KR102848324B1 - 거대 아데노-연관 벡터의 생산 - Google Patents

거대 아데노-연관 벡터의 생산

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Publication number
KR102848324B1
KR102848324B1 KR1020177032062A KR20177032062A KR102848324B1 KR 102848324 B1 KR102848324 B1 KR 102848324B1 KR 1020177032062 A KR1020177032062 A KR 1020177032062A KR 20177032062 A KR20177032062 A KR 20177032062A KR 102848324 B1 KR102848324 B1 KR 102848324B1
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aav
raav
genome
aav2
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KR20170133500A (ko
Inventor
시르카 쿄스티오-무어
데이빗 소우자
카렌 빈센트
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젠자임 코포레이션
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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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • 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)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Pulmonology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020177032062A 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산 Active KR102848324B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020257027291A KR20250126875A (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562144862P 2015-04-08 2015-04-08
US62/144,862 2015-04-08
US201562220067P 2015-09-17 2015-09-17
US62/220,067 2015-09-17
PCT/US2016/026486 WO2016164609A2 (en) 2015-04-08 2016-04-07 Production of oversized adeno-associated vectors

Related Child Applications (1)

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KR1020257027291A Division KR20250126875A (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산

Publications (2)

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KR20170133500A KR20170133500A (ko) 2017-12-05
KR102848324B1 true KR102848324B1 (ko) 2025-08-20

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KR1020177032062A Active KR102848324B1 (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산
KR1020257027291A Pending KR20250126875A (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산

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Country Status (27)

Country Link
US (1) US10815497B2 (enExample)
EP (2) EP4512429A3 (enExample)
JP (3) JP6878299B2 (enExample)
KR (2) KR102848324B1 (enExample)
CN (1) CN107864657A (enExample)
AU (3) AU2016245806A1 (enExample)
BR (1) BR112017021505A2 (enExample)
CA (1) CA2982123A1 (enExample)
CL (1) CL2017002537A1 (enExample)
CO (1) CO2017011344A2 (enExample)
CR (1) CR20170505A (enExample)
DK (1) DK3280799T3 (enExample)
EA (1) EA201792236A1 (enExample)
EC (1) ECSP17074016A (enExample)
ES (1) ES3008382T3 (enExample)
GT (1) GT201700214A (enExample)
HU (1) HUE069779T2 (enExample)
IL (3) IL283291B (enExample)
MX (1) MX2017012935A (enExample)
PE (1) PE20171800A1 (enExample)
PH (1) PH12017501836A1 (enExample)
PL (1) PL3280799T3 (enExample)
SG (3) SG10201909381WA (enExample)
TN (1) TN2017000431A1 (enExample)
TW (1) TWI707951B (enExample)
UY (1) UY36611A (enExample)
WO (1) WO2016164609A2 (enExample)

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
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
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
MX2018003702A (es) 2015-09-24 2018-04-30 Biomarin Pharm Inc Vectores del factor viii del virus adenoasociado, particulas viricas asociadas y formulaciones terapeuticas que las comprenden.
PT3411478T (pt) 2016-02-01 2022-09-13 Bioverativ Therapeutics Inc Genes do fator viii otimizados
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
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
EP3655533A1 (en) 2017-02-24 2020-05-27 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
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP3624858A4 (en) * 2017-05-19 2021-06-23 Encoded Therapeutics, Inc. HIGHLY ACTIVE REGULATORY ELEMENTS
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019032898A1 (en) 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AU2018375192B2 (en) 2017-12-01 2023-11-09 Encoded Therapeutics, Inc. Engineered DNA binding proteins
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病を治療するための組成物および方法
CA3106838A1 (en) * 2018-07-20 2020-01-23 University Of Utah Research Foundation Gene therapy for macular degeneration
SG11202101157VA (en) 2018-08-09 2021-03-30 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof for non-viral gene therapy
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
MX2021004213A (es) * 2018-10-12 2021-07-16 Genzyme Corp Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado.
EP4022097A4 (en) * 2019-08-29 2022-12-14 Siemens Healthcare Diagnostics Inc. REAGENTS AND METHODS FOR DETECTING AAV EXCRETION
WO2021067389A1 (en) 2019-09-30 2021-04-08 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
KR20220154141A (ko) * 2020-03-16 2022-11-21 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 종양-유래 바이러스 dna의 선택적 검출을 위한 조성물 및 방법
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
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
BR112023002395A2 (pt) 2020-08-23 2023-03-21 Bioverativ Therapeutics Inc Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna)
AR126846A1 (es) 2021-08-23 2023-11-22 Bioverativ Therapeutics Inc Genes del factor viii optimizados
CN117881696A (zh) 2021-08-23 2024-04-12 比奥维拉迪维治疗股份有限公司 具有反向末端重复序列的封闭末端dna产生
AU2022334711A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Baculovirus expression system
EP4408874A1 (en) 2021-09-30 2024-08-07 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
AU2022373653A1 (en) 2021-10-18 2024-05-02 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
AR132545A1 (es) * 2023-04-26 2025-07-16 Sanofi Sa Líneas celulares hospedadoras y métodos para identificar y usar dichas líneas celulares hospedadoras

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
US6995006B2 (en) 1997-09-05 2006-02-07 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
JP4827353B2 (ja) 1999-08-09 2011-11-30 ターゲティッド ジェネティクス コーポレイション 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大
WO2001027303A1 (en) 1999-10-12 2001-04-19 The University Of North Carolina At Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
WO2001092551A2 (en) 2000-06-01 2001-12-06 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
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
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
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
SMT201800554T1 (it) 2009-06-16 2018-11-09 Genzyme Corp Metodi migliorati per la purificazione di vettori di aav ricombinanti
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
SG10202007803QA (en) 2011-02-17 2020-09-29 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
JP6454643B2 (ja) * 2012-10-26 2019-01-16 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用
CA3178379A1 (en) * 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
CR20170407A (es) 2015-02-10 2017-11-14 Genzyme Corp Mejora del suministro de partículas virales al cuerpo estriado y al córtex
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Hum. Gene Ther., Vol.22, No.5, pp.613-624(2011.03.18.)*

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PH12017501836A1 (en) 2018-04-23
BR112017021505A2 (pt) 2018-07-03
US20190048362A1 (en) 2019-02-14
AU2022203942A1 (en) 2022-06-23
IL283291A (en) 2021-07-29
CL2017002537A1 (es) 2018-05-11
KR20250126875A (ko) 2025-08-25
AU2016245806A1 (en) 2017-11-30
UY36611A (es) 2016-11-30
JP2023116678A (ja) 2023-08-22
IL283291B (en) 2022-08-01
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