CA3027737A1 - Treatment of eye disorders using aav2 variant with aflibercept - Google Patents

Treatment of eye disorders using aav2 variant with aflibercept Download PDF

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Publication number
CA3027737A1
CA3027737A1 CA3027737A CA3027737A CA3027737A1 CA 3027737 A1 CA3027737 A1 CA 3027737A1 CA 3027737 A CA3027737 A CA 3027737A CA 3027737 A CA3027737 A CA 3027737A CA 3027737 A1 CA3027737 A1 CA 3027737A1
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CA
Canada
Prior art keywords
aflibercept
fold
pharmaceutical composition
acid sequence
suspension
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3027737A
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English (en)
French (fr)
Inventor
Mark Blumenkranz
Mehdi Gasmi
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Adverum Biotechnologies Inc
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Adverum Biotechnologies Inc
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Filing date
Publication date
Application filed by Adverum Biotechnologies Inc filed Critical Adverum Biotechnologies Inc
Publication of CA3027737A1 publication Critical patent/CA3027737A1/en
Pending legal-status Critical Current

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    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/179—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • 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
    • 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
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/178—Syringes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Dispersion Chemistry (AREA)
  • Hematology (AREA)
CA3027737A 2016-06-16 2017-06-16 Treatment of eye disorders using aav2 variant with aflibercept Pending CA3027737A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662351234P 2016-06-16 2016-06-16
US62/351,234 2016-06-16
PCT/US2017/038003 WO2017218974A2 (en) 2016-06-16 2017-06-16 Treatment of amd using aav2 variant with aflibercept

Publications (1)

Publication Number Publication Date
CA3027737A1 true CA3027737A1 (en) 2017-12-21

Family

ID=60663653

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3027737A Pending CA3027737A1 (en) 2016-06-16 2017-06-16 Treatment of eye disorders using aav2 variant with aflibercept

Country Status (25)

Country Link
US (2) US20190151409A1 (cg-RX-API-DMAC7.html)
EP (3) EP4671369A2 (cg-RX-API-DMAC7.html)
JP (5) JP6814822B2 (cg-RX-API-DMAC7.html)
KR (2) KR20210021113A (cg-RX-API-DMAC7.html)
CN (2) CN109641065A (cg-RX-API-DMAC7.html)
AU (3) AU2017286673B2 (cg-RX-API-DMAC7.html)
BR (1) BR112018076124A2 (cg-RX-API-DMAC7.html)
CA (1) CA3027737A1 (cg-RX-API-DMAC7.html)
CY (1) CY1123697T1 (cg-RX-API-DMAC7.html)
DK (2) DK3471780T3 (cg-RX-API-DMAC7.html)
ES (2) ES2840059T3 (cg-RX-API-DMAC7.html)
FI (1) FI3795181T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20201842T1 (cg-RX-API-DMAC7.html)
HU (1) HUE051953T2 (cg-RX-API-DMAC7.html)
IL (3) IL279919B2 (cg-RX-API-DMAC7.html)
LT (2) LT3471780T (cg-RX-API-DMAC7.html)
MX (2) MX391779B (cg-RX-API-DMAC7.html)
PL (1) PL3795181T3 (cg-RX-API-DMAC7.html)
PT (2) PT3471780T (cg-RX-API-DMAC7.html)
RS (2) RS67328B1 (cg-RX-API-DMAC7.html)
SG (1) SG11201811232XA (cg-RX-API-DMAC7.html)
SI (2) SI3471780T1 (cg-RX-API-DMAC7.html)
SM (2) SMT202100010T1 (cg-RX-API-DMAC7.html)
WO (1) WO2017218974A2 (cg-RX-API-DMAC7.html)
ZA (1) ZA201808538B (cg-RX-API-DMAC7.html)

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MY187898A (en) 2015-03-02 2021-10-27 Adverum Biotechnologies Inc Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
RS67328B1 (sr) * 2016-06-16 2025-11-28 Adverum Biotechnologies Inc Lečenje amd-a korišćenjem aav2 varijante sa afliberceptom
EP3528785A4 (en) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
US11773406B2 (en) 2017-03-17 2023-10-03 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
CN107661492A (zh) * 2017-08-31 2018-02-06 中山大学中山眼科中心 Vegf‑b的新用途
CN107537026A (zh) * 2017-08-31 2018-01-05 中山大学中山眼科中心 Vegf‑b的应用
CA3079565A1 (en) * 2017-10-18 2019-04-25 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified vegf-trap
UA127831C2 (uk) * 2017-11-27 2024-01-17 4Д Молекьюлар Терапьютикс Інк. Капсид аденоасоційованого вірусу та його застосування для інгібування ангіогенезу
MX2020006435A (es) 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
WO2019198084A1 (en) * 2018-04-12 2019-10-17 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Anti-aging compositions and methods of use
CN116585466A (zh) 2018-05-10 2023-08-15 瑞泽恩制药公司 含有高浓度vegf受体融合蛋白的制剂
US20200297869A1 (en) * 2019-03-04 2020-09-24 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
CA3137284A1 (en) * 2019-04-24 2020-10-29 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
JP2023509263A (ja) * 2019-09-11 2023-03-08 アドヴェラム バイオテクノロジーズ， インコーポレイテッド アフリベルセプトをコードするａａｖ２バリアントを使用する眼内血管新生疾患を処置する方法
AU2020345915A1 (en) * 2019-09-11 2022-03-24 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using AAV2 variants encoding aflibercept
TWI819268B (zh) 2020-01-16 2023-10-21 詹姆斯 Ｗ 希爾 藉由基因轉導改變眼睛顏色
FR3111913A1 (fr) * 2020-06-30 2021-12-31 Eyevensys Construction d’adn pour le traitement de pathologies oculaires
CN113952474A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
CN113952473A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
JP2023540464A (ja) * 2020-07-21 2023-09-25 インスピラール リミテッド 眼疾患の処置のための組成物および方法
CN113817775B (zh) * 2020-08-25 2023-02-17 南京吉迈生物技术有限公司 修饰的阿柏西普、组合物、方法及其在基因治疗中的应用
MX2023002695A (es) * 2020-09-03 2023-05-19 Univ Massachusetts Virus adeno-asociado para la administración de kh902 (conbercept) y usos del mismo.
CA3197936A1 (en) 2020-12-01 2022-06-09 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
JP2021049374A (ja) * 2020-12-09 2021-04-01 株式会社三洋物産 遊技機
US20240197915A1 (en) * 2021-04-27 2024-06-20 Adverum Biotechnologies, Inc. Methods of treating ocular diseases using aav2 variants encoding aflibercept
TW202342526A (zh) 2022-02-21 2023-11-01 大陸商上海瑞宏迪醫藥有限公司 Ｖｅｇｆ結合分子及其醫藥用途
WO2025072688A2 (en) * 2023-09-27 2025-04-03 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal cells, for nucleic acid delivery across retinal regions, or for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells
WO2025073297A1 (zh) * 2023-10-06 2025-04-10 甘李药业股份有限公司 表达抗vegf融合蛋白的aav病毒载体及其用途

