IL314807A - Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency - Google Patents

Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

Info

Publication number
IL314807A
IL314807A IL314807A IL31480724A IL314807A IL 314807 A IL314807 A IL 314807A IL 314807 A IL314807 A IL 314807A IL 31480724 A IL31480724 A IL 31480724A IL 314807 A IL314807 A IL 314807A
Authority
IL
Israel
Prior art keywords
rhasm
dose
hours
asm
administration
Prior art date
Application number
IL314807A
Other languages
English (en)
Hebrew (he)
Inventor
Edward H Schuchman
Robert J Desnick
Gerald F Cox
Laura P Andrews
James M Murray
Original Assignee
Icahn School Med Mount Sinai
Genzyme Corp
Edward H Schuchman
Robert J Desnick
Gerald F Cox
Laura P Andrews
James M Murray
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=IL314807(A) "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, Edward H Schuchman, Robert J Desnick, Gerald F Cox, Laura P Andrews, James M Murray filed Critical Icahn School Med Mount Sinai
Publication of IL314807A publication Critical patent/IL314807A/en

Links

Classifications

    • 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
    • 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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • 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
    • 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
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012—Sphingomyelin 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)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pulmonology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (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)
IL314807A 2009-08-28 2010-08-28 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency IL314807A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US23811309P 2009-08-28 2009-08-28
PCT/US2010/047057 WO2011025996A2 (en) 2009-08-28 2010-08-28 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

Publications (1)

Publication Number Publication Date
IL314807A true IL314807A (en) 2024-10-01

Family

ID=43625265

Family Applications (4)

Application Number Title Priority Date Filing Date
IL314807A IL314807A (en) 2009-08-28 2010-08-28 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
IL300625A IL300625B2 (en) 2009-08-28 2010-08-28 Increasing dose enzyme replacement for the treatment of acid sphingomyelinase deficiency
IL218324A IL218324B (en) 2009-08-28 2012-02-26 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
IL266915A IL266915B2 (en) 2009-08-28 2019-05-27 Increasing dose replacement enzyme for the treatment of acid sphingomyelinase deficiency

Family Applications After (3)

Application Number Title Priority Date Filing Date
IL300625A IL300625B2 (en) 2009-08-28 2010-08-28 Increasing dose enzyme replacement for the treatment of acid sphingomyelinase deficiency
IL218324A IL218324B (en) 2009-08-28 2012-02-26 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
IL266915A IL266915B2 (en) 2009-08-28 2019-05-27 Increasing dose replacement enzyme for the treatment of acid sphingomyelinase deficiency

Country Status (30)

