PT2470199T - Terapia de substituição enzimática com escaladas de dose para tratamento de deficência em esfingomielinase ácida - Google Patents

Terapia de substituição enzimática com escaladas de dose para tratamento de deficência em esfingomielinase ácida

Info

Publication number
PT2470199T
PT2470199T PT10812683T PT10812683T PT2470199T PT 2470199 T PT2470199 T PT 2470199T PT 10812683 T PT10812683 T PT 10812683T PT 10812683 T PT10812683 T PT 10812683T PT 2470199 T PT2470199 T PT 2470199T
Authority
PT
Portugal
Prior art keywords
replacement therapy
dose escalation
enzyme replacement
acid sphingomyelinase
treating acid
Prior art date
Application number
PT10812683T
Other languages
English (en)
Inventor
H Schuchman Edward
J Desnick Robert
f cox Gerald
P Andrews Laura
M Murray James
Original Assignee
Icahn School Med Mount Sinai
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=43625265&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PT2470199(T) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Icahn School Med Mount Sinai, Genzyme Corp filed Critical Icahn School Med Mount Sinai
Publication of PT2470199T publication Critical patent/PT2470199T/pt

Links

Classifications

    • 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
    • A61K38/43Enzymes; Proenzymes; Derivatives 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
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012Sphingomyelin phosphodiesterase (3.1.4.12)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Pulmonology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Dermatology (AREA)
PT10812683T 2009-08-28 2010-08-28 Terapia de substituição enzimática com escaladas de dose para tratamento de deficência em esfingomielinase ácida PT2470199T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US23811309P 2009-08-28 2009-08-28

Publications (1)

Publication Number Publication Date
PT2470199T true PT2470199T (pt) 2019-08-07

Family

ID=43625265

Family Applications (2)

Application Number Title Priority Date Filing Date
PT181993585T PT3482767T (pt) 2009-08-28 2010-08-28 Terapia de substituição enzimática com escaladas de dose para tratamento de deficência da esfingomielinase ácida
PT10812683T PT2470199T (pt) 2009-08-28 2010-08-28 Terapia de substituição enzimática com escaladas de dose para tratamento de deficência em esfingomielinase ácida

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PT181993585T PT3482767T (pt) 2009-08-28 2010-08-28 Terapia de substituição enzimática com escaladas de dose para tratamento de deficência da esfingomielinase ácida

Country Status (27)

