HRP20210139T1 - Sastavi i postupci za korigiranje mišične distrofije donjih udova tipa 2c upotrebom preskakanja egzona - Google Patents
Sastavi i postupci za korigiranje mišične distrofije donjih udova tipa 2c upotrebom preskakanja egzona Download PDFInfo
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- HRP20210139T1 HRP20210139T1 HRP20210139TT HRP20210139T HRP20210139T1 HR P20210139 T1 HRP20210139 T1 HR P20210139T1 HR P20210139T T HRP20210139T T HR P20210139TT HR P20210139 T HRP20210139 T HR P20210139T HR P20210139 T1 HRP20210139 T1 HR P20210139T1
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- antisense oligonucleotide
- oligonucleotide according
- modified
- pharmaceutical composition
- muscular dystrophy
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- 239000000203 mixture Substances 0.000 title claims 2
- 208000033136 Gamma-sarcoglycan-related limb-girdle muscular dystrophy R5 Diseases 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 29
- 239000000074 antisense oligonucleotide Substances 0.000 claims 22
- 238000012230 antisense oligonucleotides Methods 0.000 claims 22
- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 201000006938 muscular dystrophy Diseases 0.000 claims 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- 102100021792 Gamma-sarcoglycan Human genes 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- 102000019344 Gamma-sarcoglycan Human genes 0.000 claims 2
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims 2
- 108010083379 Sarcoglycans Proteins 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 230000004700 cellular uptake Effects 0.000 claims 2
- 230000002401 inhibitory effect Effects 0.000 claims 2
- 210000003141 lower extremity Anatomy 0.000 claims 2
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- 230000004220 muscle function Effects 0.000 claims 2
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- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 1
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 1
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims 1
- 102100034343 Integrase Human genes 0.000 claims 1
- 101710203526 Integrase Proteins 0.000 claims 1
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 1
- 239000002202 Polyethylene glycol Substances 0.000 claims 1
- 101710149951 Protein Tat Proteins 0.000 claims 1
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- 230000001413 cellular effect Effects 0.000 claims 1
- BOKOVLFWCAFYHP-UHFFFAOYSA-N dihydroxy-methoxy-sulfanylidene-$l^{5}-phosphane Chemical compound COP(O)(O)=S BOKOVLFWCAFYHP-UHFFFAOYSA-N 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 102000034240 fibrous proteins Human genes 0.000 claims 1
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- 102000006495 integrins Human genes 0.000 claims 1
- 108010044426 integrins Proteins 0.000 claims 1
- YACKEPLHDIMKIO-UHFFFAOYSA-L methylphosphonate(2-) Chemical compound CP([O-])([O-])=O YACKEPLHDIMKIO-UHFFFAOYSA-L 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 210000004165 myocardium Anatomy 0.000 claims 1
- 230000007170 pathology Effects 0.000 claims 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-N phosphoramidic acid Chemical compound NP(O)(O)=O PTMHPRAIXMAOOB-UHFFFAOYSA-N 0.000 claims 1
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- 229920001223 polyethylene glycol Polymers 0.000 claims 1
- 210000003019 respiratory muscle Anatomy 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 239000000758 substrate Substances 0.000 claims 1
- 210000001364 upper extremity Anatomy 0.000 claims 1
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- 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
- C12N15/1138—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 against receptors or cell surface proteins
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- 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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Claims (18)
1. Izolirani antisens oligonukleotid (AON) izabran iz grupe koja se sastoji od oligonukleotida sa sekvencom navedenom u SEQ ID NO: 4-9, 11, 12, 14-29 i 31-34, pri čemu antisens oligonukleotid specifično hibridizira sa ciljanim područjem egzona γ-sarkoglikanske RNK.
2. Antisens oligonukleotid prema patentnom zahtjevu 1, pri čemu oligonukleotid ne može formirati supstrat RNaze H.
3. Antisens oligonukleotid prema patentnom zahtjevu 1 ili patentnom zahtjevu 2, koji sadrži modificiranu oligonukleotidnu okosnicu koja sadrži modificirani ostatak kao supstituciju šećera.
4. Antisens oligonukleotid prema patentnom zahtjevu 3, gdje je modificirani ostatak Morfolino.
5. Antisens oligonukleotid prema patentnom zahtjevu 3 ili patentnom zahtjevu 4, gdje modificirana oligonukleotidna okosnica sadrži najmanje jednu modificiranu internukleotidnu vezu.
6. Antisens oligonukleotid prema patentnom zahtjevu 3, gdje je modificirani ostatak modifikacija tipa triciklo-DNK (tc-DNK).
7. Antisens oligonukleotid prema patentnom zahtjevu 5, gdje modificirana internukleotidna veza sadrži modificirani fosfat izabran iz grupe koja se sastoji od metil fosfonata, metil fosforotioata, fosforomorfolidata, fosforopiperazidata i fosforoamidata.