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ES2638342T3 (es) * 2011-04-22 2017-10-19 The Regents Of The University Of California Viriones de virus adenoasociado con cápside variante y métodos para su uso
PT2846836T (pt) * 2012-05-07 2019-10-29 Allergan Inc Método de tratamento de dmi em doentes refratários à terapia anti-vegf
TWI775096B (zh) * 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
US20150111275A1 (en) * 2012-06-11 2015-04-23 Daniel V. Palanker Optical regulation of gene expression in the retina
KR20230152151A (ko) * 2013-07-12 2023-11-02 이베릭 바이오, 인크. 안과적 질환을 치료하거나 예방하기 위한 방법
CA2926853C (en) * 2013-10-18 2022-04-26 Regeneron Pharmaceuticals, Inc. Methods and compositions comprising a combination of a vegf antagonist and an anti-ctla-4 antibody
MY187898A (en) * 2015-03-02 2021-10-27 Adverum Biotechnologies Inc Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
RS67328B1 (sr) * 2016-06-16 2025-11-28 Adverum Biotechnologies Inc Lečenje amd-a korišćenjem aav2 varijante sa afliberceptom

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KR102218265B1 (ko) 2021-02-25
SMT202100010T1 (it) 2021-03-15
EP3795181B1 (en) 2025-08-06
HUE051953T2 (hu) 2021-04-28
CN117531025A (zh) 2024-02-09
RS61311B1 (sr) 2021-02-26
PT3471780T (pt) 2020-12-17
SI3795181T1 (sl) 2025-11-28
IL263686B (en) 2021-02-28
ES2840059T3 (es) 2021-07-06
HRP20201842T1 (hr) 2021-04-16
IL314568B2 (en) 2025-09-01
EP3471780A4 (en) 2019-04-24
AU2021225178A1 (en) 2021-09-30
PL3795181T3 (pl) 2025-12-22
ES3049818T3 (en) 2025-12-18
CN109641065A (zh) 2019-04-16
IL279919B2 (en) 2025-01-01
AU2025202930A1 (en) 2025-05-15
WO2017218974A3 (en) 2018-01-25
WO2017218974A2 (en) 2017-12-21
RS67328B1 (sr) 2025-11-28
DK3795181T3 (da) 2025-10-13
CY1123697T1 (el) 2022-03-24
EP4671369A2 (en) 2025-12-31
JP2022137244A (ja) 2022-09-21
BR112018076124A2 (pt) 2019-03-26
IL279919B1 (en) 2024-09-01
IL314568A (en) 2024-09-01
JP2024113193A (ja) 2024-08-21
SMT202500400T1 (it) 2025-11-10
LT3795181T (lt) 2025-12-29
AU2017286673B2 (en) 2021-06-24
LT3471780T (lt) 2021-01-25
US20220265740A1 (en) 2022-08-25
AU2017286673A1 (en) 2019-01-31
JP2020203941A (ja) 2020-12-24
JP6814822B2 (ja) 2021-01-20
KR20190037228A (ko) 2019-04-05
SG11201811232XA (en) 2019-01-30
AU2021225178B2 (en) 2025-01-30
FI3795181T3 (fi) 2025-10-14
MX2018015512A (es) 2019-04-24
JP2019521990A (ja) 2019-08-08
MX391779B (es) 2025-03-21
KR20210021113A (ko) 2021-02-24
IL263686A (en) 2019-01-31
IL314568B1 (en) 2025-05-01
ZA201808538B (en) 2021-04-28
PT3795181T (pt) 2025-11-05
EP3471780A2 (en) 2019-04-24
MX2022004786A (es) 2022-05-16
JP2025186377A (ja) 2025-12-23
SI3471780T1 (sl) 2021-03-31
DK3471780T3 (da) 2020-11-23
EP3795181A1 (en) 2021-03-24
IL279919A (en) 2021-03-01
US20190151409A1 (en) 2019-05-23
EP3471780B1 (en) 2020-10-28

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