PL3833746T3 - Mini-gde do leczenia choroby spichrzeniowej glikogenu iii - Google Patents

Mini-gde do leczenia choroby spichrzeniowej glikogenu iii

Info

Publication number
PL3833746T3
PL3833746T3 PL19746522.2T PL19746522T PL3833746T3 PL 3833746 T3 PL3833746 T3 PL 3833746T3 PL 19746522 T PL19746522 T PL 19746522T PL 3833746 T3 PL3833746 T3 PL 3833746T3
Authority
PL
Poland
Prior art keywords
gde
mini
treatment
storage disease
glycogen storage
Prior art date
Application number
PL19746522.2T
Other languages
English (en)
Inventor
Giuseppe RONZITTI
Patrice VIDAL
Federico Mingozzi
Original Assignee
Genethon
Institut National de la Santé et de la Recherche Médicale
Sorbonne Université
Universite D'evry Val D'essonne
Association Institut 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.)
Filing date
Publication date
Application filed by Genethon, Institut National de la Santé et de la Recherche Médicale, Sorbonne Université, Universite D'evry Val D'essonne, Association Institut De Myologie filed Critical Genethon
Publication of PL3833746T3 publication Critical patent/PL3833746T3/pl

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Classifications

    • 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
    • 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
    • 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)
    • 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)
    • C12N9/1055—Levansucrase (2.4.1.10)
    • 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)
    • 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)
PL19746522.2T 2018-08-08 2019-08-06 Mini-gde do leczenia choroby spichrzeniowej glikogenu iii PL3833746T3 (pl)

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 (1)

Publication Number Publication Date
PL3833746T3 true PL3833746T3 (pl) 2023-12-04

Family

ID=63311942

Family Applications (1)

Application Number Title Priority Date Filing Date
PL19746522.2T PL3833746T3 (pl) 2018-08-08 2019-08-06 Mini-gde do leczenia choroby spichrzeniowej glikogenu iii

Country Status (17)

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

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 (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
CA2406687A1 (en) 2001-11-09 2003-05-09 Transgene S.A. Chimeric promoters for controlling expression in muscle cells
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 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of forbes-cori disease
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 (en) * 2014-12-23 2020-07-22 The U.S.A. 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
IL280570A (en) 2021-03-25
US12637668B2 (en) 2026-05-26
EP3833746A1 (en) 2021-06-16
WO2020030661A1 (en) 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 (en) 2023-08-23
AU2019317754B2 (en) 2025-07-10
MA53268A (fr) 2021-11-17
EP3833746B1 (en) 2023-03-29
CN112654698A (zh) 2021-04-13
DK3833746T3 (da) 2023-05-30
JP2024054398A (ja) 2024-04-16
CA3107572A1 (en) 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

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