PL3360580T3 - Sposób leczenia genetycznego z użyciem wirusa aav-xbp1s/gfp i jego zastosowanie do zapobiegania i leczenia stwardzenia zanikowego bocznego - Google Patents
Sposób leczenia genetycznego z użyciem wirusa aav-xbp1s/gfp i jego zastosowanie do zapobiegania i leczenia stwardzenia zanikowego bocznegoInfo
- Publication number
- PL3360580T3 PL3360580T3 PL16852956T PL16852956T PL3360580T3 PL 3360580 T3 PL3360580 T3 PL 3360580T3 PL 16852956 T PL16852956 T PL 16852956T PL 16852956 T PL16852956 T PL 16852956T PL 3360580 T3 PL3360580 T3 PL 3360580T3
- Authority
- PL
- Poland
- Prior art keywords
- treatment
- xbp1s
- aav
- prevention
- lateral
- Prior art date
Links
- 208000034189 Sclerosis Diseases 0.000 title 1
- 241000700605 Viruses Species 0.000 title 1
- 230000002068 genetic effect Effects 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 230000002265 prevention Effects 0.000 title 1
Classifications
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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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
-
- 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
- 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)
Landscapes
- 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)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- 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)
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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3360580T3 true PL3360580T3 (pl) | 2022-05-23 |
Family
ID=56081079
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL16852956T PL3360580T3 (pl) | 2015-10-09 | 2016-09-30 | Sposób leczenia genetycznego z użyciem wirusa aav-xbp1s/gfp i jego zastosowanie do zapobiegania i leczenia stwardzenia zanikowego bocznego |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US10512698B2 (pl) |
| EP (2) | EP4035687B1 (pl) |
| AU (1) | AU2016336063B2 (pl) |
| CL (1) | CL2015003024A1 (pl) |
| CY (1) | CY1125031T1 (pl) |
| DK (1) | DK3360580T3 (pl) |
| ES (2) | ES2907684T3 (pl) |
| HR (1) | HRP20220199T1 (pl) |
| HU (1) | HUE057861T2 (pl) |
| LT (1) | LT3360580T (pl) |
| PL (1) | PL3360580T3 (pl) |
| PT (1) | PT3360580T (pl) |
| SI (2) | SI3360580T1 (pl) |
| WO (1) | WO2017059554A1 (pl) |
Families Citing this family (3)
| 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. |
| US20220184175A1 (en) * | 2019-02-01 | 2022-06-16 | 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101460621B (zh) | 2006-06-07 | 2016-01-27 | 建新公司 | 肌萎缩性侧索硬化和其他脊髓失调症的基因治疗 |
| DK2066791T3 (da) | 2006-10-03 | 2012-10-15 | Genzyme Corp | Genterapi for amyotrofisk lateralsklerose og andre rygmarvssygdomme |
| 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. |
-
2015
- 2015-10-09 CL CL2015003024A patent/CL2015003024A1/es unknown
-
2016
- 2016-09-30 ES ES16852956T patent/ES2907684T3/es active Active
- 2016-09-30 PT PT168529568T patent/PT3360580T/pt unknown
- 2016-09-30 SI SI201631478T patent/SI3360580T1/sl unknown
- 2016-09-30 AU AU2016336063A patent/AU2016336063B2/en active Active
- 2016-09-30 ES ES21209131T patent/ES3031262T3/es active Active
- 2016-09-30 WO PCT/CL2016/000056 patent/WO2017059554A1/es not_active Ceased
- 2016-09-30 HR HRP20220199TT patent/HRP20220199T1/hr unknown
- 2016-09-30 EP EP21209131.8A patent/EP4035687B1/en active Active
- 2016-09-30 PL PL16852956T patent/PL3360580T3/pl unknown
- 2016-09-30 LT LTEPPCT/CL2016/000056T patent/LT3360580T/lt unknown
- 2016-09-30 DK DK16852956.8T patent/DK3360580T3/da active
- 2016-09-30 US US15/767,086 patent/US10512698B2/en active Active
- 2016-09-30 EP EP16852956.8A patent/EP3360580B1/en active Active
- 2016-09-30 HU HUE16852956A patent/HUE057861T2/hu unknown
- 2016-09-30 SI SI201631899T patent/SI4035687T1/sl unknown
-
2022
- 2022-03-01 CY CY20221100171T patent/CY1125031T1/el unknown
Also Published As
| Publication number | Publication date |
|---|---|
| ES2907684T3 (es) | 2022-04-26 |
| EP3360580B1 (en) | 2021-12-01 |
| EP4035687B1 (en) | 2025-04-16 |
| EP3360580A4 (en) | 2019-03-13 |
| HRP20220199T1 (hr) | 2022-04-29 |
| EP4035687A1 (en) | 2022-08-03 |
| AU2016336063B2 (en) | 2023-07-13 |
| HUE057861T2 (hu) | 2022-06-28 |
| EP3360580A1 (en) | 2018-08-15 |
| CL2015003024A1 (es) | 2016-05-13 |
| DK3360580T3 (da) | 2022-02-21 |
| SI3360580T1 (sl) | 2022-05-31 |
| AU2016336063A1 (en) | 2018-05-24 |
| US10512698B2 (en) | 2019-12-24 |
| WO2017059554A1 (es) | 2017-04-13 |
| LT3360580T (lt) | 2022-05-10 |
| US20190030188A1 (en) | 2019-01-31 |
| PT3360580T (pt) | 2022-02-24 |
| CY1125031T1 (el) | 2023-03-24 |
| SI4035687T1 (sl) | 2025-07-31 |
| ES3031262T3 (en) | 2025-07-07 |
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