HRP20220776T1 - Sustav višestrukih vektora i njegova upotreba - Google Patents
Sustav višestrukih vektora i njegova upotreba Download PDFInfo
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- HRP20220776T1 HRP20220776T1 HRP20220776TT HRP20220776T HRP20220776T1 HR P20220776 T1 HRP20220776 T1 HR P20220776T1 HR P20220776T T HRP20220776T T HR P20220776TT HR P20220776 T HRP20220776 T HR P20220776T HR P20220776 T1 HRP20220776 T1 HR P20220776T1
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- 239000013598 vector Substances 0.000 title claims 23
- 108091026890 Coding region Proteins 0.000 claims 25
- 239000002773 nucleotide Substances 0.000 claims 25
- 125000003729 nucleotide group Chemical group 0.000 claims 23
- 230000015556 catabolic process Effects 0.000 claims 12
- 238000006731 degradation reaction Methods 0.000 claims 12
- 239000013607 AAV vector Substances 0.000 claims 11
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 101710083129 50S ribosomal protein L10, chloroplastic Proteins 0.000 claims 3
- 101710114762 50S ribosomal protein L11, chloroplastic Proteins 0.000 claims 3
- 101710082414 50S ribosomal protein L12, chloroplastic Proteins 0.000 claims 3
- 101710177347 50S ribosomal protein L15, chloroplastic Proteins 0.000 claims 3
- 230000008488 polyadenylation Effects 0.000 claims 3
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 2
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 2
- 208000027073 Stargardt disease Diseases 0.000 claims 2
- 241000700605 Viruses Species 0.000 claims 2
- 208000006623 congenital stationary night blindness Diseases 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 230000007170 pathology Effects 0.000 claims 2
- 230000002265 prevention Effects 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 101150039555 ABCA4 gene Proteins 0.000 claims 1
- 102100036799 Adhesion G-protein coupled receptor V1 Human genes 0.000 claims 1
- 206010068783 Alstroem syndrome Diseases 0.000 claims 1
- 201000005932 Alstrom Syndrome Diseases 0.000 claims 1
- 102100032360 Alstrom syndrome protein 1 Human genes 0.000 claims 1
- -1 CACNA1 Proteins 0.000 claims 1
- 102100022509 Cadherin-23 Human genes 0.000 claims 1
- 102100035673 Centrosomal protein of 290 kDa Human genes 0.000 claims 1
- 101710198317 Centrosomal protein of 290 kDa Proteins 0.000 claims 1
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 1
- 201000003883 Cystic fibrosis Diseases 0.000 claims 1
- 102100032248 Dysferlin Human genes 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 206010064571 Gene mutation Diseases 0.000 claims 1
- 102100028893 Hemicentin-1 Human genes 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 101000928167 Homo sapiens Adhesion G-protein coupled receptor V1 Proteins 0.000 claims 1
- 101000797795 Homo sapiens Alstrom syndrome protein 1 Proteins 0.000 claims 1
- 101000899442 Homo sapiens Cadherin-23 Proteins 0.000 claims 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 1
- 101001016184 Homo sapiens Dysferlin Proteins 0.000 claims 1
- 101000839060 Homo sapiens Hemicentin-1 Proteins 0.000 claims 1
- 101000957756 Homo sapiens Microtubule-associated protein RP/EB family member 2 Proteins 0.000 claims 1
- 101000854060 Homo sapiens Oxygen-regulated protein 1 Proteins 0.000 claims 1
- 101001105683 Homo sapiens Pre-mRNA-processing-splicing factor 8 Proteins 0.000 claims 1
- 101001028804 Homo sapiens Protein eyes shut homolog Proteins 0.000 claims 1
- 101001072259 Homo sapiens Protocadherin-15 Proteins 0.000 claims 1
- 101000801643 Homo sapiens Retinal-specific phospholipid-transporting ATPase ABCA4 Proteins 0.000 claims 1
- 101000854044 Homo sapiens Retinitis pigmentosa 1-like 1 protein Proteins 0.000 claims 1
- 101000628575 Homo sapiens Serine/threonine-protein kinase 19 Proteins 0.000 claims 1
- 101000659545 Homo sapiens U5 small nuclear ribonucleoprotein 200 kDa helicase Proteins 0.000 claims 1