8. Antisens oligonukleotid prema bilo kojem od patentnih zahtjeva 3-7, pri čemu je oligonukleotid 2'-O-metil-oligoribonukleotid.
9. Antisens oligonukleotid prema bilo kojem od patentnih zahtjeva 1-8, pri čemu oligonukleotid sadrži peptidnu nukleinsku kiselinu.
10. Antisens oligonukleotid prema bilo kojem od patentnih zahtjeva 1-9, pri čemu je oligonukleotid kemijski povezan sa jednim ili sa više konjugata koji pojačavaju aktivnost, staničnu distribuciju ili stanično preuzimanje antisens oligonukleotida.
11. Antisens oligonukleotid prema patentnom zahtjevu 10, gdje je oligonukleotid kemijski povezan sa molekulom polietilen glikola.
12. Antisens oligonukleotid prema patentnom zahtjevu 10 ili patentnom zahtjevu 11, gdje je konjugat peptid koji pojačava stanično preuzimanje, pri čemu je peptid izabran iz grupe koja se sastoji od signala jedarne lokalizacije (NLS), HIV-1 TAT proteina, peptida koji sadrži područje vezivanja integrina, oligolizina, adenovirusnog vlaknastog proteina i peptida koji sadrži područje za endocitozu posredovanu receptorima (RME).
13. Farmaceutski sastav, koja sadrži antisens oligonukleotid prema bilo kojem od patentnih zahtjeva 1-12 i fiziološki kompatibilan pufer.
14. Antisens oligonukleotid prema bilo kojem od patentnih zahtjeva 1-12 ili farmaceutski sastav prema patentnom zahtjevu 13, za upotrebu u induciranju preskakanja egzona gama sarkoglikanske RNK u stanici, opcionalno pri čemu je stanica ljudska mišićna stanica, opcionalno pri čemu je ljudska mišićna stanica u pacijentu koji boluje od mišićne distrofije.
15. Antisens oligonukleotid ili farmaceutski sastav za upotrebu prema patentnom zahtjevu 14, pri čemu je mišićna distrofija mišićna distrofija donjih udova tipa 2C (LGMD2C).
16. Antisens oligonukleotid prema bilo kojem od patentnih zahtjeva 1-12 ili farmaceutski sastav prema patentnom zahtjevu 13, za upotrebu u ublažavanju, inhibiranju ili poboljšanju mišićne distrofije donjih udova tipa 2C (LGMD2C) kod pacijenta kome je to potrebno, pri čemu je pacijentu potrebno primjenjivati terapijski efektivnu količinu antisens oligonukleotida ili sastava, čime se ublažava LGMD2C, inhibira napredak distrofične patologije ili poboljšava mišićna funkcija.
17. Antisens oligonukleotid ili farmaceutski sastav prema patentnom zahtjevu 16, pri čemu poboljšanje obuhvaća poboljšanje funkcije srčanog mišića, poboljšanje snage respiratornih mišića, poboljšanje motorne stabilnosti ili poboljšanje snage gornjih udova.
18. Farmaceutski sastav koja sadrži fiziološki kompatibilan pufer i dva ili više antisens oligonukleotida sa sekvencom koja je kao što je to navedeno u SEQ ID NO: 4-9, 11, 12, 14-29 ili 31-34.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562144712P | 2015-04-08 | 2015-04-08 | |
| PCT/US2016/026477 WO2016164602A1 (en) | 2015-04-08 | 2016-04-07 | Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping |
| EP16777291.2A EP3283500B1 (en) | 2015-04-08 | 2016-04-07 | Compositions and methods for correcting limb girdle muscular dystrophy type 2c using exon skipping |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20210139T1 true HRP20210139T1 (hr) | 2021-03-19 |
Family
ID=57073329
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20210139TT HRP20210139T1 (hr) | 2015-04-08 | 2021-01-26 | Sastavi i postupci za korigiranje mišične distrofije donjih udova tipa 2c upotrebom preskakanja egzona |
Country Status (17)
| Country | Link |
|---|---|
| US (5) | US10273483B2 (hr) |
| EP (1) | EP3283500B1 (hr) |
| BR (1) | BR112017021485A2 (hr) |
| CA (1) | CA2981960C (hr) |
| CY (1) | CY1123599T1 (hr) |
| DK (1) | DK3283500T3 (hr) |
| ES (1) | ES2846902T3 (hr) |
| HK (1) | HK1250720A1 (hr) |
| HR (1) | HRP20210139T1 (hr) |
| HU (1) | HUE052604T2 (hr) |
| LT (1) | LT3283500T (hr) |
| PL (1) | PL3283500T3 (hr) |
| PT (1) | PT3283500T (hr) |
| RS (1) | RS61349B1 (hr) |
| SI (1) | SI3283500T1 (hr) |
| TN (1) | TN2017000427A1 (hr) |
| WO (1) | WO2016164602A1 (hr) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2892617T (pt) * | 2012-09-06 | 2018-10-18 | Univ Chicago | Polinucleótidos antisenso para induzir o salto de exão e métodos de tratamentos de distrofias |
