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SI4035687T1 - Postopek za genetsko zdravljenje z uporabo virusa aav-xbp1s/gfp in njegova uporaba pri preprečevanju in zdravljenju amiotrofične lateralne skleroze - Google Patents

Postopek za genetsko zdravljenje z uporabo virusa aav-xbp1s/gfp in njegova uporaba pri preprečevanju in zdravljenju amiotrofične lateralne skleroze

Info

Publication number
SI4035687T1
SI4035687T1 SI201631899T SI201631899T SI4035687T1 SI 4035687 T1 SI4035687 T1 SI 4035687T1 SI 201631899 T SI201631899 T SI 201631899T SI 201631899 T SI201631899 T SI 201631899T SI 4035687 T1 SI4035687 T1 SI 4035687T1
Authority
SI
Slovenia
Prior art keywords
treatment
xbp1s
gfp
prevention
lateral sclerosis
Prior art date
Application number
SI201631899T
Other languages
English (en)
Inventor
Flores Claudio Andres Hetz
Paterakis Vicente Spiro Valenzuela
Original Assignee
Universidad De Chile
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 Universidad De Chile filed Critical Universidad De Chile
Publication of SI4035687T1 publication Critical patent/SI4035687T1/sl

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • CCHEMISTRY; METALLURGY
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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • CCHEMISTRY; METALLURGY
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0089Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
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    • 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
    • C12YENZYMES
    • C12Y115/00Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001Superoxide dismutase (1.15.1.1)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SI201631899T 2015-10-09 2016-09-30 Postopek za genetsko zdravljenje z uporabo virusa aav-xbp1s/gfp in njegova uporaba pri preprečevanju in zdravljenju amiotrofične lateralne skleroze SI4035687T1 (sl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CL2015003024A CL2015003024A1 (es) 2015-10-09 2015-10-09 Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica.
EP21209131.8A EP4035687B1 (en) 2015-10-09 2016-09-30 Method of genetic treatment using the virus aav-xbp1s/gfp, and its use in the prevention and treatment of amyotrophic lateral sclerosis

Publications (1)

Publication Number Publication Date
SI4035687T1 true SI4035687T1 (sl) 2025-07-31

Family

ID=56081079

Family Applications (2)

Application Number Title Priority Date Filing Date
SI201631899T SI4035687T1 (sl) 2015-10-09 2016-09-30 Postopek za genetsko zdravljenje z uporabo virusa aav-xbp1s/gfp in njegova uporaba pri preprečevanju in zdravljenju amiotrofične lateralne skleroze
SI201631478T SI3360580T1 (sl) 2015-10-09 2016-09-30 Postopek za genetsko zdravljenje z uporabo virusa AAV-XBP1S/GFP in njegova uporaba pri preprečevanju in zdravljenju amiotrofične lateralne skleroze

Family Applications After (1)

Application Number Title Priority Date Filing Date
SI201631478T SI3360580T1 (sl) 2015-10-09 2016-09-30 Postopek za genetsko zdravljenje z uporabo virusa AAV-XBP1S/GFP in njegova uporaba pri preprečevanju in zdravljenju amiotrofične lateralne skleroze

Country Status (14)

Country Link
US (1) US10512698B2 (sl)
EP (2) EP4035687B1 (sl)
AU (1) AU2016336063B2 (sl)
CL (1) CL2015003024A1 (sl)
CY (1) CY1125031T1 (sl)
DK (1) DK3360580T3 (sl)
ES (2) ES3031262T3 (sl)
HR (1) HRP20220199T1 (sl)
HU (1) HUE057861T2 (sl)
LT (1) LT3360580T (sl)
PL (1) PL3360580T3 (sl)
PT (1) PT3360580T (sl)
SI (2) SI4035687T1 (sl)
WO (1) WO2017059554A1 (sl)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CL2015003242A1 (es) * 2015-11-04 2016-10-14 Univ Chile Virus aav/upr-plus, proteína de fusión upr-plus, método de tratamiento genético y su uso en el tratamiento enfermedades neurodegenerativas, tales como las enfermedades de parkinson y huntington, entre otras.
EP3917624A4 (en) * 2019-02-01 2022-10-19 Universidad De Chile TREATMENT OF AGING OR AGING-RELATED DISORDERS USING XBP1
CN112143693B (zh) * 2019-06-28 2024-12-27 杭州康万达医药科技有限公司 一种生产病毒的方法及收获液组合物

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2596885T3 (es) 2006-06-07 2017-01-12 Genzyme Corporation Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la medula espinal
SI2497500T1 (sl) 2006-10-03 2017-01-31 Genzyme Corporation Genska terapija za spinalno mišično atrofijo
US20110142799A1 (en) * 2008-06-23 2011-06-16 President And Fellows Of Harvard College Modulation of neurodegenerative disease by modulating xbp-1 activity
CL2014003590A1 (es) 2014-12-30 2015-07-10 Univ Chile Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimizacion y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje.

Also Published As

Publication number Publication date
ES2907684T3 (es) 2022-04-26
EP4035687B1 (en) 2025-04-16
AU2016336063A1 (en) 2018-05-24
ES3031262T3 (en) 2025-07-07
WO2017059554A1 (es) 2017-04-13
EP4035687A1 (en) 2022-08-03
SI3360580T1 (sl) 2022-05-31
PT3360580T (pt) 2022-02-24
CY1125031T1 (el) 2023-03-24
AU2016336063B2 (en) 2023-07-13
US10512698B2 (en) 2019-12-24
EP3360580B1 (en) 2021-12-01
DK3360580T3 (da) 2022-02-21
HUE057861T2 (hu) 2022-06-28
EP3360580A4 (en) 2019-03-13
HRP20220199T1 (hr) 2022-04-29
CL2015003024A1 (es) 2016-05-13
PL3360580T3 (pl) 2022-05-23
EP3360580A1 (en) 2018-08-15
US20190030188A1 (en) 2019-01-31
LT3360580T (lt) 2022-05-10

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