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FR3126005B1 - Activateur pharmacologique et/ou génétique pour son utilisation pour préserver et régénérer la structure et la fonction musculaire en bloquant la sénescence - Google Patents

Activateur pharmacologique et/ou génétique pour son utilisation pour préserver et régénérer la structure et la fonction musculaire en bloquant la sénescence Download PDF

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Publication number
FR3126005B1
FR3126005B1 FR2108489A FR2108489A FR3126005B1 FR 3126005 B1 FR3126005 B1 FR 3126005B1 FR 2108489 A FR2108489 A FR 2108489A FR 2108489 A FR2108489 A FR 2108489A FR 3126005 B1 FR3126005 B1 FR 3126005B1
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Prior art keywords
activator
pharmacological
preserving
function
genetic
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FR2108489A
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English (en)
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FR3126005A1 (fr
Inventor
Valentina Taglietti
Frédéric Relaix
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Upec
Institut National de la Sante et de la Recherche Medicale INSERM
Association Francaise Contre les Myopathies
Original Assignee
Upec
Institut National de la Sante et de la Recherche Medicale INSERM
Association Francaise Contre les Myopathies
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Application filed by Upec, Institut National de la Sante et de la Recherche Medicale INSERM, Association Francaise Contre les Myopathies filed Critical Upec
Priority to FR2108489A priority Critical patent/FR3126005B1/fr
Priority to PCT/EP2022/071914 priority patent/WO2023012264A2/fr
Priority to EP22765409.2A priority patent/EP4380965A2/fr
Priority to US18/294,626 priority patent/US20240343779A1/en
Publication of FR3126005A1 publication Critical patent/FR3126005A1/fr
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Publication of FR3126005B1 publication Critical patent/FR3126005B1/fr
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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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/72—Receptors; Cell surface antigens; Cell surface determinants for hormones
    • C07K14/723—G protein coupled receptor, e.g. TSHR-thyrotropin-receptor, LH/hCG receptor, FSH receptor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
    • A61K31/352—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. methantheline 
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Endocrinology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Diabetes (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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

L’invention concerne un activateur pharmacologique et/ou génétique pour son utilisation pour préserver et régénérer la structure et la fonction musculaire. L’invention se caractérise en ce que ledit activateur pharmacologique et/ou génétique est un activateur de la voie de signalisation du récepteur de la thyréostimuline (TSHR) dans des cellules souches musculaires (cellules satellites) pour préserver et régénérer la structure et la fonction musculaire en bloquant la sénescence. L’invention concerne également une combinaison d’un activateur pharmacologique pour son utilisation selon l’invention et d’un activateur génétique pour son utilisation selon l’invention, ainsi qu’un vecteur adénoviral comprenant une séquence protéique du récepteur de la thyréostimuline (TSHR) de SEQ ID NO :1 ou un variant fonctionnel ayant une séquence protéique identique à au moins 60% à la séquence SEQ ID NO :1, ou une séquence nucléotidique du TSHR de SEQ ID NO :2 ou un variant fonctionnel ayant une séquence nucléotidique identique à au moins 60% à la séquence SEQ ID NO :2 pour son utilisation à titre de médicament. Figure 7L
FR2108489A 2021-08-04 2021-08-04 Activateur pharmacologique et/ou génétique pour son utilisation pour préserver et régénérer la structure et la fonction musculaire en bloquant la sénescence Active FR3126005B1 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
FR2108489A FR3126005B1 (fr) 2021-08-04 2021-08-04 Activateur pharmacologique et/ou génétique pour son utilisation pour préserver et régénérer la structure et la fonction musculaire en bloquant la sénescence
PCT/EP2022/071914 WO2023012264A2 (fr) 2021-08-04 2022-08-04 Activateur pharmacologique et/ou genetique pour son utilisation pour preserver et regenerer la structure et la fonction musculaire en bloquant la senescence
EP22765409.2A EP4380965A2 (fr) 2021-08-04 2022-08-04 Activateur pharmacologique et/ou genetique pour son utilisation pour preserver et regenerer la structure et la fonction musculaire en bloquant la senescence
US18/294,626 US20240343779A1 (en) 2021-08-04 2022-08-04 Pharmacological and/or genetic activator for use in preserving and regenerating muscle structure and function by blocking senescence

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
FR2108489 2021-08-04
FR2108489A FR3126005B1 (fr) 2021-08-04 2021-08-04 Activateur pharmacologique et/ou génétique pour son utilisation pour préserver et régénérer la structure et la fonction musculaire en bloquant la sénescence

Publications (2)

Publication Number Publication Date
FR3126005A1 FR3126005A1 (fr) 2023-02-10
FR3126005B1 true FR3126005B1 (fr) 2024-11-29

Family

ID=80122380

Family Applications (1)

Application Number Title Priority Date Filing Date
FR2108489A Active FR3126005B1 (fr) 2021-08-04 2021-08-04 Activateur pharmacologique et/ou génétique pour son utilisation pour préserver et régénérer la structure et la fonction musculaire en bloquant la sénescence

Country Status (4)

Country Link
US (1) US20240343779A1 (fr)
EP (1) EP4380965A2 (fr)
FR (1) FR3126005B1 (fr)
WO (1) WO2023012264A2 (fr)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH05504683A (ja) * 1989-12-20 1993-07-22 ニコルス　インスティテュート　ダイアグノスティックス 組み換えチロトロピン受容体
WO2018155913A1 (fr) 2017-02-21 2018-08-30 성균관대학교산학협력단 Procédé de différenciation en cellule de muscle squelettique à l'aide d'un composé de faible poids moléculaire
JP7025041B2 (ja) * 2019-12-26 2022-02-24 ヤマサ醤油株式会社 変異が導入された甲状腺刺激ホルモン受容体

Also Published As

Publication number Publication date
FR3126005A1 (fr) 2023-02-10
WO2023012264A3 (fr) 2023-03-30
US20240343779A1 (en) 2024-10-17
EP4380965A2 (fr) 2024-06-12
WO2023012264A2 (fr) 2023-02-09

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