Country Link
US (9) US8349319B2 (pt)
EP (3) EP3998078A1 (pt)
JP (7) JP5955219B2 (pt)
KR (5) KR102094253B1 (pt)
CN (3) CN106047835A (pt)
AU (5) AU2010286550B2 (pt)
BR (1) BR112012004377A2 (pt)
CA (1) CA2773133A1 (pt)
CL (1) CL2012000495A1 (pt)
CY (1) CY1122128T1 (pt)
DK (2) DK3482767T3 (pt)
ES (2) ES2738859T3 (pt)
FR (1) FR22C1062I2 (pt)
HR (2) HRP20211992T1 (pt)
HU (3) HUE046598T2 (pt)
IL (4) IL300625B1 (pt)
LT (2) LT3482767T (pt)
MX (1) MX356873B (pt)
NL (1) NL301211I2 (pt)
NO (1) NO2022054I1 (pt)
PL (2) PL3482767T3 (pt)
PT (2) PT3482767T (pt)
RU (3) RU2569744C2 (pt)
SI (2) SI3482767T1 (pt)
TR (1) TR201911293T4 (pt)
WO (1) WO2011025996A2 (pt)
ZA (2) ZA201201435B (pt)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8889127B2 (en) 2004-07-01 2014-11-18 Icahn School Of Medicine At Mount Sinai Targeted protein replacement for the treatment of lysosomal storage disorders
US20080228056A1 (en) 2007-03-13 2008-09-18 Michael Blomquist Basal rate testing using frequent blood glucose input
US7751907B2 (en) 2007-05-24 2010-07-06 Smiths Medical Asd, Inc. Expert system for insulin pump therapy
US8221345B2 (en) 2007-05-30 2012-07-17 Smiths Medical Asd, Inc. Insulin pump based expert system
ES2905616T3 (es) 2007-06-06 2022-04-11 Genzyme Corp Terapia génica para enfermedades de almacenamiento lisosómico
PT3482767T (pt) * 2009-08-28 2021-12-29 Icahn School Med Mount Sinai Terapia de substituição enzimática com escaladas de dose para tratamento de deficência da esfingomielinase ácida
US8882701B2 (en) * 2009-12-04 2014-11-11 Smiths Medical Asd, Inc. Advanced step therapy delivery for an ambulatory infusion pump and system
WO2011146143A2 (en) 2010-05-21 2011-11-24 Ohmx Corporation Detection of cancer by assaying psa enzymatic activity
DK2685986T3 (da) 2011-03-18 2020-03-02 Genzyme Corp Glucosylceramidsyntasehæmmer
HUE051021T2 (hu) 2011-09-07 2021-01-28 Sinai School Medicine Ceramidáz és sejtek differenciálódása
MX359328B (es) 2011-11-15 2018-09-25 Centogene Ag Metodo para el diagnostico de enfermedad de niemann-pick.
DK2854910T3 (da) 2012-06-01 2020-05-04 Icahn School Med Mount Sinai Ceramidniveauer i behandling og forebyggelse af infektioner
MA37975B2 (fr) 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
CA2905449A1 (en) 2013-03-14 2014-10-02 Icahn School Of Medicine At Mount Sinai Therapeutic acid ceramidase compositions and methods of making and using them
JO3713B1 (ar) 2013-03-15 2021-01-31 Genzyme Corp أشكال ملح (s)-كوينوكليدين-3-يل(2-(2-(4-فلوروفينيل)ثيازول-4-يل)بروبان-2-يل)كارباميت
CA2912274A1 (en) * 2013-05-14 2014-11-20 Centogene Ag Method for the diagnosis of niemann-pick disease
EA035342B1 (ru) * 2013-06-07 2020-05-29 Джензим Корпорейшн Способы контроля лечения пациента, имеющего ассоциированное с кислой сфингомиелиназой (asm) нарушение
JP6984854B2 (ja) * 2015-10-08 2021-12-22 国立大学法人千葉大学 ニーマン・ピック病c型を予防または治療するための医薬組成物
US10569016B2 (en) 2015-12-29 2020-02-25 Tandem Diabetes Care, Inc. System and method for switching between closed loop and open loop control of an ambulatory infusion pump
EP3672622B1 (en) * 2017-08-24 2023-11-08 Genzyme Corporation Recombinant human acid sphingomyelinase for use in treating abnormal bone conditions in acid sphingomyelinase deficiency patients
CA3076369A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
UY38238A (es) * 2018-05-25 2019-12-31 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida
AU2021215396A1 (en) 2020-02-03 2022-09-29 Genzyme Corporation Methods for treating neurological symptoms associated with lysosomal storage diseases
IL300090A (en) 2020-07-24 2023-03-01 Genzyme Corp Pharmaceutical preparations containing VENGLUSTAT

