HRP20171165T1 - Genska terapija za lizosomske bolesti nakupljanja - Google Patents
Genska terapija za lizosomske bolesti nakupljanja Download PDFInfo
- Publication number
- HRP20171165T1 HRP20171165T1 HRP20171165TT HRP20171165T HRP20171165T1 HR P20171165 T1 HRP20171165 T1 HR P20171165T1 HR P20171165T T HRP20171165T T HR P20171165TT HR P20171165 T HRP20171165 T HR P20171165T HR P20171165 T1 HRP20171165 T1 HR P20171165T1
- Authority
- HR
- Croatia
- Prior art keywords
- aav vector
- recombinant aav
- use according
- vector
- brain
- Prior art date
Links
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 239000013607 AAV vector Substances 0.000 claims 9
- 238000000034 method Methods 0.000 claims 5
- 210000004556 brain Anatomy 0.000 claims 4
- 239000013598 vector Substances 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 208000035343 Infantile neurovisceral acid sphingomyelinase deficiency Diseases 0.000 claims 2
- 201000000794 Niemann-Pick disease type A Diseases 0.000 claims 2
- 108700019146 Transgenes Proteins 0.000 claims 2
- 208000007824 Type A Niemann-Pick Disease Diseases 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 208000035374 Chronic visceral acid sphingomyelinase deficiency Diseases 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 201000000791 Niemann-Pick disease type B Diseases 0.000 claims 1
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 1
- 208000008291 Type B Niemann-Pick Disease Diseases 0.000 claims 1
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 210000004055 fourth ventricle Anatomy 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 210000003140 lateral ventricle Anatomy 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 210000001835 viscera 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
- A61K48/0075—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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
-
- 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
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Claims (6)
1. Rekombinantni AAV vektor koji sadrži transgen kodira kiselinu sfingomijelinazu za upotrebu u postupku liječenja Niemann–Pickove bolesti tip A ili Niemann–Pickove bolesti tip B kod subjekta, naznačen time da navedeni rekombinantni AAV vektor je AAV5 vektor i pri čemu postupak sadrži davanje vektora u barem jednu ventrikulu mozga od subjekta, pri čemu se navedeni transgen eksprimira i eksprimirani proizvod proteina se dovodi u središnji živčani sustav, te proizvoljno, u visceralne organe.
2. Rekombinantni AAV vektor za uporabu prema zahtjevu 1, naznačen time da navedeni rekombinantni AAV vektor je AAV vektor koji sadrži AAV5 ovojnicu i AAV2 genom.
3. Rekombinantni AAV vektor za uporabu prema zahtjevu 1 ili zahtjevu 2, naznačen time da postupak sadrži davanje vektora izravnim ubrizgavanjem u: (i) ventrikulu mozga; (ii) lateralnu ventrikulu mozga; ili (iii) četvrtu ventrikulu mozga.
4. Rekombinantni AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 3, naznačen time da subjekt je sisavac.
5. Rekombinantni AAV vektor za uporabu prema zahtjevu 4, naznačen time da subjekt je ljudski pacijent.
6. Rekombinantni AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 5, naznačen time da navedeni postupak je postupak liječenja Niemann–Pickove bolesti tip A.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US92494707P | 2007-06-06 | 2007-06-06 | |
PCT/US2008/065481 WO2008154198A1 (en) | 2007-06-06 | 2008-06-02 | Gene therapy for lysosomal storage diseases |
EP08756596.6A EP2158322B1 (en) | 2007-06-06 | 2008-06-02 | Gene therapy for lysosomal storage diseases |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20171165T1 true HRP20171165T1 (hr) | 2017-12-15 |
Family
ID=40130114
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20171165TT HRP20171165T1 (hr) | 2007-06-06 | 2017-07-31 | Genska terapija za lizosomske bolesti nakupljanja |
Country Status (14)
Country | Link |
---|---|
US (2) | US8796236B2 (hr) |
EP (2) | EP3252161B1 (hr) |
AR (1) | AR070000A1 (hr) |
CY (1) | CY1119295T1 (hr) |
DK (1) | DK2158322T3 (hr) |
ES (2) | ES2635726T3 (hr) |
HR (1) | HRP20171165T1 (hr) |
