DK3488007T3 - Skalerbare fremgangsmåder med høj indvinding til fremstilling af højt udbytte rekombinant adeno-associeret viral (raav) vektor og rekombinante adeno-associeret virale (raav) vektorer produceret derved - Google Patents
Skalerbare fremgangsmåder med høj indvinding til fremstilling af højt udbytte rekombinant adeno-associeret viral (raav) vektor og rekombinante adeno-associeret virale (raav) vektorer produceret derved Download PDFInfo
- Publication number
- DK3488007T3 DK3488007T3 DK17831956.2T DK17831956T DK3488007T3 DK 3488007 T3 DK3488007 T3 DK 3488007T3 DK 17831956 T DK17831956 T DK 17831956T DK 3488007 T3 DK3488007 T3 DK 3488007T3
- Authority
- DK
- Denmark
- Prior art keywords
- raav
- high yield
- associated viral
- recombinant adeno
- scalable
- Prior art date
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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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
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
- C12N15/861—Adenoviral vectors
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
- C12N7/02—Recovery or purification
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/485—Exopeptidases (3.4.11-3.4.19)
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6472—Cysteine endopeptidases (3.4.22)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
- C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Toxicology (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662365312P | 2016-07-21 | 2016-07-21 | |
| PCT/US2017/043291 WO2018017956A2 (en) | 2016-07-21 | 2017-07-21 | Scalable high recovery methods for producing high yield recombinant adeno-associated viral (raav) vector and recombinant adeno-associated viral (raav) vectors produced thereby |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3488007T3 true DK3488007T3 (da) | 2024-02-19 |
Family
ID=60992897
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK17831956.2T DK3488007T3 (da) | 2016-07-21 | 2017-07-21 | Skalerbare fremgangsmåder med høj indvinding til fremstilling af højt udbytte rekombinant adeno-associeret viral (raav) vektor og rekombinante adeno-associeret virale (raav) vektorer produceret derved |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US20190284576A1 (da) |
| EP (1) | EP3488007B1 (da) |
| JP (1) | JP7153009B2 (da) |
| KR (3) | KR20190045174A (da) |
| CN (1) | CN109790549A (da) |
| AU (1) | AU2017299779B2 (da) |
| CA (1) | CA3031095A1 (da) |
| DK (1) | DK3488007T3 (da) |
| ES (1) | ES2972859T3 (da) |
| FI (1) | FI3488007T3 (da) |
| IL (2) | IL301963A (da) |
| MX (1) | MX2019000882A (da) |
| MY (1) | MY194198A (da) |
| PT (1) | PT3488007T (da) |
| RU (1) | RU2768045C2 (da) |
| SG (1) | SG11201900366QA (da) |
| WO (1) | WO2018017956A2 (da) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| EP3510161A4 (en) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| FI3953483T3 (fi) | 2019-04-11 | 2023-11-30 | Regenxbio Inc | Kokoekskluusiokromatografiamenetelmiä rekombinanttien adenoassosioituneiden viruskoostumusten karakterisoimiseksi |
| CN110499287B (zh) * | 2019-08-30 | 2021-07-23 | 博雅干细胞科技有限公司 | 简易制备胎盘间充质干细胞外泌体的方法 |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| KR102167829B1 (ko) * | 2020-02-10 | 2020-10-20 | 주식회사 이노테라피 | 아데노연관바이러스용 안정화제 및 이를 이용한 아데노연관바이러스의 안정화 방법 |
| KR20220157944A (ko) | 2020-02-21 | 2022-11-29 | 아카우오스, 인크. | 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법 |
| SI3919613T1 (sl) * | 2020-06-05 | 2024-10-30 | Sartorius Bia Separations D.O.O. | Izboljšana izolacija adeno-asociiranega virusa za učinkovitejše odstranjevanje kontaminirane dna |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| CN116529389A (zh) * | 2020-08-20 | 2023-08-01 | 贝克顿迪金森公司 | 用于从血培养物中分离细菌的血细胞裂解剂 |
| WO2023085382A1 (ja) * | 2021-11-12 | 2023-05-19 | Ａｇｃ株式会社 | ウイルスの精製方法 |
| JP2023141423A (ja) * | 2022-03-24 | 2023-10-05 | 国立大学法人 東京大学 | ウイルスの精製方法 |