| ES2846902T3 (es) * | 2015-04-08 | 2021-07-30 | Univ Chicago | Composiciones y procedimientos para corregir la distrofia muscular de cinturas tipo 2C mediante omisión de exones |
Family Cites Families (60)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
| FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| AU598946B2 (en) | 1987-06-24 | 1990-07-05 | Howard Florey Institute Of Experimental Physiology And Medicine | Nucleoside derivatives |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| US5585362A (en) | 1989-08-22 | 1996-12-17 | The Regents Of The University Of Michigan | Adenovirus vectors for gene therapy |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| WO1991018088A1 (en) | 1990-05-23 | 1991-11-28 | The United States Of America, Represented By The Secretary, United States Department Of Commerce | Adeno-associated virus (aav)-based eucaryotic vectors |
| BR9106702A (pt) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals Inc | Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo |
| US5328688A (en) | 1990-09-10 | 1994-07-12 | Arch Development Corporation | Recombinant herpes simplex viruses vaccines and methods |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| US5849571A (en) | 1990-10-10 | 1998-12-15 | University Of Pittsburgh Of The Commonwealth System Of Higher Education | Latency active herpes virus promoters and their use |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
| US7223833B1 (en) | 1991-05-24 | 2007-05-29 | Isis Pharmaceuticals, Inc. | Peptide nucleic acid conjugates |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
| US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| DE637965T1 (de) | 1991-11-26 | 1995-12-14 | Gilead Sciences Inc | Gesteigerte bildung von triple- und doppelhelices aus oligomeren mit modifizierten pyrimidinen. |
| TW393513B (en) | 1991-11-26 | 2000-06-11 | Isis Pharmaceuticals Inc | Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| US5879934A (en) | 1992-07-31 | 1999-03-09 | University Of Pittsburgh Of The Commonwealth System Of Higher Education | Herpes simplex virus strains for gene transfer |
| US20040005707A1 (en) | 2002-07-02 | 2004-01-08 | Isis Pharmaceuticals Inc. | Antisense modulation of integrin beta 5 expression |
| US5661033A (en) | 1992-11-25 | 1997-08-26 | The Board Of Trustees Of The Leland Stanford Junior University | Gene transfer using herpes virus vectors as a tool for neuroprotection |
| US5631237A (en) | 1992-12-22 | 1997-05-20 | Dzau; Victor J. | Method for producing in vivo delivery of therapeutic agents via liposomes |
| DE4311651A1 (de) | 1993-04-08 | 1994-10-13 | Boehringer Ingelheim Int | Virus für den Transport von Fremd-DNA in höhere eukaryotische Zellen |
| US5834441A (en) | 1993-09-13 | 1998-11-10 | Rhone-Poulenc Rorer Pharmaceuticals Inc. | Adeno-associated viral (AAV) liposomes and methods related thereto |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| DE69433592T2 (de) | 1993-11-09 | 2005-02-10 | Targeted Genetics Corp., Seattle | Die erzielung hoher titer des rekombinanten aav-vektors |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| FR2716682B1 (fr) | 1994-01-28 | 1996-04-26 | Centre Nat Rech Scient | Procédé de préparation de virus adéno-associés (AAV) recombinants et utilisations. |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| US5792453A (en) | 1995-02-28 | 1998-08-11 | The Regents Of The University Of California | Gene transfer-mediated angiogenesis therapy |
| US5707618A (en) | 1995-03-24 | 1998-01-13 | Genzyme Corporation | Adenovirus vectors for gene therapy |
| FR2732357B1 (fr) | 1995-03-31 | 1997-04-30 | Rhone Poulenc Rorer Sa | Vecteurs viraux et utilisation pour le traitement des desordres hyperproliferatifs, notamment de la restenose |
| US5773289A (en) | 1995-06-06 | 1998-06-30 | University Of Pittsburgh | AAV directed targeted integration |
| US5622856A (en) | 1995-08-03 | 1997-04-22 | Avigen | High efficiency helper system for AAV vector production |