- 101000805941 Homo sapiens Usherin Proteins 0.000 claims 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 1
- 208000035719 Maculopathy Diseases 0.000 claims 1
- 102000026889 Myosin VIIa Human genes 0.000 claims 1
- 108010009047 Myosin VIIa Proteins 0.000 claims 1
- 102100021231 Pre-mRNA-processing-splicing factor 8 Human genes 0.000 claims 1
- 102100037166 Protein eyes shut homolog Human genes 0.000 claims 1
- 102100036382 Protocadherin-15 Human genes 0.000 claims 1
- 208000022583 Qualitative or quantitative defects of dysferlin Diseases 0.000 claims 1
- 201000007737 Retinal degeneration Diseases 0.000 claims 1
- 102100033617 Retinal-specific phospholipid-transporting ATPase ABCA4 Human genes 0.000 claims 1
- 102100035670 Retinitis pigmentosa 1-like 1 protein Human genes 0.000 claims 1
- 101001128051 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) 60S ribosomal protein L3 Proteins 0.000 claims 1
- 102100026757 Serine/threonine-protein kinase 19 Human genes 0.000 claims 1
- 102100036230 U5 small nuclear ribonucleoprotein 200 kDa helicase Human genes 0.000 claims 1
- 102100037930 Usherin Human genes 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000004927 fusion Effects 0.000 claims 1
- 238000001415 gene therapy Methods 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 claims 1
- 201000006756 occult macular dystrophy Diseases 0.000 claims 1
- 239000000825 pharmaceutical preparation Substances 0.000 claims 1
- 210000001525 retina Anatomy 0.000 claims 1
- 230000004258 retinal degeneration Effects 0.000 claims 1
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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
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- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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Claims (13)
1. Sustav višestrukih vektora AAV za ekspresiju kodirajuće sekvence gena od interesa u stanici, navedena kodirajuća sekvenca sadrži prvi dio i drugi dio, navedeni vektorski sustav sadrži:
a) prvi vektor koji sadrži:
- navedeni prvi dio navedene kodirajuće sekvence (CDS1),
- prvu rekonstitucijsku sekvencu; i
b) drugi vektor koji sadrži:
- navedeni drugi dio navedene kodirajuće sekvence (CDS2),
- drugu rekonstitucijsku sekvencu,
pri čemu su navedena prva i druga rekonstitucijska sekvenca odabrane iz skupine:
i] prva rekonstitucijska sekvenca se sastoji od 3' kraja navedenog prvog dijela kodirajuće sekvence, a druga rekonstitucijska sekvenca se sastoji od 5' kraja navedenog drugog dijela kodirajuće sekvence, pri čemu su navedena prva i druga rekonstitucijska sekvenca preklapajuće sekvence; ili
ii] prva rekonstitucijska sekvenca sadrži signal donora za spajanje (SD) i druga rekonstitucijska sekvenca sadrži signal akceptora spajanja (SA), izborno svaka od prve i druge rekonstitucijske sekvence nadalje sadrži rekombinogenu sekvencu,
naznačen time što bilo jedan ili oba od prvog i drugog vektora nadalje sadrže nukleotidnu sekvencu signala degradacije, pri čemu se navedena sekvenca nalazi u slučaju i) na 3' kraju od CDS1 i/ili na 5' kraju od CDS2 i u slučaju ii) u 3' položaju u odnosu na SD i/ili u 5' položaju u odnosu na SA, pri čemu
- rekombinogena sekvenca je odabrana iz skupine koju čine:
GGGATTTTGCCGATTTCGGCCTATTGGTTAAAAAATGAGCTGATTTAACAAAAATTT AACGCGAATTTTAACAAAAT (SEQ ID No. 22, AK) ili
GGGATTTTTCCGATTTCGGCCTATTGGTTAAAAAATGAGCTGATTTAACAAAAATTT AACGCGAATTTTAACAAAAT (SEQ ID NO. 23, AK), SEQ ID NO. 24 (AP1), SEQ ID NO. 25 (AP2), i SEQ ID NO. 26 (AP) i
- nukleotidna sekvenca signala degradacije sadrži ili se sastoji od sekvence koja kodira SEQ ID No. 1 (CL1), SEQ ID No. 2 (CL2), SEQ ID No. 3 (CL6), SEQ ID No. 4 (CL9), SEQ ID No. 5 (CL10), SEQ ID No. 6 (CL11), SEQ ID No. 7 (CL12), SEQ ID No. 8 (CL15), SEQ ID No. 9 (CL16), ili nukleotidna sekvenca signala degradacije sadrži ili se sastoji od SEQ ID No. 16.