| WO2026070787A1 (ja) * | 2024-09-30 | 2026-04-02 | 富士フイルム和光純薬株式会社 | 脂質二重膜粒子の製造方法及び溶液組成物 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE360683T1 (de) | 2002-05-14 | 2007-05-15 | Merck & Co Inc | Verfahren zur reinigung von adenovirus |
| BRPI0511764B8 (pt) * | 2004-06-01 | 2021-05-25 | Avigen Inc | método de prevenção de agregação de vírions de vírus adeno-associado recombinante (raav) em uma preparação purificada de virions raav |
| GT200600027A (es) * | 2005-01-25 | 2006-08-31 | Protectores contra el esfuerzo cortante en la microfiltracion de la cosecha | |
| FR2884255B1 (fr) * | 2005-04-11 | 2010-11-05 | Vivalis | Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe |
| CN102257134B (zh) * | 2008-02-12 | 2014-03-05 | 赛诺菲巴斯德有限公司 | 使用离子交换和凝胶过滤色谱进行痘病毒纯化的方法 |
| EP2344634A4 (en) * | 2008-09-24 | 2012-08-29 | Medimmune Llc | METHODS OF CELL CULTURE, AND PROPAGATION AND PURIFICATION OF VIRUSES |
| FR2944292B1 (fr) * | 2009-04-08 | 2013-08-23 | Sanofi Pasteur | Procede de purification du virus rabique |
| WO2011094198A1 (en) * | 2010-01-28 | 2011-08-04 | The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| WO2012057363A1 (ja) * | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| DK2970948T3 (da) * | 2013-03-15 | 2019-04-08 | Glaxosmithkline Biologicals Sa | Fremgangsmåder til rna-oprensning |
| CN105612250A (zh) * | 2013-10-07 | 2016-05-25 | 诺华股份有限公司 | 经处理的滤器 |
| EP3054007A1 (en) * | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step |
| AU2016366560A1 (en) | 2015-12-11 | 2018-06-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
-
2017
- 2017-07-21 IL IL301963A patent/IL301963A/en unknown
- 2017-07-21 RU RU2019104759A patent/RU2768045C2/ru active
- 2017-07-21 CN CN201780045164.8A patent/CN109790549A/zh active Pending
- 2017-07-21 MY MYPI2019000137A patent/MY194198A/en unknown
- 2017-07-21 FI FIEP17831956.2T patent/FI3488007T3/fi active
- 2017-07-21 KR KR1020197005094A patent/KR20190045174A/ko not_active Ceased
- 2017-07-21 JP JP2019502241A patent/JP7153009B2/ja active Active
- 2017-07-21 KR KR1020247026900A patent/KR20240126073A/ko not_active Withdrawn
- 2017-07-21 ES ES17831956T patent/ES2972859T3/es active Active
- 2017-07-21 US US16/319,216 patent/US20190284576A1/en active Pending
- 2017-07-21 AU AU2017299779A patent/AU2017299779B2/en active Active
- 2017-07-21 PT PT178319562T patent/PT3488007T/pt unknown
- 2017-07-21 WO PCT/US2017/043291 patent/WO2018017956A2/en not_active Ceased
- 2017-07-21 SG SG11201900366QA patent/SG11201900366QA/en unknown
- 2017-07-21 CA CA3031095A patent/CA3031095A1/en active Pending
- 2017-07-21 MX MX2019000882A patent/MX2019000882A/es unknown
- 2017-07-21 EP EP17831956.2A patent/EP3488007B1/en active Active
- 2017-07-21 DK DK17831956.2T patent/DK3488007T3/da active
- 2017-07-21 KR KR1020227040710A patent/KR102695908B1/ko active Active
-
2019
- 2019-01-16 IL IL264270A patent/IL264270A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| JP2019524101A (ja) | 2019-09-05 |
| RU2768045C2 (ru) | 2022-03-23 |
| KR20190045174A (ko) | 2019-05-02 |
| AU2017299779B2 (en) | 2021-12-02 |
| JP7153009B2 (ja) | 2022-10-13 |
| MY194198A (en) | 2022-11-21 |
| IL264270A (en) | 2019-02-28 |
| CN109790549A (zh) | 2019-05-21 |
| MX2019000882A (es) | 2019-08-12 |
| US20190284576A1 (en) | 2019-09-19 |
| FI3488007T3 (fi) | 2024-03-19 |
| IL301963A (en) | 2023-06-01 |
| KR102695908B1 (ko) | 2024-08-14 |
| RU2019104759A3 (da) | 2020-11-17 |
| EP3488007A2 (en) | 2019-05-29 |
| EP3488007A4 (en) | 2020-01-01 |
| BR112019001146A2 (pt) | 2019-04-30 |
| WO2018017956A3 (en) | 2018-03-08 |
| KR20240126073A (ko) | 2024-08-20 |
| KR20220162834A (ko) | 2022-12-08 |
| AU2017299779A1 (en) | 2019-01-31 |
| WO2018017956A2 (en) | 2018-01-25 |
| EP3488007B1 (en) | 2023-12-20 |
| SG11201900366QA (en) | 2019-02-27 |
| RU2019104759A (ru) | 2020-08-21 |
| CA3031095A1 (en) | 2018-01-25 |
| ES2972859T3 (es) | 2024-06-17 |
| PT3488007T (pt) | 2024-03-14 |
| NZ750016A (en) | 2025-09-26 |
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