MA53268A - Mini-gde pour le traitement de la maladie de stockage du glycogène iii - Google Patents

Mini-gde pour le traitement de la maladie de stockage du glycogène iii

Info

Publication number
MA53268A
MA53268A MA053268A MA53268A MA53268A MA 53268 A MA53268 A MA 53268A MA 053268 A MA053268 A MA 053268A MA 53268 A MA53268 A MA 53268A MA 53268 A MA53268 A MA 53268A
Authority
MA
Morocco
Prior art keywords
edg
glycogen
mini
treatment
storage disease
Prior art date
Application number
MA053268A
Other languages
English (en)
Other versions
MA53268B1 (fr
Inventor
Federico Mingozzi
Giuseppe Ronzitti
Patrice Vidal
Original Assignee
Association Inst De Myologie
Genethon
Inst Nat Sante Rech Med
Univ Sorbonne
Univ Devry Val Dessonne
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 Inst De Myologie, Genethon, Inst Nat Sante Rech Med, Univ Sorbonne, Univ Devry Val Dessonne filed Critical Association Inst De Myologie
Publication of MA53268A publication Critical patent/MA53268A/fr
Publication of MA53268B1 publication Critical patent/MA53268B1/fr

Links

Classifications

    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
    • C12N9/1055—Levansucrase (2.4.1.10)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01025—4-Alpha-glucanotransferase (2.4.1.25)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01033—Amylo-alpha-1,6-glucosidase (3.2.1.33)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
MA53268A 2018-08-08 2019-08-06 Mini-gde pour le traitement de maladies de stockage de glycogène de type iii MA53268B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18306088 2018-08-08
PCT/EP2019/071158 WO2020030661A1 (fr) 2018-08-08 2019-08-06 Mini-gde pour le traitement de la maladie de stockage du glycogène iii

Publications (2)

Publication Number Publication Date
MA53268A true MA53268A (fr) 2021-11-17
MA53268B1 MA53268B1 (fr) 2023-06-28

Family

ID=63311942

Family Applications (1)

Application Number Title Priority Date Filing Date
MA53268A MA53268B1 (fr) 2018-08-08 2019-08-06 Mini-gde pour le traitement de maladies de stockage de glycogène de type iii

Country Status (17)

Country Link
US (1) US12637668B2 (fr)
EP (2) EP3833746B1 (fr)
JP (2) JP7548899B2 (fr)
KR (1) KR20210053902A (fr)
CN (1) CN112654698A (fr)
AU (1) AU2019317754B2 (fr)
BR (1) BR112021002202A2 (fr)
CA (1) CA3107572A1 (fr)
DK (1) DK3833746T5 (fr)
EA (1) EA202190475A1 (fr)
ES (1) ES2946415T3 (fr)
FI (1) FI3833746T3 (fr)
IL (1) IL280570B1 (fr)
MA (1) MA53268B1 (fr)
PL (1) PL3833746T3 (fr)
PT (1) PT3833746T (fr)
WO (1) WO2020030661A1 (fr)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IT202000003371A1 (it) * 2020-02-19 2021-08-19 Enea Agenzia Naz Per Le Nuove Tecnologie Lenergia E Lo Sviluppo Economico Sostenibile Composto per il trattamento di una glicogenosi
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
JP2023539219A (ja) 2020-08-24 2023-09-13 ジェネトン 糖原病ｉｉｉの治療のためのｃ末端切断ｇｄｅ
JP2024517427A (ja) * 2021-04-20 2024-04-22 アンジャリウム バイオサイエンシズ エージー アミロ－α－１，６－グルコシダーゼ、４－α－グルカノトランスフェラーゼをコードするＤＮＡ分子の組成物、その作製の方法、及びその使用の方法
WO2023237731A1 (fr) 2022-06-09 2023-12-14 Genethon Gde tronquée au niveau du terminal n pour le traitement de la maladie de stockage du glycogène de type iii
WO2025003477A1 (fr) 2023-06-29 2025-01-02 Genethon Enzymes de débranchement du glycogène tronquées n-terminales pour le traitement de la glycogénose de type iii