2. Sustav višestrukih vektora AAV prema patentnom zahtjevu 1, naznačen time što oba prvi i drugi vektor dalje sadrže navedenu nukleotidnu sekvencu signala degradacije, pri čemu je nukleotidna sekvenca signala degradacije u prvom vektoru identična ili se razlikuje od one u drugom vektoru, ili pri čemu prva rekonstitucijska sekvenca sadrži signal donora za spajanje (SD) i rekombinogenu regiju na 3' položaju u odnosu na navedeni SD, druga rekonstitucijska sekvenca sadrži signal akceptora spajanja (SA) i rekombinogenu sekvencu u 5' položaju u odnosu na SA; pri čemu je navedena nukleotidna sekvenca signala degradacije lokalizirana na 5' kraju i/ili na 3' kraju nukleotidne sekvence rekombinogene regije bilo jednog ili oba prvog i drugog vektora.
3. Sustav višestrukih vektora AAV prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time što prvi vektor dalje sadrži promotorsku sekvencu koja je operativno povezana s 5' krajnjim dijelom navedenog prvog dijela kodirajuće sekvence (CDS1).
4. Sustav višestrukih vektora AAV prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time što oba prvi i drugi vektor dalje sadrže nukleotidnu sekvencu 5'-terminalnog ponavljanja (5'-TR) i nukleotidnu sekvencu 3'-terminalnog ponavljanja (3'-TR), poželjno 5'-TR je nukleotidna sekvenca 5'-invertiranog terminalnog ponavljanja i 3'-TR je nukleotidna sekvenca 3'-invertiranog terminalnog ponavljanja, poželjno ITR-ovi potječu od istog serotipa virusa ili od različitih serotipova virusa.
5. Sustav višestrukih vektora AAV prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time što signal donora za spajanje sadrži ili se sastoji od sekvence koja je najmanje 70%, 75%, 80%, 85%, 90%, 95% ili 100 % identična sa
GTAAGTATCAAGGTTACAAGACAGGTTTAAGGAGACCAATAGAAACTGGGCTTGTC GAGACAGAGAAGACTCTTGCGTTTCT (SEQ ID No. 27), ili pri čemu signal akceptora spajanja sadrži ili se sastoji od sekvence koja je najmanje 70%, 75%, 80%, 85%, 90%, 95% ili 100 % identična sa
GATAGGCACCTATTGGTCTTACTGACATCCACTTTGCCTTTCTCTCCACAG (SEQ ID No. 28)
6. Sustav višestrukih vektora AAV prema bilo kojem prethodnom patentnom zahtjevu naznačen time je kodirajuća sekvenca prvog vektora kodirajuća sekvenca gena odabranog iz skupine koju čine: ABCA4, MYO7A, CEP290, CDH23, EYS, PCDH15, CACNA1, SNRNP200, RP1, PRPF8, RP1L1, ALMS1, USH2A, GPR98, HMCN1, poželjno kodirajuća sekvenca je kodirajuća sekvenca gena odabranog iz skupine koju čine: DMD, CFTR, F8 i DYSF.
7. Sustav višestrukih vektora AAV prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time što prvi vektor ne sadrži poli-adenilacijsku signalnu nukleotidnu sekvencu.
