ECSP17074016A - Producción de vectores adeno-asociados de gran tamaño - Google Patents

Producción de vectores adeno-asociados de gran tamaño

Info

Publication number
ECSP17074016A
ECSP17074016A ECIEPI201774016A ECPI201774016A ECSP17074016A EC SP17074016 A ECSP17074016 A EC SP17074016A EC IEPI201774016 A ECIEPI201774016 A EC IEPI201774016A EC PI201774016 A ECPI201774016 A EC PI201774016A EC SP17074016 A ECSP17074016 A EC SP17074016A
Authority
EC
Ecuador
Prior art keywords
aav
production
adeno
associated vectors
genomes
Prior art date
Application number
ECIEPI201774016A
Other languages
English (en)
Spanish (es)
Inventor
David Souza
Karen Vincent
Sirkka Kyostio-Moore
Original Assignee
Genzyme Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=56943906&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ECSP17074016(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of ECSP17074016A publication Critical patent/ECSP17074016A/es

Links

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10041Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Pulmonology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
ECIEPI201774016A 2015-04-08 2017-11-08 Producción de vectores adeno-asociados de gran tamaño ECSP17074016A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562144862P 2015-04-08 2015-04-08
US201562220067P 2015-09-17 2015-09-17

Publications (1)

Publication Number Publication Date
ECSP17074016A true ECSP17074016A (es) 2018-02-28

Family

ID=56943906

Family Applications (1)

Application Number Title Priority Date Filing Date
ECIEPI201774016A ECSP17074016A (es) 2015-04-08 2017-11-08 Producción de vectores adeno-asociados de gran tamaño

Country Status (27)

Country Link
US (1) US10815497B2 (cg-RX-API-DMAC7.html)
EP (2) EP4512429A3 (cg-RX-API-DMAC7.html)
JP (3) JP6878299B2 (cg-RX-API-DMAC7.html)
KR (2) KR102848324B1 (cg-RX-API-DMAC7.html)
CN (1) CN107864657A (cg-RX-API-DMAC7.html)
AU (3) AU2016245806A1 (cg-RX-API-DMAC7.html)
BR (1) BR112017021505A2 (cg-RX-API-DMAC7.html)
CA (1) CA2982123A1 (cg-RX-API-DMAC7.html)
CL (1) CL2017002537A1 (cg-RX-API-DMAC7.html)
CO (1) CO2017011344A2 (cg-RX-API-DMAC7.html)
CR (1) CR20170505A (cg-RX-API-DMAC7.html)
DK (1) DK3280799T3 (cg-RX-API-DMAC7.html)
EA (1) EA201792236A1 (cg-RX-API-DMAC7.html)
EC (1) ECSP17074016A (cg-RX-API-DMAC7.html)
ES (1) ES3008382T3 (cg-RX-API-DMAC7.html)
GT (1) GT201700214A (cg-RX-API-DMAC7.html)
HU (1) HUE069779T2 (cg-RX-API-DMAC7.html)
IL (3) IL294965A (cg-RX-API-DMAC7.html)
MX (1) MX2017012935A (cg-RX-API-DMAC7.html)
PE (1) PE20171800A1 (cg-RX-API-DMAC7.html)
PH (1) PH12017501836A1 (cg-RX-API-DMAC7.html)
PL (1) PL3280799T3 (cg-RX-API-DMAC7.html)
SG (3) SG11201708203UA (cg-RX-API-DMAC7.html)
TN (1) TN2017000431A1 (cg-RX-API-DMAC7.html)
TW (1) TWI707951B (cg-RX-API-DMAC7.html)
UY (1) UY36611A (cg-RX-API-DMAC7.html)
WO (1) WO2016164609A2 (cg-RX-API-DMAC7.html)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2956477T5 (pl) 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Zoptymalizowany gen czynnika viii
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3352787A1 (en) 2015-09-24 2018-08-01 BioMarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
RS63548B1 (sr) 2016-02-01 2022-09-30 Bioverativ Therapeutics Inc Optimizovani geni faktora viii
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
IL302748B1 (en) 2016-05-18 2025-09-01 Voyager Therapeutics Inc Modulatory polynucleotides
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
WO2018154096A1 (en) 2017-02-24 2018-08-30 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
CN118956965A (zh) * 2017-05-19 2024-11-15 编码治疗公司 高活性调控元件
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
CN111448308A (zh) 2017-08-03 2020-07-24 沃雅戈治疗公司 递送aav的组合物和方法
KR20250040746A (ko) 2017-08-09 2025-03-24 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
WO2019067840A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
AU2018375192B2 (en) 2017-12-01 2023-11-09 Encoded Therapeutics, Inc. Engineered DNA binding proteins
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US20210338838A1 (en) * 2018-07-20 2021-11-04 University Of Utah Research Foundation Gene therapy for macular degeneration
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
JP2022504740A (ja) * 2018-10-12 2022-01-13 ジェンザイム・コーポレーション 肝臓に向けられた遺伝子補充療法によって重度のpkuを処置するための改善されたヒトpahの生成
CN119061198A (zh) * 2019-08-29 2024-12-03 美国西门子医学诊断股份有限公司 用于检测aav脱落的试剂和方法
US12403164B2 (en) 2019-09-30 2025-09-02 Bioverativ Therapeutics Inc. Lentiviral vector formulations
CN111218446B (zh) * 2019-12-25 2023-03-28 劲帆生物医药科技(武汉)有限公司 一种肝脏特异性启动子及其应用
CN115552037A (zh) * 2020-03-16 2022-12-30 北卡罗来纳大学教堂山分校 用于选择性检测肿瘤源性病毒dna的组合物和方法
BR112022026127A2 (pt) 2020-06-24 2023-01-17 Bioverativ Therapeutics Inc Métodos para remoção de fator viii livre de preparações de vetores lentivirais modificados para expressar referida proteína
CN116323953A (zh) 2020-07-30 2023-06-23 沙普治疗股份有限公司 诱导产生raav病毒粒子的稳定细胞系
AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
JP2024534123A (ja) 2021-08-23 2024-09-18 バイオベラティブ セラピューティクス インコーポレイテッド バキュロウイルス発現システム
TW202323274A (zh) 2021-08-23 2023-06-16 美商百歐維拉提夫治療公司 優化因子viii基因
WO2023028455A1 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
AU2022355088A1 (en) 2021-09-30 2024-05-16 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
WO2023069926A1 (en) 2021-10-18 2023-04-27 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
WO2024224361A1 (en) * 2023-04-26 2024-10-31 Sanofi Host cell lines and methods for identifying and using such host cell lines

