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WO2023012264A3 - Activateur pharmacologique et/ou genetique pour son utilisation pour preserver et regenerer la structure et la fonction musculaire en bloquant la senescence - Google Patents

Activateur pharmacologique et/ou genetique pour son utilisation pour preserver et regenerer la structure et la fonction musculaire en bloquant la senescence Download PDF

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Publication number
WO2023012264A3
WO2023012264A3 PCT/EP2022/071914 EP2022071914W WO2023012264A3 WO 2023012264 A3 WO2023012264 A3 WO 2023012264A3 EP 2022071914 W EP2022071914 W EP 2022071914W WO 2023012264 A3 WO2023012264 A3 WO 2023012264A3
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WO
WIPO (PCT)
Prior art keywords
activator
function
senescence
preserving
blocking
Prior art date
Application number
PCT/EP2022/071914
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English (en)
Other versions
WO2023012264A2 (fr
Inventor
Valentina TAGLIETTI
Frédéric RELAIX
Original Assignee
Association Francaise Contre Les Myopathies
Université Paris-Est Créteil (Upec)
Institut National De La Sante Et De La Recherche Medicale (Inserm)
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
Application filed by Association Francaise Contre Les Myopathies, Université Paris-Est Créteil (Upec), Institut National De La Sante Et De La Recherche Medicale (Inserm) filed Critical Association Francaise Contre Les Myopathies
Priority to EP22765409.2A priority Critical patent/EP4380965A2/fr
Priority to US18/294,626 priority patent/US20240343779A1/en
Publication of WO2023012264A2 publication Critical patent/WO2023012264A2/fr
Publication of WO2023012264A3 publication Critical patent/WO2023012264A3/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

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  • 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)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Endocrinology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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 pour son utilisation pour préserver et régénérer la structure et la fonction musculaire. L'invention se caractérise en ce que ledit activateur 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 une séquence nucléotidique du TSHR de SEQ ID NO :2 pour son utilisation à titre de médicament.
PCT/EP2022/071914 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 WO2023012264A2 (fr)

Priority Applications (2)

Application Number Priority Date Filing Date Title
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
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
FRFR2108489 2021-08-04

Publications (2)

Publication Number Publication Date
WO2023012264A2 WO2023012264A2 (fr) 2023-02-09
WO2023012264A3 true WO2023012264A3 (fr) 2023-03-30

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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

Country Status (4)

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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991009137A1 (fr) * 1989-12-20 1991-06-27 Basil Rapoport Recepteur de thyrotropine recombinant
JP2021103947A (ja) * 2019-12-26 2021-07-26 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

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991009137A1 (fr) * 1989-12-20 1991-06-27 Basil Rapoport Recepteur de thyrotropine recombinant
JP2021103947A (ja) * 2019-12-26 2021-07-26 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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MCINTOSH L.M. ET AL: "Hypothyroidism prolongs and increases mdx muscle precursor proliferation and delays myotube formation in normal and dystrophic limb muscle", vol. 73, no. 3-4, 1 March 1995 (1995-03-01), CA, pages 181 - 190, XP055929874, ISSN: 0829-8211, Retrieved from the Internet <URL:http://dx.doi.org/10.1139/o95-022> DOI: 10.1139/o95-022 *
NEUMANN SUSANNE ET AL: "Small-molecule agonists for the thyrotropin receptor stimulate thyroid function in human thyrocytes and mice", vol. 106, no. 30, 28 July 2009 (2009-07-28), pages 12471 - 12476, XP055929513, ISSN: 0027-8424, Retrieved from the Internet <URL:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2708975/pdf/zpq12471.pdf> DOI: 10.1073/pnas.0904506106 *

Also Published As

Publication number Publication date
FR3126005A1 (fr) 2023-02-10
FR3126005B1 (fr) 2024-11-29
US20240343779A1 (en) 2024-10-17
WO2023012264A2 (fr) 2023-02-09
EP4380965A2 (fr) 2024-06-12

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