HU (1) | HUE035779T2 (hr) |
IL (3) | IL202522A (hr) |
LT (1) | LT2158322T (hr) |
PL (2) | PL3252161T3 (hr) |
PT (2) | PT2158322T (hr) |
SI (1) | SI2158322T1 (hr) |
WO (1) | WO2008154198A1 (hr) |
Families Citing this family (32)
Publication number | Priority date | Publication date | Assignee | Title |
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AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
DK1988823T3 (en) | 2006-02-09 | 2018-12-03 | Genzyme Corp | SLOW INTRAVENTRICULAR ADMINISTRATION |
SI2158322T1 (sl) | 2007-06-06 | 2017-10-30 | Genzyme Corporation | Genska terapija za bolezni lizosomskega shranjevanja |
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US8242086B2 (en) * | 2008-11-12 | 2012-08-14 | Duke University | Methods and compositions for treating disorders caused by a deficiency in a gene product of a CLN gene |
KR101793615B1 (ko) | 2009-04-30 | 2017-11-03 | 오스페달레 산 라파엘 에스.알.엘. | 유전자 벡터 |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
US9546369B2 (en) | 2010-04-23 | 2017-01-17 | University Of Massachusetts | Multicistronic expression constructs |
US9849195B2 (en) | 2011-03-31 | 2017-12-26 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
US20130090374A1 (en) * | 2011-04-20 | 2013-04-11 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis |
US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
DK2751279T3 (da) | 2011-08-31 | 2017-11-20 | St Jude Children's Res Hospital | Fremgangsmåder og sammensætninger til påvisning af niveauet af lysosomal exocytose-aktivitet og fremgangsmåder til anvendelse |
KR20220119187A (ko) * | 2013-05-15 | 2022-08-26 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
WO2015031686A1 (en) | 2013-08-30 | 2015-03-05 | Amgen Inc. | High titer recombinant aav vector production in adherent and suspension cells |
RU2020140209A (ru) | 2014-10-21 | 2021-01-25 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
CA2966620A1 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
WO2016172155A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
CN108603199A (zh) * | 2015-12-15 | 2018-09-28 | 建新公司 | 用于治疗粘脂贮积症ii型的腺伴随病毒载体 |
DK3405215T3 (da) * | 2016-01-19 | 2022-09-19 | Univ California | Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser |
IL305449A (en) | 2016-04-15 | 2023-10-01 | Univ Pennsylvania | Gene therapy for the treatment of type II mucositis |
AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
EP3528852A4 (en) | 2016-10-20 | 2020-06-03 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR THE TREATMENT OF FABRY'S DISEASE |
WO2018085688A1 (en) * | 2016-11-04 | 2018-05-11 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
US20210228739A1 (en) * | 2018-05-15 | 2021-07-29 | University Of Massachusetts | Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a |
CA3106010A1 (en) | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
EP3952924A4 (en) * | 2019-04-12 | 2023-05-24 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS |
KR20220112283A (ko) | 2019-12-10 | 2022-08-10 | 다케다 야쿠힌 고교 가부시키가이샤 | Hunter 질환 치료용 아데노-연합된 바이러스 벡터 |
EP4135776A1 (en) * | 2020-04-14 | 2023-02-22 | Genethon | Vectors for the treatment of acid ceramidase deficiency |
Family Cites Families (26)
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US5672344A (en) | 1987-12-30 | 1997-09-30 | The Regents Of The University Of Michigan | Viral-mediated gene transfer system |
US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
EP0705344B8 (en) | 1993-06-24 | 2006-05-10 | Advec Inc. | Adenovirus vectors for gene therapy |
DE69434594T2 (de) | 1993-10-25 | 2006-09-21 | Canji, Inc., San Diego | Rekombinante adenoviren-vektor und verfahren zur verwendung |
CA2176117C (en) | 1993-11-09 | 2006-01-03 | Terence R. Flotte | Generation of high titers of recombinant aav vectors |
US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
EP0755454B1 (en) | 1994-04-13 | 2008-02-13 | The Rockefeller University | Aav-mediated delivery of dna to cells of the nervous system |
US6274309B1 (en) | 1996-07-26 | 2001-08-14 | Richard Kolesnick | Method for the modulation of acid-sphingomyelinase-related apoptosis |