8. Sustav višestrukih vektora AAV prema bilo kojem od prethodnih patentnih zahtjeva naznačen time što sadrži:
a) prvi vektor koji sadrži u smjeru 5'-3':
- sekvencu 5'-invertiranog terminalnog ponavljanja (5'-ITR);
- promotorsku sekvencu;
- 5' krajnji dio kodirajuće sekvence gena od interesa (CDS1), pri čemu je navedeni 5' krajnji dio operativno vezan za i pod kontrolom navedenog promotora;
- nukleotidnu sekvencu signala donora za spajanje;
- nukleotidnu sekvencu rekombinogene regije; i
- sekvencu 3'-invertiranog terminalnog ponavljanja (3'-ITR); i
b) drugi vektor koji sadrži u smjeru 5'-3':
- sekvencu 5'-invertiranog terminalnog ponavljanja (5'-ITR);
- nukleotidnu sekvencu rekombinogene regije;
- nukleotidnu sekvencu signala akceptora spajanja;
- 3' kraj kodirajuće sekvence (CDS2);
- poli-adenilacijsku signalnu nukleotidnu sekvencu; i
- sekvencu 3'-invertiranog terminalnog ponavljanja (3'-ITR),
naznačen time što dalje sadrži nukleotidnu sekvencu signala degradacije, pri čemu je navedena sekvenca lokalizirana na 5' kraju ili 3' kraju nukleotidne sekvence rekombinogene regije bilo jednog ili oba od prvog i drugog vektora, pri čemu
- rekombinogena sekvenca je odabrana iz skupine koju čine:
GGGATTTTGCCGATTTCGGCCTATTGGTTAAAAAATGAGCTGATTTAACAAAAATTT AACGCGAATTTTAACAAAAT (SEQ ID No. 22, AK) ili
GGGATTTTTCCGATTTCGGCCTATTGGTTAAAAAATGAGCTGATTTAACAAAAATTT AACGCGAATTTTAACAAAAT (SEQ ID NO. 23, AK), SEQ ID NO. 24 (AP1), SEQ ID NO. 25 (AP2) i SEQ ID NO. 26 (AP) i
- nukleotidna sekvenca signala degradacije sadrži ili se sastoji od sekvence koja kodira SEQ ID No. 1 (CL1), SEQ ID No. 2 (CL2), SEQ ID No. 3 (CL6), SEQ ID No. 4 (CL9), SEQ ID No. 5 (CL10), SEQ ID No. 6 (CL11), SEQ ID No. 7 (CL12), SEQ ID No. 8 (CL15), SEQ ID No. 9 (CL16), ili nukleotidna sekvenca signala degradacije sadrži ili se sastoji od SEQ ID No. 16.
9. Sustav višestrukih vektora AAV prema bilo kojem od prethodnih patentnih zahtjeva nadalje sadrži treći vektor koji sadrži treći dio navedene kodirajuće sekvence (CDS3) i rekonstitucijsku sekvencu, pri čemu drugi vektor sadrži dvije rekonstitucijske sekvence, pri čemu se svaka rekonstitucijska sekvenca nalazi na svakom kraju CDS2, poželjno treći vektor dalje sadrži najmanje jednu nukleotidnu sekvencu signala degradacije, poželjno drugi vektor dalje sadrži poli-adenilacijsku signalnu nukleotidnu sekvencu povezanu s 3'-krajnjim dijelom navedene kodirajuće sekvence (CDS2).
10. Stanica domaćin koja sadrži sustav višestrukih vektora AAV prema bilo kojem prethodnom zahtjevu.
11. Sustav višestrukih vektora AAV prema bilo kojem od patentnih zahtjeva 1 do 9 ili stanica domaćin prema patentnom zahtjevu 10 za medicinsku upotrebu, poželjno za upotrebu u genskoj terapiji, poželjno za upotrebu u liječenju i/ili prevenciji patologije ili bolesti naznačene time što je degeneracija mrežnice, poželjno degeneracija mrežnice je naslijeđena, poželjno je da se patologija ili bolest bira iz skupine koju čine: retinitis pigmentosa (RP), Leberova urođena amauroza (LCA), Stargardtova bolest (STGD), Usherova bolest (USH), Alstromov sindrom, urođena stacionarna noćna sljepoća (CSNB), makularna distrofija, okultna makularna distrofija, bolest uzrokovana mutacijom gena ABCA4, poželjno za upotrebu u prevenciji i/ili liječenju Duchenneove mišićne distrofije, cistične fibroze, hemofilije A i disferlinopatije.
12. Farmaceutski pripravak naznačen time što sadrži Sustav višestrukih vektora AAV prema bilo kojem od patentnih zahtjeva 1 do 9 ili stanicu domaćina prema zahtjevu 10 i farmaceutski prihvatljiv nosač.
13. Nukleotidna sekvenca signala degradacije koja sadrži ili se sastoji od sekvence koja kodira SEQ ID No. 1 (CL1), SEQ ID No. 2 (CL2), SEQ ID No. 3 (CL6), SEQ ID No. 4 (CL9), SEQ ID No. 5 (CL10), SEQ ID No. 6 (CL11), SEQ ID No. 7 (CL12), SEQ ID No. 8 (CL15), SEQ ID No. 9 (CL16), ili koja sadrži ili se sastoji od SEQ ID No. 16 u AAV višestrukom vektorskom sustavu za upotrebu u postupku za smanjenje ekspresije proteina u skraćenom obliku ili za upotrebu u postupku za smanjenje ekspresije proteina u skraćenom obliku koji obuhvaća umetanje navedene nukleotidne sekvence signala degradacije u jedan ili više vektora AAV viševektorskog sustava.