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4039388A (en) 1976-06-04 1977-08-02 The United States Of America As Represented By The Government Diagnostic test for Niemann-Pick disease
US4082781A (en) 1976-06-04 1978-04-04 The United States Of America As Represented By The Department Of Health, Education And Welfare Synthesis of 2-alkanoylamino-4-nitrophenyl phosphorylcholine-hydroxide
IL74761A0 (en) 1984-04-13 1985-06-30 Hughes Howard Med Inst Human phenylalanine hydroxylase cdna clones,their production and their use in diagnosing classical phenylketonuria
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4863195A (en) * 1987-10-02 1989-09-05 Capozzola Carl A Identification tag
US5047335A (en) 1988-12-21 1991-09-10 The Regents Of The University Of Calif. Process for controlling intracellular glycosylation of proteins
US5549892A (en) 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
US5484719A (en) 1991-08-26 1996-01-16 Edible Vaccines, Inc. Vaccines produced and administered through edible plants
US5612487A (en) 1991-08-26 1997-03-18 Edible Vaccines, Inc. Anti-viral vaccines expressed in plants
US6034298A (en) 1991-08-26 2000-03-07 Prodigene, Inc. Vaccines expressed in plants
US5641670A (en) 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US5716614A (en) 1994-08-05 1998-02-10 Molecular/Structural Biotechnologies, Inc. Method for delivering active agents to mammalian brains in a complex with eicosapentaenoic acid or docosahexaenoic acid-conjugated polycationic carrier
WO1997010353A1 (en) 1995-09-14 1997-03-20 Virginia Tech Intellectual Property, Inc. Production of lysosomal enzymes in plant-based expression systems
JP2820106B2 (ja) 1996-02-29 1998-11-05 日本電気株式会社 トラヒックシェーパ装置
IL155588A0 (en) 2003-04-27 2003-11-23 Metabogal Ltd Methods for expression of enzymatically active recombinant lysosomal enzymes in transgenic plant root cells and vectors used thereby
US20050032211A1 (en) 1996-09-26 2005-02-10 Metabogal Ltd. Cell/tissue culturing device, system and method
US6551820B1 (en) 1998-12-23 2003-04-22 Boyce Thompson Institute For Plant Research Expression of immunogenic hepatitis B surface antigens in transgenic plants
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
AU2001250856A1 (en) 2000-03-17 2001-10-03 Charles Arntzen Expression of recombinant human acetylcholinesterase in transgenic plants
US20020095135A1 (en) * 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
AR034413A1 (es) 2000-08-25 2004-02-25 Nestor Abel Chamoles Metodo para determinar la actividad de las enzimas lisosomales
US7723296B2 (en) 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
US7001994B2 (en) 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1974752B1 (en) 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subcellular targeting of therapeutic proteins
IES20011101A2 (en) * 2001-12-21 2003-06-25 Japkinstill Services Ltd A Bi-directional Messaging System
US7658916B2 (en) 2002-04-05 2010-02-09 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density
US7585518B2 (en) 2002-11-19 2009-09-08 Kimberly-Clark Worldwide, Inc. Products and methods for maintaining or increasing ceramide levels in skin
US7388079B2 (en) 2002-11-27 2008-06-17 The Regents Of The University Of California Delivery of pharmaceutical agents via the human insulin receptor
US7951557B2 (en) 2003-04-27 2011-05-31 Protalix Ltd. Human lysosomal proteins from plant cell culture
CA2528427A1 (en) 2003-06-12 2004-12-23 Genzyme Corporation Modified human acid sphingomyelinase having increased activity, and methods for making the same
US20050026823A1 (en) * 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
AU2004293115A1 (en) 2003-11-25 2005-06-09 Mount Sinai School Of Medicine Of New York University Chaperone-based therapy for Niemann-Pick disease
KR20070085291A (ko) 2004-10-13 2007-08-27 프로탈릭스 리미티드 식물 세포 배양에서 항체를 생산하는 시스템 및 방법
US7615224B2 (en) 2004-12-02 2009-11-10 Women's And Children's Hospital Multiplex-bead complex for determination of lysosomal storage disorders
EP1960534A4 (en) 2005-12-15 2009-03-25 Univ New York State Res Found ENZYMATIC METHODS FOR MEASURING THE SPHINGOMYLELIN AND PHOSPHATIDYLCHOLIN SHARE IN PLASMA AND TISSUE
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
US10265412B2 (en) 2006-02-06 2019-04-23 Ramot At Tel-Aviv University Ltd. Enzyme replacement therapy for treating lysosomal storage diseases
EP3459441A1 (en) 2006-02-09 2019-03-27 Genzyme Corporation Slow intraventricular delivery
KR20080106433A (ko) 2006-02-13 2008-12-05 프라운호퍼 유에스에이, 인코포레이티드 인플루엔자 항원, 백신 조성물 및 관련 방법
WO2008136451A1 (ja) 2007-04-27 2008-11-13 Nagase & Co., Ltd. スフィンゴミエリナーゼ
WO2010015816A2 (en) 2008-08-06 2010-02-11 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
CN105879047A (zh) 2008-12-16 2016-08-24 建新公司 寡糖-蛋白缀合物
PT3482767T (pt) * 2009-08-28 2021-12-29 Icahn School Med Mount Sinai Terapia de substituição enzimática com escaladas de dose para tratamento de deficência da esfingomielinase ácida
CN103764824B (zh) * 2011-06-20 2018-04-24 西奈山医学院 粘多糖贮积症和其它溶酶体病症的抗TNF-α疗法
EA035342B1 (ru) * 2013-06-07 2020-05-29 Джензим Корпорейшн Способы контроля лечения пациента, имеющего ассоциированное с кислой сфингомиелиназой (asm) нарушение
EP3672622B1 (en) * 2017-08-24 2023-11-08 Genzyme Corporation Recombinant human acid sphingomyelinase for use in treating abnormal bone conditions in acid sphingomyelinase deficiency patients