Country Link
US (9) US8349319B2 (enExample)
EP (4) EP2470199B1 (enExample)
JP (8) JP5955219B2 (enExample)
KR (6) KR102094253B1 (enExample)
CN (3) CN105999238A (enExample)
AU (6) AU2010286550B2 (enExample)
BR (1) BR112012004377A2 (enExample)
CA (1) CA2773133A1 (enExample)
CL (1) CL2012000495A1 (enExample)
CY (1) CY1122128T1 (enExample)
DK (3) DK3482767T3 (enExample)
ES (3) ES3052684T3 (enExample)
FI (2) FI3998078T3 (enExample)
FR (1) FR22C1062I2 (enExample)
HR (3) HRP20251310T1 (enExample)
HU (3) HUE057440T2 (enExample)
IL (4) IL314807A (enExample)
LT (4) LT3998078T (enExample)
LU (1) LUC00288I2 (enExample)
MX (2) MX356873B (enExample)
NL (1) NL301211I2 (enExample)
NO (1) NO2022054I1 (enExample)
PL (3) PL2470199T3 (enExample)
PT (3) PT3482767T (enExample)
RU (2) RU2731616C2 (enExample)
SI (3) SI2470199T1 (enExample)
SM (1) SMT202500432T1 (enExample)
TR (1) TR201911293T4 (enExample)
WO (1) WO2011025996A2 (enExample)
ZA (2) ZA201201435B (enExample)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006007560A2 (en) 2004-07-01 2006-01-19 University Of Pennsylvania 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
PL2158322T3 (pl) 2007-06-06 2017-10-31 Genzyme Corp Terapia genowa dla lizosomalnych chorób spichrzeniowych
CA2773133A1 (en) * 2009-08-28 2011-03-03 Edward H. Schuchman Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
US8882701B2 (en) 2009-12-04 2014-11-11 Smiths Medical Asd, Inc. Advanced step therapy delivery for an ambulatory infusion pump and system
EP2571997A2 (en) 2010-05-21 2013-03-27 Ohmx Corporation Enzymatic activity of psa as diagnostic marker for prostate cancer
LT2685986T (lt) 2011-03-18 2020-03-10 Genzyme Corporation Gliukozilceramido sintazės slopiklis
US10350277B2 (en) 2011-09-07 2019-07-16 Icahn School Of Medicine At Mount Sinai Ceramidase and cell differentiation
JP6243846B2 (ja) 2011-11-15 2017-12-06 セントジーン アーゲー ニーマン・ピック病の診断のための方法
ES2786128T3 (es) 2012-06-01 2020-10-08 Icahn School Med Mount Sinai Niveles de ceramida en el tratamiento y prevención de infecciones
MA37975B2 (fr) 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
JP6832158B2 (ja) 2013-03-14 2021-02-24 アイカーン スクール オブ メディシン アット マウント サイナイ 治療用酸性セラミダーゼ組成物ならびにそれを作製および使用する方法
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
TR201906195T4 (tr) * 2013-05-14 2019-05-21 Centogene Ag Niemann pıck hastalığının teşhisi için metot.
MX370570B (es) 2013-06-07 2019-12-17 Genzyme Corp Marcador para trastornos de la esfingomielinasa acida y sus usos.
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
PL3672622T3 (pl) * 2017-08-24 2024-04-02 Genzyme Corporation Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy
FR3070383B1 (fr) 2017-08-31 2021-08-20 Solable Appareil de traitement d'eau
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
PY1939780A (es) * 2018-05-25 2019-12-04 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida
PT3920912T (pt) 2019-02-04 2025-09-09 Genzyme Corp Tratamento de ciliopatias usando inibidores da glucosilceramida sintase (gcs)
IL295255A (en) 2020-02-03 2022-10-01 Genzyme Corp Methods for treating neurological symptoms associated with lysosomal storage diseases
IL300090A (en) 2020-07-24 2023-03-01 Genzyme Corp Pharmaceutical preparations containing VENGLUSTAT
CN117396517A (zh) * 2021-05-27 2024-01-12 梨树爱博思股份有限公司 与酸性鞘磷脂酶蛋白特异性结合的抗体

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US4039388A (en) 1976-06-04 1977-08-02 The United States Of America As Represented By The Government Diagnostic test for Niemann-Pick disease
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
EP0865499B1 (en) 1995-09-14 2009-03-18 Virginia Tech Intellectual Properties, 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
US20050032211A1 (en) 1996-09-26 2005-02-10 Metabogal Ltd. Cell/tissue culturing device, system and method
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
MXPA01006491A (es) 1998-12-23 2003-06-06 Thompson Boyce Plant Res Expresion de antigenos de superficie de hepatitis b inmunogenicos en plantas transgenicas.
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US6770799B2 (en) 2000-03-17 2004-08-03 Thompson Boyce Plant Res 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
AU2001286636A1 (en) 2000-08-25 2002-03-13 Lysometrix Corporation Method for assaying the activity of lysosomal enzymes
US7001994B2 (en) 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
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
EP1436316B1 (en) 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted 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
BRPI0411291A (pt) * 2003-06-12 2006-08-01 Genzyme Corp esfingomielinase acida humana modificada tendo atividade aumentada e métodos para a fabricação da mesma
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
CN1897957A (zh) 2003-11-25 2007-01-17 纽约大学西奈山医学院 尼曼－皮克病的基于陪伴分子的治疗
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
CA2634042A1 (en) 2005-12-15 2007-07-12 The Research Foundation Of State University Of New York Enzymatic methods for measuring plasma and tissue sphingomylelin and phosphatidylcholine
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
CA2641070C (en) 2006-02-06 2017-01-03 Ramot At Tel-Aviv University Ltd. Enzyme replacement therapy for treating lysosomal storage diseases
CN101404927A (zh) * 2006-02-09 2009-04-08 建新公司 慢速脑室内递送
US8124103B2 (en) 2006-02-13 2012-02-28 Fraunhofer Usa, Inc Influenza antigens, vaccine compositions, and related methods
JP5302189B2 (ja) 2007-04-27 2013-10-02 長瀬産業株式会社 スフィンゴミエリナーゼ
US20110237538A1 (en) 2008-08-06 2011-09-29 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
EP3608330B1 (en) 2008-12-16 2022-11-09 Genzyme Corporation Synthetic intermediates for preparing oligosaccharide-protein conjugates
CA2773133A1 (en) * 2009-08-28 2011-03-03 Edward H. Schuchman Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
ES2664844T3 (es) * 2011-06-20 2018-04-23 Mount Sinai School Of Medicine Tratamiento dirigido contra TNF para las mucopolisacaridosis y otros trastornos lisosómicos
MX370570B (es) * 2013-06-07 2019-12-17 Genzyme Corp Marcador para trastornos de la esfingomielinasa acida y sus usos.
PL3672622T3 (pl) * 2017-08-24 2024-04-02 Genzyme Corporation Rekombinowana ludzka kwaśna sfingomielinaza do zastosowania w leczeniu nieprawidłowych stanów kości u pacjentów z niedoborem kwaśnej sfingomielinazy