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| EP3719134B1 (en) * | 2015-03-11 | 2023-08-23 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Rp2 vectors for treating x-linked retinitis pigmentosa |
| US11072803B2 (en) | 2015-12-22 | 2021-07-27 | Inserm (Institut National De La Sante Et De La Recherche Medicale) | Hybrid dual recombinant AAV vector systems for gene therapy |
| JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
| CN110892062A (zh) * | 2017-05-05 | 2020-03-17 | 佛罗里达大学研究基金会 | 表达耳畸蛋白的组合物和方法 |
| CN113056561A (zh) * | 2018-04-05 | 2021-06-29 | 牛津大学科技创新有限公司 | 用于治疗黄斑营养不良的组合物和方法 |
| EP3781213A4 (en) * | 2018-04-17 | 2022-10-19 | The Trustees of the University of Pennsylvania | TRANS-SPLICE MOLECULES |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| EP3867387A2 (en) * | 2018-10-15 | 2021-08-25 | Fondazione Telethon | Intein proteins and uses thereof |
| EP3870148A4 (en) * | 2018-10-25 | 2022-11-09 | Takeda Pharmaceutical Company Limited | AAV TRIPLE PLASMID SYSTEM |
| EP3956453A4 (en) * | 2019-04-19 | 2023-02-22 | University of Massachusetts | GENE THERAPIES FOR USHER SYNDROME (USH1B) |
| US20220315948A1 (en) * | 2019-04-26 | 2022-10-06 | President And Fellows Of Harvard College | Aav vectors encoding mini-pcdh15 and uses thereof |
| WO2021116138A1 (en) * | 2019-12-09 | 2021-06-17 | Ucl Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
| EP4100518A2 (en) * | 2020-02-07 | 2022-12-14 | The Children's Medical Center Corporation | Large gene vectors and delivery and uses thereof |
| AU2021225035A1 (en) | 2020-02-21 | 2022-10-13 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| CN112501209B (zh) * | 2020-12-07 | 2024-02-13 | 和元生物技术（上海）股份有限公司 | 外源基因可控表达的腺相关病毒包装方法 |
| CA3218195A1 (en) * | 2021-05-07 | 2022-11-10 | Robin Ali | Abca4 genome editing |
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| US5827702A (en) | 1994-10-31 | 1998-10-27 | Genentech, Inc. | Ocular gene therapy |
| GB9720465D0 (en) * | 1997-09-25 | 1997-11-26 | Oxford Biomedica Ltd | Dual-virus vectors |
| US6106826A (en) | 1997-12-17 | 2000-08-22 | Wisconsin Alumni Research Foundation | Replication competent, avirulent Herpes simplex virus as a vector for neural and ocular gene therapy |
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| JP4948845B2 (ja) * | 2006-02-08 | 2012-06-06 | 財団法人 東京都医学総合研究所 | 神経変性疾患治療用物質のスクリーニング方法 |
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| WO2007127428A2 (en) | 2006-04-28 | 2007-11-08 | University Of Florida Research Foundation, Inc. | Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery |
| US8236557B2 (en) * | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| WO2010059763A1 (en) * | 2008-11-19 | 2010-05-27 | Amyris Biotechnologies, Inc. | Compositions and methods for the assembly of polynucleotides |
| AU2010267963B2 (en) * | 2009-07-02 | 2015-09-24 | Merz Pharma Gmbh & Co. Kgaa | Neurotoxins exhibiting shortened biological activity |
| ES2704677T3 (es) * | 2013-04-18 | 2019-03-19 | Fond Telethon | Suministro eficaz de genes grandes por vectores de AAV duales |
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| BR112017018728A2 (pt) | 2018-04-17 |
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| KR102240180B1 (ko) | 2021-04-14 |
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| PT3265571T (pt) | 2022-06-29 |
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| RS63416B1 (sr) | 2022-08-31 |
| EP4089172A2 (en) | 2022-11-16 |
| MX2023004479A (es) | 2023-05-04 |
| CA2979120A1 (en) | 2016-09-09 |
| AU2016227668A1 (en) | 2017-08-31 |
| ES2919880T3 (es) | 2022-07-28 |
| US20220002749A1 (en) | 2022-01-06 |
| US20180327779A1 (en) | 2018-11-15 |
| EP3265571A1 (en) | 2018-01-10 |
| SI3265571T1 (sl) | 2022-10-28 |
| JP2022174192A (ja) | 2022-11-22 |
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