| US5912340A (en) | 1995-10-04 | 1999-06-15 | Epoch Pharmaceuticals, Inc. | Selective binding complementary oligonucleotides |
| US5830727A (en) | 1995-11-18 | 1998-11-03 | Human Gene Therapy Research Institute | Herpes simplex virus amplicon mini-vector gene transfer system |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| CN1273476C (zh) | 1997-09-12 | 2006-09-06 | 埃克西康有限公司 | 寡核苷酸类似物 |
| EP1072679A3 (en) | 1999-07-20 | 2002-07-31 | Agilent Technologies, Inc. (a Delaware corporation) | Method of producing nucleic acid molecules with reduced secondary structure |
| US7339051B2 (en) | 2003-04-28 | 2008-03-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for the treatment of severe acute respiratory syndrome (SARS) |
| EP1752536A4 (en) | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME |
| US7807816B2 (en) | 2004-06-28 | 2010-10-05 | University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| WO2010115993A1 (en) | 2009-04-10 | 2010-10-14 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
| GB201117880D0 (en) | 2011-10-17 | 2011-11-30 | Ucl Business Plc | Antisense oligonucleotides |
| PT2892617T (pt) * | 2012-09-06 | 2018-10-18 | Univ Chicago | Polinucleótidos antisenso para induzir o salto de exão e métodos de tratamentos de distrofias |
| AU2013364158A1 (en) * | 2012-12-20 | 2015-07-09 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| ES2846902T3 (es) * | 2015-04-08 | 2021-07-30 | Univ Chicago | Composiciones y procedimientos para corregir la distrofia muscular de cinturas tipo 2C mediante omisión de exones |
-
2016
- 2016-04-07 ES ES16777291T patent/ES2846902T3/es active Active
- 2016-04-07 WO PCT/US2016/026477 patent/WO2016164602A1/en active Application Filing
- 2016-04-07 LT LTEP16777291.2T patent/LT3283500T/lt unknown
- 2016-04-07 CA CA2981960A patent/CA2981960C/en active Active
- 2016-04-07 PL PL16777291T patent/PL3283500T3/pl unknown
- 2016-04-07 EP EP16777291.2A patent/EP3283500B1/en active Active
- 2016-04-07 DK DK16777291.2T patent/DK3283500T3/da active
- 2016-04-07 US US15/564,681 patent/US10273483B2/en active Active
- 2016-04-07 BR BR112017021485A patent/BR112017021485A2/pt active Search and Examination
- 2016-04-07 PT PT167772912T patent/PT3283500T/pt unknown
- 2016-04-07 RS RS20210068A patent/RS61349B1/sr unknown
- 2016-04-07 TN TNP/2017/000427A patent/TN2017000427A1/en unknown
- 2016-04-07 SI SI201630981T patent/SI3283500T1/sl unknown
- 2016-04-07 HU HUE16777291A patent/HUE052604T2/hu unknown
-
2018
- 2018-08-07 HK HK18110160.3A patent/HK1250720A1/zh unknown
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2019
- 2019-04-26 US US16/395,741 patent/US10801029B2/en active Active
-
2020
- 2020-09-14 US US17/020,328 patent/US20210032631A1/en not_active Abandoned
- 2020-12-04 CY CY20201101145T patent/CY1123599T1/el unknown
-
2021
- 2021-01-26 HR HRP20210139TT patent/HRP20210139T1/hr unknown
-
2022
- 2022-01-07 US US17/571,302 patent/US20220119820A1/en not_active Abandoned
- 2022-08-11 US US17/819,291 patent/US20230167452A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| BR112017021485A2 (pt) | 2018-07-03 |
| US20210032631A1 (en) | 2021-02-04 |
| US10273483B2 (en) | 2019-04-30 |
| PL3283500T3 (pl) | 2021-05-31 |
| PT3283500T (pt) | 2021-01-28 |
| CA2981960A1 (en) | 2016-10-13 |
| EP3283500A1 (en) | 2018-02-21 |
| WO2016164602A1 (en) | 2016-10-13 |
| US20180080030A1 (en) | 2018-03-22 |
| EP3283500B1 (en) | 2020-11-11 |
| TN2017000427A1 (en) | 2019-04-12 |
| RS61349B1 (sr) | 2021-02-26 |
| US20190249180A1 (en) | 2019-08-15 |
| HK1250720A1 (zh) | 2019-01-11 |
| CY1123599T1 (el) | 2022-03-24 |
| ES2846902T3 (es) | 2021-07-30 |
| EP3283500A4 (en) | 2019-01-23 |
| HUE052604T2 (hu) | 2021-05-28 |
| US20230167452A1 (en) | 2023-06-01 |
| CA2981960C (en) | 2023-09-19 |
| DK3283500T3 (en) | 2020-11-16 |
| SI3283500T1 (sl) | 2020-12-31 |
| US20220119820A1 (en) | 2022-04-21 |
| US10801029B2 (en) | 2020-10-13 |
| LT3283500T (lt) | 2020-12-10 |
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