Also Published As

Publication number Publication date
CN106047835A (zh) 2016-10-26
NL301211I2 (nl) 2023-02-02
KR20210107153A (ko) 2021-08-31
US20180055916A1 (en) 2018-03-01
ES3052684T3 (en) 2026-01-13
JP7605949B2 (ja) 2024-12-24
PL2470199T3 (pl) 2019-11-29
HUS2200050I1 (hu) 2022-12-28
ZA201201435B (en) 2020-05-27
SI2470199T1 (sl) 2019-11-29
CA2773133A1 (en) 2011-03-03
RU2020128994A (ru) 2022-03-02
TR201911293T4 (tr) 2019-08-21
SI3998078T1 (sl) 2026-02-27
EP4681775A2 (en) 2026-01-21
US20110052559A1 (en) 2011-03-03
IL266915B1 (en) 2023-03-01
RU2569744C2 (ru) 2015-11-27
AU2016216625B2 (en) 2018-07-05
LT2470199T (lt) 2019-10-10
JP7395693B2 (ja) 2023-12-11
US20130078231A1 (en) 2013-03-28
CN102596232A (zh) 2012-07-18
ES2902229T3 (es) 2022-03-25
LUC00288I2 (enExample) 2025-05-12
DK3998078T3 (da) 2025-11-17
IL300625B2 (en) 2025-01-01
IL300625B1 (en) 2024-09-01
JP7167281B2 (ja) 2022-11-08
AU2020256322A1 (en) 2020-11-12
US20190151419A1 (en) 2019-05-23
JP6952810B2 (ja) 2021-10-20
AU2025205539A1 (en) 2025-08-07
AU2020256322B2 (en) 2024-02-29
RU2731616C2 (ru) 2020-09-07
LTC3482767I2 (enExample) 2024-03-25
PL3998078T3 (pl) 2026-02-16
JP2024026214A (ja) 2024-02-28
DK3482767T3 (da) 2021-12-20
FI3998078T3 (fi) 2025-12-04
US8658162B2 (en) 2014-02-25
CL2012000495A1 (es) 2014-02-07
KR102893390B1 (ko) 2025-11-28
US8349319B2 (en) 2013-01-08
EP3482767B1 (en) 2021-10-06
IL218324A0 (en) 2012-04-30
BR112012004377A2 (pt) 2017-12-12
SMT202500432T1 (it) 2026-01-12
JP2020090513A (ja) 2020-06-11
HUE046598T2 (hu) 2020-03-30
FR22C1062I1 (fr) 2023-02-03
PT3482767T (pt) 2021-12-29
KR20220104271A (ko) 2022-07-26
JP5955219B2 (ja) 2016-07-20
RU2015145255A (ru) 2019-01-11
AU2010286550A1 (en) 2012-03-15
HRP20211992T1 (hr) 2022-04-01
WO2011025996A3 (en) 2012-03-15
AU2024201085B2 (en) 2025-04-24
NO2022054I1 (no) 2022-12-01
KR20250171458A (ko) 2025-12-08
JP2013502933A (ja) 2013-01-31
US10188705B2 (en) 2019-01-29
AU2018204156B2 (en) 2020-07-16
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