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DK3833746T3 - Mini-gde til behandlingen af glykogenlagringssygdom iii - Google Patents

Mini-gde til behandlingen af glykogenlagringssygdom iii Download PDF

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Publication number
DK3833746T3
DK3833746T3 DK19746522.2T DK19746522T DK3833746T3 DK 3833746 T3 DK3833746 T3 DK 3833746T3 DK 19746522 T DK19746522 T DK 19746522T DK 3833746 T3 DK3833746 T3 DK 3833746T3
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DK
Denmark
Prior art keywords
gde
mini
treatment
storage disease
glycogen storage
Prior art date
Application number
DK19746522.2T
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English (en)
Other versions
DK3833746T5 (da
Inventor
Giuseppe Ronzitti
Patrice Vidal
Federico Mingozzi
Original Assignee
Genethon
Inst Nat Sante Rech Med
Univ Sorbonne
Univ Devry Val Dessonne
Association Inst De Myologie
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.)
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Application filed by Genethon, Inst Nat Sante Rech Med, Univ Sorbonne, Univ Devry Val Dessonne, Association Inst De Myologie filed Critical Genethon
Publication of DK3833746T3 publication Critical patent/DK3833746T3/da
Application granted granted Critical
Publication of DK3833746T5 publication Critical patent/DK3833746T5/da

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405Glucanases
    • C12N9/2451Glucanases acting on alpha-1,6-glucosidic bonds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1048Glycosyltransferases (2.4)
    • C12N9/1051Hexosyltransferases (2.4.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1048Glycosyltransferases (2.4)
    • C12N9/1051Hexosyltransferases (2.4.1)
    • C12N9/1055Levansucrase (2.4.1.10)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/01Hexosyltransferases (2.4.1)
    • C12Y204/010254-Alpha-glucanotransferase (2.4.1.25)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01033Amylo-alpha-1,6-glucosidase (3.2.1.33)

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  • 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)
  • Zoology (AREA)
  • Wood Science & Technology (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)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
DK19746522.2T 2018-08-08 2019-08-06 Mini-gde til behandlingen af glykogenlagringssygdom iii DK3833746T5 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18306088 2018-08-08
PCT/EP2019/071158 WO2020030661A1 (en) 2018-08-08 2019-08-06 Mini-gde for the treatment of glycogen storage disease iii

Publications (2)

Publication Number Publication Date
DK3833746T3 true DK3833746T3 (da) 2023-05-30
DK3833746T5 DK3833746T5 (da) 2024-08-19

Family

ID=63311942

Family Applications (1)

Application Number Title Priority Date Filing Date
DK19746522.2T DK3833746T5 (da) 2018-08-08 2019-08-06 Mini-gde til behandlingen af glykogenlagringssygdom iii

Country Status (18)

Country Link
US (1) US20210292724A1 (da)
EP (2) EP3833746B1 (da)
JP (2) JP7548899B2 (da)
KR (1) KR20210053902A (da)
CN (1) CN112654698A (da)
AU (1) AU2019317754B2 (da)
BR (1) BR112021002202A2 (da)
CA (1) CA3107572A1 (da)
DK (1) DK3833746T5 (da)
EA (1) EA202190475A1 (da)
ES (1) ES2946415T3 (da)
FI (1) FI3833746T3 (da)
IL (1) IL280570A (da)
MA (1) MA53268B1 (da)
NZ (1) NZ773183A (da)
PL (1) PL3833746T3 (da)
PT (1) PT3833746T (da)
WO (1) WO2020030661A1 (da)

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
AU2021314809A1 (en) 2020-07-27 2023-02-23 Anjarium Biosciences Ag Compositions of DNA molecules, methods of making therefor, and methods of use thereof
US20230313152A1 (en) 2020-08-24 2023-10-05 Genethon C-terminal truncated gde for the treatment of glycogen storage disease iii
EP4326860A1 (en) 2021-04-20 2024-02-28 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
WO2023237731A1 (en) 2022-06-09 2023-12-14 Genethon N-terminal truncated gde for the treatment of glycogen storage disease iii
WO2025003477A1 (en) 2023-06-29 2025-01-02 Genethon N-terminal truncated glycogen debranching enzymes for the treatment of glycogen storage disease iii

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030157064A1 (en) 2001-11-09 2003-08-21 Pascal Neuville Chimeric promoters for controlling expression in muscle cells
EP4170024A1 (en) 2004-06-01 2023-04-26 Genzyme Corporation Compositions and methods to prevent aav vector aggregation
ES2746907T5 (es) 2008-04-22 2024-12-11 Vib Vzw Elementos reguladores de ácido nucleico específicos de hígado y métodos y uso de los mismos
AU2014218853A1 (en) 2013-02-20 2015-08-20 Valerion Therapeutics, Llc Methods and compositions for treatment of Forbes-Cori disease
HUE047996T2 (hu) 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
PL3097197T3 (pl) 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
WO2015162302A2 (en) 2014-04-25 2015-10-29 Genethon Treatment of hyperbilirubinemia
WO2016106303A1 (en) * 2014-12-23 2016-06-30 THE UNITED STATES OF AMERICA, as representd 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
PT3833746T (pt) 2023-05-25
AU2019317754B2 (en) 2025-07-10
US20210292724A1 (en) 2021-09-23
EP3833746A1 (en) 2021-06-16
EP3833746B1 (en) 2023-03-29
IL280570A (en) 2021-03-25
CN112654698A (zh) 2021-04-13
DK3833746T5 (da) 2024-08-19
EP4230733A1 (en) 2023-08-23
EA202190475A1 (ru) 2021-04-16
JP2021532810A (ja) 2021-12-02
FI3833746T3 (fi) 2023-06-01
CA3107572A1 (en) 2020-02-13
WO2020030661A1 (en) 2020-02-13
MA53268A (fr) 2021-11-17
AU2019317754A1 (en) 2021-02-18
PL3833746T3 (pl) 2023-12-04
NZ773183A (en) 2025-10-31
BR112021002202A2 (pt) 2021-05-04
JP2024054398A (ja) 2024-04-16
JP7548899B2 (ja) 2024-09-10
ES2946415T3 (es) 2023-07-18
KR20210053902A (ko) 2021-05-12
MA53268B1 (fr) 2023-06-28

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