EA202190475A1 - Мини-gde для лечения гликогеноза iii типа - Google Patents

Мини-gde для лечения гликогеноза iii типа

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Publication number
EA202190475A1
EA202190475A1 EA202190475A EA202190475A EA202190475A1 EA 202190475 A1 EA202190475 A1 EA 202190475A1 EA 202190475 A EA202190475 A EA 202190475A EA 202190475 A EA202190475 A EA 202190475A EA 202190475 A1 EA202190475 A1 EA 202190475A1
Authority
EA
Eurasian Patent Office
Prior art keywords
gde
mini
treatment
type iii
glycogenosis type
Prior art date
Application number
EA202190475A
Other languages
English (en)
Inventor
Джузеппе РОНЦИТТИ
Патрис Видаль
Федерико МИНГОЦЦИ
Original Assignee
Женетон
Энститю Насьональ Де Ля Санте Э Де Ля Решерш Медикаль
Сорбонн Юниверсите
Юниверсите Д'Эври Валь Д'Эссонн
Ассосиасьен Энститю Де Миоложи
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 Женетон, Энститю Насьональ Де Ля Санте Э Де Ля Решерш Медикаль, Сорбонн Юниверсите, Юниверсите Д'Эври Валь Д'Эссонн, Ассосиасьен Энститю Де Миоложи filed Critical Женетон
Publication of EA202190475A1 publication Critical patent/EA202190475A1/ru

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
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    • 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
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    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • 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)

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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)
  • 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)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Virology (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)
  • Medicines Containing Plant Substances (AREA)

Abstract

Настоящее изобретение относится к мини-GDE для лечения гликогеноза III типа.
EA202190475A 2018-08-08 2019-08-06 Мини-gde для лечения гликогеноза iii типа EA202190475A1 (ru)

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
EA202190475A1 true EA202190475A1 (ru) 2021-04-16

Family

ID=63311942

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202190475A EA202190475A1 (ru) 2018-08-08 2019-08-06 Мини-gde для лечения гликогеноза iii типа

Country Status (17)

Country Link
US (1) US20210292724A1 (ru)
EP (2) EP3833746B1 (ru)
JP (2) JP7548899B2 (ru)
KR (1) KR20210053902A (ru)
CN (1) CN112654698A (ru)
AU (1) AU2019317754A1 (ru)
BR (1) BR112021002202A2 (ru)
CA (1) CA3107572A1 (ru)
DK (1) DK3833746T5 (ru)
EA (1) EA202190475A1 (ru)
ES (1) ES2946415T3 (ru)
FI (1) FI3833746T3 (ru)
IL (1) IL280570A (ru)
MA (1) MA53268B1 (ru)
PL (1) PL3833746T3 (ru)
PT (1) PT3833746T (ru)
WO (1) WO2020030661A1 (ru)

Families Citing this family (5)

* 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 (en) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions of dna molecules, methods of making therefor, and methods of use thereof
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

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
MX360727B (es) 2004-06-01 2018-11-14 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 (en) * 2013-02-20 2016-11-09 Valerion Therapeutics Llc METHOD AND COMPOSITIONS FOR TREATING 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
AU2015208247B2 (en) 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
CA2942451A1 (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
PL3833746T3 (pl) 2023-12-04
AU2019317754A1 (en) 2021-02-18
IL280570A (en) 2021-03-25
MA53268A (fr) 2021-11-17
EP3833746A1 (en) 2021-06-16
EP3833746B1 (en) 2023-03-29
JP7548899B2 (ja) 2024-09-10
WO2020030661A1 (en) 2020-02-13
EP4230733A1 (en) 2023-08-23
MA53268B1 (fr) 2023-06-28
CN112654698A (zh) 2021-04-13
ES2946415T3 (es) 2023-07-18
PT3833746T (pt) 2023-05-25
CA3107572A1 (en) 2020-02-13
US20210292724A1 (en) 2021-09-23
JP2024054398A (ja) 2024-04-16
BR112021002202A2 (pt) 2021-05-04
DK3833746T5 (da) 2024-08-19
DK3833746T3 (da) 2023-05-30
JP2021532810A (ja) 2021-12-02
FI3833746T3 (fi) 2023-06-01
KR20210053902A (ko) 2021-05-12

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