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2001500015A (ja) 1996-09-06 2001-01-09 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
CA2387484A1 (en) * 1999-10-12 2001-04-19 Haim Burstein Adeno-associated virus vectors encoding factor viii and methods of using the same
CA2410828C (en) 2000-06-01 2012-01-24 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
MX346493B (es) 2001-11-13 2017-03-21 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
US20040092008A1 (en) * 2002-11-12 2004-05-13 Snyder Richard O. Recombinant AAV vectors for gene therapy of hemophilia A
WO2004075861A2 (en) 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
ES2521682T3 (es) 2003-05-21 2014-11-13 Genzyme Corporation Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
SG10201403290SA (en) 2009-06-16 2014-10-30 Genzyme Corp Improved methods for purification of recombinant aav vectors
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
WO2014064277A1 (en) * 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
RS61039B1 (sr) * 2013-09-12 2020-12-31 Biomarin Pharm Inc Aav vektori koji sadrže gen koji kodira faktor viii
IL248102B (en) 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
KR102863734B1 (ko) 2015-02-10 2025-09-25 젠자임 코포레이션 선조체 및 피질로의 바이러스 입자의 향상된 전달
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造

Also Published As

Publication number Publication date
US10815497B2 (en) 2020-10-27
TWI707951B (zh) 2020-10-21
KR20170133500A (ko) 2017-12-05
MX2017012935A (es) 2018-02-12
EP4512429A3 (en) 2025-05-21
NZ774736A (en) 2024-08-30
IL254933B (en) 2021-06-30
SG11201708203UA (en) 2017-11-29
JP2021118724A (ja) 2021-08-12
AU2022203942B2 (en) 2023-12-14
PE20171800A1 (es) 2017-12-28
CL2017002537A1 (es) 2018-05-11
EA201792236A1 (ru) 2018-03-30
UY36611A (es) 2016-11-30
EP4512429A2 (en) 2025-02-26
TW201710498A (zh) 2017-03-16
IL254933A0 (en) 2017-12-31
SG10201912912QA (en) 2020-02-27
JP6878299B2 (ja) 2021-05-26
BR112017021505A2 (pt) 2018-07-03
AU2016245806A1 (en) 2017-11-30
IL283291A (en) 2021-07-29
AU2024201619A1 (en) 2024-03-28
CR20170505A (es) 2018-01-26
DK3280799T3 (da) 2025-01-27
CN107864657A (zh) 2018-03-30
PL3280799T3 (pl) 2025-04-14
WO2016164609A2 (en) 2016-10-13
IL294965A (en) 2022-09-01
PH12017501836A1 (en) 2018-04-23
GT201700214A (es) 2018-10-24
JP2018510648A (ja) 2018-04-19
IL283291B (en) 2022-08-01
KR102848324B1 (ko) 2025-08-20
CO2017011344A2 (es) 2018-03-28
ES3008382T3 (en) 2025-03-24
EP3280799A2 (en) 2018-02-14
KR20250126875A (ko) 2025-08-25
AU2022203942A1 (en) 2022-06-23
WO2016164609A3 (en) 2016-12-15
EP3280799B1 (en) 2024-10-30
HUE069779T2 (hu) 2025-04-28
NZ737009A (en) 2024-01-26
SG10201909381WA (en) 2019-11-28
CA2982123A1 (en) 2016-10-13
TN2017000431A1 (en) 2019-04-12
JP2023116678A (ja) 2023-08-22
US20190048362A1 (en) 2019-02-14
HK1246339A1 (en) 2018-09-07

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