2310501T3 (pl) * 2008-07-23 2013-12-31 Boehringer Ingelheim Pharma Nowe elementy regulatorowe
SG10202109219SA (en) * 2009-05-02 2021-10-28 Genzyme Corp Gene therapy for neurodegenerative disorders
EP2634253B1 (fr) * 2010-10-27 2016-05-11 Jichi Medical University Virions de virus adéno-associé pour le transfert de gènes dans des cellules neuronales
WO2017106354A1 (fr) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Vecteurs viraux adéno-associés à utiliser dans le traitement de l'amyotrophie spinale
EP3411059A4 (fr) * 2016-02-02 2019-10-16 University Of Massachusetts Procédé pour améliorer l'efficacité de l'administration au système nerveux central d'un gène aav par voie systémique
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
AU2022213262A9 (en) 2024-10-17
TW202246501A (zh) 2022-12-01
BR112023015177A2 (pt) 2023-11-14
AU2022213262A1 (en) 2023-08-24
ECSP23056133A (es) 2023-08-31
CO2023009633A2 (es) 2023-12-20
PE20250601A1 (es) 2025-02-26
UY39621A (es) 2022-08-31
CN117545842A (zh) 2024-02-09
PY2205136A (es) 2022-08-16
WO2022164351A1 (fr) 2022-08-04
CR20230363A (es) 2024-02-20
AR124736A1 (es) 2023-04-26
IL304612A (en) 2023-09-01
CA3206671A1 (fr) 2022-08-04
JOP20230168A1 (ar) 2023-07-27
CL2023002218A1 (es) 2024-02-02
MX2023008825A (es) 2023-08-10
US20240091383A1 (en) 2024-03-21

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