Also Published As

Publication number Publication date
US20110052559A1 (en) 2011-03-03
US8349319B2 (en) 2013-01-08
WO2011025996A2 (en) 2011-03-03
BR112012004377A2 (pt) 2017-12-12
KR102608546B1 (ko) 2023-12-04
JP2020090513A (ja) 2020-06-11
JP2024026214A (ja) 2024-02-28
US20130078230A1 (en) 2013-03-28
CN102596232A (zh) 2012-07-18
AU2010286550B2 (en) 2016-05-19
RU2569744C2 (ru) 2015-11-27
AU2018204156B2 (en) 2020-07-16
EP3482767A1 (en) 2019-05-15
NO2022054I1 (no) 2022-12-01
HUE046598T2 (hu) 2020-03-30
IL218324B (en) 2019-05-30
IL266915B2 (en) 2023-07-01
IL300625B1 (en) 2024-09-01
PT3482767T (pt) 2021-12-29
IL314807A (en) 2024-10-01
HUS2200050I1 (hu) 2022-12-28
PL3482767T3 (pl) 2022-02-14
SI3482767T1 (sl) 2022-03-31
ZA201601362B (en) 2017-05-31
IL266915B1 (en) 2023-03-01
AU2020256322B2 (en) 2024-02-29
HRP20191385T1 (hr) 2019-11-01
EP3482767B1 (en) 2021-10-06
KR20220104271A (ko) 2022-07-26
DK2470199T3 (da) 2019-08-05
RU2012111823A (ru) 2013-10-10
DK3482767T3 (da) 2021-12-20
JP2013502933A (ja) 2013-01-31
JP2016153408A (ja) 2016-08-25
JP7167281B2 (ja) 2022-11-08
NL301211I1 (nl) 2022-12-28
WO2011025996A9 (en) 2011-08-25
EP3998078A1 (en) 2022-05-18
LT3482767T (lt) 2022-01-10
WO2011025996A3 (en) 2012-03-15
HUE057440T2 (hu) 2022-05-28
US20210077593A1 (en) 2021-03-18
CN105999238A (zh) 2016-10-12
AU2024201085A1 (en) 2024-03-07
CY1122128T1 (el) 2020-11-25
KR20120081077A (ko) 2012-07-18
KR20200034828A (ko) 2020-03-31
JP2018076322A (ja) 2018-05-17
SI2470199T1 (sl) 2019-11-29
RU2020128994A (ru) 2022-03-02
IL218324A0 (en) 2012-04-30
US20140335070A1 (en) 2014-11-13
JP2023012499A (ja) 2023-01-25
AU2016216625A1 (en) 2016-09-15
PL2470199T3 (pl) 2019-11-29
US8658162B2 (en) 2014-02-25
US20220305091A1 (en) 2022-09-29
US10188705B2 (en) 2019-01-29
AU2020256322A1 (en) 2020-11-12
RU2015145255A (ru) 2019-01-11
ES2902229T3 (es) 2022-03-25
CN106047835A (zh) 2016-10-26
KR102094253B1 (ko) 2020-03-31
JP7395693B2 (ja) 2023-12-11
US20180055916A1 (en) 2018-03-01
ZA201201435B (en) 2020-05-27
RU2731616C2 (ru) 2020-09-07
CA2773133A1 (en) 2011-03-03
JP2022003065A (ja) 2022-01-11
US8709408B2 (en) 2014-04-29
EP2470199A4 (en) 2013-03-27
US9114139B2 (en) 2015-08-25
ES2738859T3 (es) 2020-01-27
HRP20211992T1 (hr) 2022-04-01
EP2470199A2 (en) 2012-07-04
US20190151419A1 (en) 2019-05-23
FR22C1062I1 (fr) 2023-02-03
MX356873B (es) 2018-06-18
JP6251307B2 (ja) 2017-12-20
RU2015145255A3 (pt) 2019-08-12
FR22C1062I2 (fr) 2023-12-29
LT2470199T (lt) 2019-10-10
KR102608551B1 (ko) 2023-12-04
KR20210107153A (ko) 2021-08-31
MX2012002449A (es) 2012-03-14
AU2010286550A1 (en) 2012-03-15
JP6952810B2 (ja) 2021-10-20
US20160120957A1 (en) 2016-05-05
JP5955219B2 (ja) 2016-07-20
IL266915A (en) 2019-07-31
NL301211I2 (nl) 2023-02-02
EP2470199B1 (en) 2019-05-22
KR20230164773A (ko) 2023-12-04
IL300625A (en) 2023-04-01
US9655954B2 (en) 2017-05-23
US20130078231A1 (en) 2013-03-28
CL2012000495A1 (es) 2014-02-07
AU2018204156A1 (en) 2018-06-28
AU2016216625B2 (en) 2018-07-05
TR201911293T4 (tr) 2019-08-21

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