HRP20220199T1 - Postupak genetskog liječenja korištenjem virusa aav-xbp1s/gfp i njegova uporaba u prevenciji i liječenju amiotrofične lateralne skleroze - Google Patents

Postupak genetskog liječenja korištenjem virusa aav-xbp1s/gfp i njegova uporaba u prevenciji i liječenju amiotrofične lateralne skleroze Download PDF

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HRP20220199T1
HRP20220199T1 HRP20220199TT HRP20220199T HRP20220199T1 HR P20220199 T1 HRP20220199 T1 HR P20220199T1 HR P20220199T T HRP20220199T T HR P20220199TT HR P20220199 T HRP20220199 T HR P20220199T HR P20220199 T1 HRP20220199 T1 HR P20220199T1
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Croatia
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vector
use according
aav
treatment
mammal
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HRP20220199TT
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Claudio Andrés HETZ FLORES
Vicente Spiro VALENZUELA PATERAKIS
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Universidad De Chile
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0066—Manipulation 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
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds 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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    • C12N15/00—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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
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    • 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
    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)

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  • Health & Medical Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)

Claims (15)

1. Vektor za upotrebu u prevenciji, liječenju i/ili odgađanju amiotrofične lateralne skleroze (ALS) kod sisavca, naznačen time što vektor inducira prekomjernu ekspresiju neurona X-Box proteina 1 (XBP1) u središnjem živčanom sustavu (SŽS) sisavca, i pri čemu je vektor, vektor adeno-povezanog virusa (AAV).
2. Vektor za upotrebu prema patentnom zahtjevu 1, naznačen time što navedeni vektor AAV sadrži rekombinantnu adeno-povezanu virusnu sekvencu koja sadrži ekspresijsku kasetu koja sadrži transkripcijski regulacijski element za ekspresiju u živčanim tkivima koji je operativno povezan s polinukleotidom koji kodira XBP1.
3. Vektor za upotrebu prema patentnom zahtjevu 2, naznačen time što navedeni transkripcijski regulacijski element sadrži promotor.
4. Vektor za upotrebu prema patentnom zahtjevu 3, naznačen time što je navedeni promotor odabran iz skupine koju čine E F-1, cmv, cba, Pgk1, Cam2, CamIIK, ChAT i Thy1.
5. Vektor za upotrebu prema bilo kojem od patentnih zahtjeva 2 do 4, naznačen time što navedena rekombinantna sekvenca adeno-povezanog virusa nadalje sadrži AAV invertirana terminalna ponavljanja (ITR-ove).
6. Vektor za upotrebu prema patentnom zahtjevu 5, naznačen time što su navedeni ITR-ovi izvedeni iz AAV serotipa odabranog iz skupine koju čine AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11.
7. Vektor za upotrebu prema patentnom zahtjevu 6, naznačen time što su navedeni ITR-ovi izvedeni iz serotipa AAV2.
8. Vektor za upotrebu prema bilo kojem od patentnih zahtjeva 2 do 7, naznačen time što navedeni polinukleotid koji kodira XBP1 djeluje sustavno, blizu ili unutar neuronskih stanica.
9. Vektor za upotrebu prema patentnom zahtjevu 8, naznačen time što je navedeni polinukleotid koji kodira XBP1 specifičan za stanice u korteksu i leđnoj moždini, i/ili Purkinjeove stanice u malom mozgu.
10. Vektor za upotrebu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što navedeni vektor stvara smanjenje astroglioze kod sisavca.
11. Vektor za upotrebu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što navedeni vektor odgađa simptomatsku fazu fenotipa amiotrofične lateralne skleroze kod sisavca.
12. Vektor za upotrebu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što je navedeni sisavac čovjek.
13. Vektor za upotrebu prema bilo kojem od patentnih zahtjeva 2 do 12, naznačen time što polinukleotid sadrži sekvencu kako je definirana u SEQ ID NO:2.
14. Vektor za upotrebu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što vektor AAV sadrži polinukleotidnu sekvencu kako je definirana u SEQ ID NO:1.
15. Farmaceutski pripravak koji sadrži vektor prema bilo kojem od zahtjeva 1 do 14, i farmaceutski prihvatljivu pomoćnu tvar, za upotrebu u prevenciji, liječenju i/ili odgađanju amiotrofične lateralne skleroze (ALS) kod sisavaca.
HRP20220199TT 2015-10-09 2016-09-30 Postupak genetskog liječenja korištenjem virusa aav-xbp1s/gfp i njegova uporaba u prevenciji i liječenju amiotrofične lateralne skleroze HRP20220199T1 (hr)

Applications Claiming Priority (3)

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.
EP16852956.8A EP3360580B1 (en) 2015-10-09 2016-09-30 Method for genetic treatment using the aav-xbp1s/gfp virus and use thereof in the prevention and treatment of amyotrophic lateral sclerosis
PCT/CL2016/000056 WO2017059554A1 (es) 2015-10-09 2016-09-30 Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrófica

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HRP20220199T1 true HRP20220199T1 (hr) 2022-04-29

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HRP20220199TT HRP20220199T1 (hr) 2015-10-09 2016-09-30 Postupak genetskog liječenja korištenjem virusa aav-xbp1s/gfp i njegova uporaba u prevenciji i liječenju amiotrofične lateralne skleroze

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US (1) US10512698B2 (hr)
EP (2) EP3360580B1 (hr)
AU (1) AU2016336063B2 (hr)
CL (1) CL2015003024A1 (hr)
CY (1) CY1125031T1 (hr)
DK (1) DK3360580T3 (hr)
ES (2) ES3031262T3 (hr)
HR (1) HRP20220199T1 (hr)
HU (1) HUE057861T2 (hr)
LT (1) LT3360580T (hr)
PL (1) PL3360580T3 (hr)
PT (1) PT3360580T (hr)
SI (2) SI3360580T1 (hr)
WO (1) WO2017059554A1 (hr)

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.
WO2020157700A1 (en) * 2019-02-01 2020-08-06 Universidad De Chile Treatment of aging or age-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
BRPI0711965A2 (pt) 2006-06-07 2012-01-24 Genzyme Corporation terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal
DK2066791T3 (da) 2006-10-03 2012-10-15 Genzyme Corp Genterapi for amyotrofisk lateralsklerose og andre rygmarvssygdomme
WO2010008860A1 (en) * 2008-06-23 2010-01-21 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.

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Publication number Publication date
DK3360580T3 (da) 2022-02-21
EP3360580A1 (en) 2018-08-15
ES2907684T3 (es) 2022-04-26
AU2016336063B2 (en) 2023-07-13
CY1125031T1 (el) 2023-03-24
EP3360580A4 (en) 2019-03-13
WO2017059554A1 (es) 2017-04-13
PL3360580T3 (pl) 2022-05-23
HUE057861T2 (hu) 2022-06-28
AU2016336063A1 (en) 2018-05-24
LT3360580T (lt) 2022-05-10
US20190030188A1 (en) 2019-01-31
CL2015003024A1 (es) 2016-05-13
SI3360580T1 (sl) 2022-05-31
EP4035687B1 (en) 2025-04-16
EP3360580B1 (en) 2021-12-01
US10512698B2 (en) 2019-12-24
SI4035687T1 (sl) 2025-07-31
PT3360580T (pt) 2022-02-24
EP4035687A1 (en) 2022-08-03
ES3031262T3 (en) 2025-07-07

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