US20040076613A1 (en) * | 2000-11-03 | 2004-04-22 | Nicholas Mazarakis | Vector system |
US20050026832A1 (en) * | 1997-11-25 | 2005-02-03 | Genentech, Inc. | Fibroblast growth factor-19 (FGF-19) nucleic acids and polypeptides and methods of use for the treatment of obesity and related disorders |
US6544785B1 (en) | 1998-09-14 | 2003-04-08 | Mount Sinai School Of Medicine Of New York University | Helper-free rescue of recombinant negative strand RNA viruses |
US6730297B1 (en) * | 1999-05-28 | 2004-05-04 | Chiron Corporation | Use of recombinant gene delivery vectors for treating or preventing lysosomal storage disorders |
WO2001036603A2 (en) * | 1999-11-17 | 2001-05-25 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
AU2001236713A1 (en) * | 2000-02-17 | 2001-08-27 | Genzyme Corporation | Methods for treatment of lysosomal storage diseases |
US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
US6667174B2 (en) | 2000-09-18 | 2003-12-23 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
CN101027388A (zh) | 2003-06-12 | 2007-08-29 | 建新公司 | 具有增强活性的修饰的人酸性神经磷脂酶及其制备方法 |
US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
US20060171926A1 (en) * | 2004-04-30 | 2006-08-03 | Passini Marco A | Gene therapy for neurometabolic disorders |
CN107007842A (zh) * | 2005-05-02 | 2017-08-04 | 建新公司 | 神经代谢疾病的基因治疗 |
AR059371A1 (es) * | 2006-02-08 | 2008-03-26 | Genzyme Corp | Terapia genica para la enfermedad de niemann-pick tipo a |
DK1988823T3 (en) * | 2006-02-09 | 2018-12-03 | Genzyme Corp | SLOW INTRAVENTRICULAR ADMINISTRATION |
SI2158322T1 (sl) | 2007-06-06 | 2017-10-30 | Genzyme Corporation | Genska terapija za bolezni lizosomskega shranjevanja |
PL3482767T3 (pl) | 2009-08-28 | 2022-02-14 | Icahn School Of Medicine At Mount Sinai | Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy |
-
2008
- 2008-06-02 SI SI200831847T patent/SI2158322T1/sl unknown
- 2008-06-02 EP EP17168189.3A patent/EP3252161B1/en active Active
- 2008-06-02 PL PL17168189T patent/PL3252161T3/pl unknown
- 2008-06-02 PL PL08756596T patent/PL2158322T3/pl unknown
- 2008-06-02 LT LTEP08756596.6T patent/LT2158322T/lt unknown
- 2008-06-02 ES ES08756596.6T patent/ES2635726T3/es active Active
- 2008-06-02 DK DK08756596.6T patent/DK2158322T3/en active
- 2008-06-02 EP EP08756596.6A patent/EP2158322B1/en active Active
- 2008-06-02 HU HUE08756596A patent/HUE035779T2/en unknown
- 2008-06-02 PT PT87565966T patent/PT2158322T/pt unknown
- 2008-06-02 PT PT171681893T patent/PT3252161T/pt unknown
- 2008-06-02 ES ES17168189T patent/ES2905616T3/es active Active
- 2008-06-02 WO PCT/US2008/065481 patent/WO2008154198A1/en active Application Filing
- 2008-06-06 AR ARP080102421A patent/AR070000A1/es unknown
-
2009
- 2009-12-04 US US12/631,336 patent/US8796236B2/en active Active
- 2009-12-06 IL IL202522A patent/IL202522A/en active IP Right Grant
-
2014
- 2014-07-10 US US14/328,621 patent/US11369693B2/en active Active
-
2017
- 2017-06-14 IL IL252898A patent/IL252898B/en active IP Right Grant
- 2017-07-31 HR HRP20171165TT patent/HRP20171165T1/hr unknown
- 2017-08-03 CY CY20171100836T patent/CY1119295T1/el unknown
-
2019
- 2019-05-19 IL IL266734A patent/IL266734B/en unknown
Also Published As
Publication number | Publication date |
---|---|
EP2158322A1 (en) | 2010-03-03 |
US20100173979A1 (en) | 2010-07-08 |
EP2158322A4 (en) | 2011-06-01 |
SI2158322T1 (sl) | 2017-10-30 |
US11369693B2 (en) | 2022-06-28 |
IL252898A0 (en) | 2017-08-31 |
CY1119295T1 (el) | 2018-02-14 |
PT2158322T (pt) | 2017-08-09 |
IL252898B (en) | 2019-06-30 |
EP3252161B1 (en) | 2021-11-17 |
ES2635726T3 (es) | 2017-10-04 |
PL3252161T3 (pl) | 2022-03-07 |
IL266734B (en) | 2021-10-31 |
EP3252161A1 (en) | 2017-12-06 |
US8796236B2 (en) | 2014-08-05 |
LT2158322T (lt) | 2017-08-25 |
IL202522A0 (en) | 2011-08-01 |
PL2158322T3 (pl) | 2017-10-31 |
IL202522A (en) | 2017-10-31 |
PT3252161T (pt) | 2022-02-01 |
HUE035779T2 (en) | 2018-05-28 |
WO2008154198A1 (en) | 2008-12-18 |
US20150151007A1 (en) | 2015-06-04 |
AR070000A1 (es) | 2010-03-10 |
DK2158322T3 (en) | 2017-08-28 |
IL266734A (en) | 2019-07-31 |
ES2905616T3 (es) | 2022-04-11 |
EP2158322B1 (en) | 2017-05-03 |
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