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)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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 (18)

Country Link
US (1) US20210292724A1 (fr)
EP (2) EP4230733A1 (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) IL280570A (fr)
MA (1) MA53268B1 (fr)
NZ (1) NZ773183A (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
EP4189098A1 (fr) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions de molécules d'adn, leurs procédés de fabrication et leurs procédés d'utilisation
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
AU2022260111A1 (en) 2021-04-20 2023-11-30 Anjarium Biosciences Ag Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
EP4536816A1 (fr) 2022-06-09 2025-04-16 Genethon Gde tronquée au niveau du terminal n pour le traitement de la maladie de stockage du glycogène de type iii
CN121420060A (zh) 2023-06-29 2026-01-27 吉尼松公司 用于治疗糖原贮积病iii的n-端截短的糖原脱支酶

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
MXPA06014030A (es) 2004-06-01 2007-10-08 Genzyme Corp Composiciones y metodos para evitar la agregacion del vector aav.
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
EP2958936A4 (fr) * 2013-02-20 2016-11-09 Valerion Therapeutics Llc Méthodes et compositions pour le traitement de la maladie de forbes-cori
CA3209883A1 (fr) 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Compositions et variants de virus adeno-associes, et methodes et utilisations pour un transfert de genes dans des cellules, des organes et des tissus
WO2015110449A1 (fr) 2014-01-21 2015-07-30 Vrije Universiteit Brussel Éléments régulateurs d'acide nucléique exprimé dans un muscle, méthodes et utilisation associées
DK3546585T3 (da) 2014-04-25 2025-11-03 Genethon Nukleinsyrer med en modificeret intron til brug i behandlingen af hyperbilirubinæmi
US10415044B2 (en) * 2014-12-23 2019-09-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Adeno-associated virus vectors encoding modified G6PC and uses thereof

Also Published As

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

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