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 PDFInfo
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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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- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 title claims 6
- 230000002265 prevention Effects 0.000 title claims 3
- 241000700605 Viruses Species 0.000 title 1
- 230000002068 genetic effect Effects 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 239000013598 vector Substances 0.000 claims 20
- 241000124008 Mammalia Species 0.000 claims 6
- 102000040430 polynucleotide Human genes 0.000 claims 5
- 108091033319 polynucleotide Proteins 0.000 claims 5
- 239000002157 polynucleotide Substances 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 239000013607 AAV vector Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 108700029229 Transcriptional Regulatory Elements Proteins 0.000 claims 2
- 210000003169 central nervous system Anatomy 0.000 claims 2
- 230000014509 gene expression Effects 0.000 claims 2
- 210000002569 neuron Anatomy 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 101150114882 CALM2 gene Proteins 0.000 claims 1
- -1 CamIIK Proteins 0.000 claims 1
- 206010018341 Gliosis Diseases 0.000 claims 1
- 101150052863 THY1 gene Proteins 0.000 claims 1
- 208000037875 astrocytosis Diseases 0.000 claims 1
- 230000007341 astrogliosis Effects 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 210000001638 cerebellum Anatomy 0.000 claims 1
- 230000001934 delay Effects 0.000 claims 1
- 230000002018 overexpression Effects 0.000 claims 1
- 101150079312 pgk1 gene Proteins 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 210000000449 purkinje cell Anatomy 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 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
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- C—CHEMISTRY; METALLURGY
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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
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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
-
- 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
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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
- A61K48/0075—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
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- 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
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- 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
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- 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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- 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
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- 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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- 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
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- 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)
- 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)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (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.
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 |
|---|---|
| HRP20220199T1 true HRP20220199T1 (hr) | 2022-04-29 |
Family
ID=56081079
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US10512698B2 (hr) |
| EP (2) | EP4035687A1 (hr) |
| AU (1) | AU2016336063B2 (hr) |
| CL (1) | CL2015003024A1 (hr) |
| CY (1) | CY1125031T1 (hr) |
| DK (1) | DK3360580T3 (hr) |
| ES (1) | ES2907684T3 (hr) |
| HR (1) | HRP20220199T1 (hr) |
| HU (1) | HUE057861T2 (hr) |
| LT (1) | LT3360580T (hr) |
| PL (1) | PL3360580T3 (hr) |
| PT (1) | PT3360580T (hr) |
| SI (1) | SI3360580T1 (hr) |
| WO (1) | WO2017059554A1 (hr) |
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. |
| EP3917624A4 (en) * | 2019-02-01 | 2022-10-19 | Universidad De Chile | TREATMENT OF AGING OR AGE-RELATED DISORDERS USING XBP1 |
| CN112143693A (zh) * | 2019-06-28 | 2020-12-29 | 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 |
|---|---|---|---|---|
| EP2489733B1 (en) | 2006-06-07 | 2019-02-13 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| PT2066791E (pt) | 2006-10-03 | 2012-12-05 | Genzyme Corp | Terapia génica para esclerose lateral amiotrófica e outros distúrbios da medula espinal |
| 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. |
-
2015
- 2015-10-09 CL CL2015003024A patent/CL2015003024A1/es unknown
-
2016
- 2016-09-30 LT LTEPPCT/CL2016/000056T patent/LT3360580T/lt unknown
- 2016-09-30 PL PL16852956T patent/PL3360580T3/pl unknown
- 2016-09-30 AU AU2016336063A patent/AU2016336063B2/en active Active
- 2016-09-30 HU HUE16852956A patent/HUE057861T2/hu unknown
- 2016-09-30 PT PT168529568T patent/PT3360580T/pt unknown
- 2016-09-30 EP EP21209131.8A patent/EP4035687A1/en active Pending
- 2016-09-30 US US15/767,086 patent/US10512698B2/en active Active
- 2016-09-30 DK DK16852956.8T patent/DK3360580T3/da active
- 2016-09-30 EP EP16852956.8A patent/EP3360580B1/en active Active
- 2016-09-30 HR HRP20220199TT patent/HRP20220199T1/hr unknown
- 2016-09-30 WO PCT/CL2016/000056 patent/WO2017059554A1/es active Application Filing
- 2016-09-30 ES ES16852956T patent/ES2907684T3/es active Active
- 2016-09-30 SI SI201631478T patent/SI3360580T1/sl unknown
-
2022
- 2022-03-01 CY CY20221100171T patent/CY1125031T1/el unknown
Also Published As
| Publication number | Publication date |
|---|---|
| DK3360580T3 (da) | 2022-02-21 |
| US20190030188A1 (en) | 2019-01-31 |
| EP3360580B1 (en) | 2021-12-01 |
| US10512698B2 (en) | 2019-12-24 |
| PT3360580T (pt) | 2022-02-24 |
| LT3360580T (lt) | 2022-05-10 |
| HUE057861T2 (hu) | 2022-06-28 |
| AU2016336063A1 (en) | 2018-05-24 |
| EP3360580A4 (en) | 2019-03-13 |
| CL2015003024A1 (es) | 2016-05-13 |
| PL3360580T3 (pl) | 2022-05-23 |
| EP4035687A1 (en) | 2022-08-03 |
| WO2017059554A1 (es) | 2017-04-13 |
| ES2907684T3 (es) | 2022-04-26 |
| SI3360580T1 (sl) | 2022-05-31 |
| AU2016336063B2 (en) | 2023-07-13 |
| CY1125031T1 (el) | 2023-03-24 |
| EP3360580A1 (en) | 2018-08-15 |
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