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2406687A1 (fr) 2001-11-09 2003-05-09 Transgene S.A. Promoteurs chimeriques pour le controle de l'expression dans des cellules musculaires
ES2932278T3 (es) 2004-06-01 2023-01-17 Genzyme Corp Composiciones y métodos para prevenir la agregación del vector AAV
US9617548B2 (en) 2008-04-22 2017-04-11 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
WO2014130722A1 (fr) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Méthodes et compositions pour le traitement de la maladie de forbes-cori
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
AU2015208247B2 (en) 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
ES2744565T3 (es) 2014-04-25 2020-02-25 Genethon Tratamiento de la hiperbilirrubinemia
EP3236984B1 (fr) * 2014-12-23 2020-07-22 The U.S.A. as represented by the Secretary, Department of Health and Human Services Vecteurs à base de virus adéno-associé codant pour une g6pc modifiée, et utilisations de ces derniers

Also Published As

Publication number Publication date
IL280570A (en) 2021-03-25
US12637668B2 (en) 2026-05-26
PL3833746T3 (pl) 2023-12-04
EP3833746A1 (fr) 2021-06-16
WO2020030661A1 (fr) 2020-02-13
BR112021002202A2 (pt) 2021-05-04
DK3833746T5 (da) 2024-08-19
JP2021532810A (ja) 2021-12-02
AU2019317754A1 (en) 2021-02-18
IL280570B1 (en) 2026-08-01
US20210292724A1 (en) 2021-09-23
NZ773183A (en) 2025-10-31
PT3833746T (pt) 2023-05-25
JP7548899B2 (ja) 2024-09-10
EP4230733A1 (fr) 2023-08-23
AU2019317754B2 (en) 2025-07-10
EP3833746B1 (fr) 2023-03-29
CN112654698A (zh) 2021-04-13
DK3833746T3 (da) 2023-05-30
JP2024054398A (ja) 2024-04-16
CA3107572A1 (fr) 2020-02-13
FI3833746T3 (fi) 2023-06-01
KR20210053902A (ko) 2021-05-12
EA202190475A1 (ru) 2021-04-16
ES2946415T3 (es) 2023-07-18
MA53268B1 (fr) 2023-06-28

Similar Documents

Publication Publication Date Title
EP3630964A4 (fr) Agents thérapeutiques pour la maladie du stockage du glycogène de type iii
EP3728271A4 (fr) Composés macrocycliques pour le traitement de maladie
EP3790867A4 (fr) Inhibiteurs de kdm1a pour le traitement d'une maladie
EP3852735A4 (fr) Agonistes du récepteur farnésoïde x pour le traitement d'une maladie
MA44864A (fr) Trithérapie pour le traitement d'une maladie intestinale inflammatoire
EP3763815A4 (fr) Agent thérapeutique pour la maladie du stockage du glycogène de type ia
MA49144A (fr) Polythérapies pour le traitement du cancer
EP3019491A4 (fr) Inhibiteurs de kinase pour le traitement d'une maladie
EP3431105A4 (fr) Composition médicinale pour le traitement du cancer
MA50409A (fr) Polythérapies pour le traitement du cancer
MA48637A (fr) Polythérapies pour le traitement du cancer
EP3641811A4 (fr) Cellules rénales bioactives à immuno-privilège pour le traitement d'une maladie rénale
EP3737361A4 (fr) Inhibiteurs de la dihydrocéramide désaturase pour le traitement d'une maladie
EP3402533A4 (fr) Méthodes et compositions pour le traitement d'une maladie neurologique
EP3843737A4 (fr) Inhibiteurs de pde9 pour le traitement de la drépanocytose
EP3285795A4 (fr) Oligopeptides amers administrés par voie entérique pour le traitement du diabète de type 2
EP3393468A4 (fr) Méthodes pour le traitement d'une maladie immunodéficiente
EP3766497A4 (fr) Médicament pour le traitement de la toux
EP3496708A4 (fr) Métabolites pour le traitement et la prévention de maladie auto-immune
EP3716966A4 (fr) Compositions d'alpha-cétoacides pour le traitement de l'hypoalbuminémie
EP3430400A4 (fr) Schémas posologiques pour le traitement d'infections fongiques
EP2959903A4 (fr) Médicament pour le traitement de maladie des yeux
IL280570A (en) Mini-gde for the treatment of glycogen storage disease iii
EP3773221A4 (fr) Traitement pour l'hydrocéphalie
MA47395A (fr) Méthode pour le traitement d'une maladie neurologique