DK2504435T3 - Sirna-forbindelser omfattende terminale substitutioner - Google Patents
Sirna-forbindelser omfattende terminale substitutioner Download PDFInfo
- Publication number
- DK2504435T3 DK2504435T3 DK10785571T DK10785571T DK2504435T3 DK 2504435 T3 DK2504435 T3 DK 2504435T3 DK 10785571 T DK10785571 T DK 10785571T DK 10785571 T DK10785571 T DK 10785571T DK 2504435 T3 DK2504435 T3 DK 2504435T3
- Authority
- DK
- Denmark
- Prior art keywords
- sirna
- including terminal
- connections including
- terminal substitutions
- substitutions
- Prior art date
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/02—Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- 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
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- 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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
- A61P11/16—Central respiratory analeptics
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- A—HUMAN NECESSITIES
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- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P27/00—Drugs for disorders of the senses
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- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
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- 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
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- 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
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/333—Modified A
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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Ophthalmology & Optometry (AREA)
- Immunology (AREA)
- Pulmonology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Urology & Nephrology (AREA)
- Epidemiology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Dermatology (AREA)
- Transplantation (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US26466809P | 2009-11-26 | 2009-11-26 | |
| US29572110P | 2010-01-17 | 2010-01-17 | |
| US37207210P | 2010-08-09 | 2010-08-09 | |
| PCT/US2010/058123 WO2011066475A1 (en) | 2009-11-26 | 2010-11-25 | Sirna compounds comprising terminal substitutions |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2504435T3 true DK2504435T3 (da) | 2019-12-09 |
Family
ID=43589717
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK10785571T DK2504435T3 (da) | 2009-11-26 | 2010-11-25 | Sirna-forbindelser omfattende terminale substitutioner |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US8796239B2 (enExample) |
| EP (1) | EP2504435B1 (enExample) |
| JP (1) | JP2013511990A (enExample) |
| KR (1) | KR20120102630A (enExample) |
| CN (1) | CN102597239A (enExample) |
| AU (1) | AU2010324658A1 (enExample) |
| CA (1) | CA2776568A1 (enExample) |
| DK (1) | DK2504435T3 (enExample) |
| ES (1) | ES2759003T3 (enExample) |
| IL (1) | IL218965A0 (enExample) |
| NZ (1) | NZ599237A (enExample) |
| TW (1) | TW201124160A (enExample) |
| WO (1) | WO2011066475A1 (enExample) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8444983B2 (en) | 2009-03-23 | 2013-05-21 | Quark Pharmaceuticals, Inc. | Composition of anti-ENDO180 antibodies and methods of use for the treatment of cancer and fibrotic diseases |
| TW201124160A (en) * | 2009-11-26 | 2011-07-16 | Quark Pharmaceuticals Inc | SiRNA compounds comprising terminal substitutions |
| CA2781896C (en) | 2009-12-09 | 2021-03-30 | Nitto Denko Corporation | Modulation of hsp47 expression |
| WO2011084193A1 (en) | 2010-01-07 | 2011-07-14 | Quark Pharmaceuticals, Inc. | Oligonucleotide compounds comprising non-nucleotide overhangs |
| CA2818024C (en) | 2010-12-06 | 2019-09-24 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising positional modifications |
| AU2012223366B2 (en) | 2011-03-03 | 2017-02-23 | Quark Pharmaceuticals, Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| EP2681314B1 (en) | 2011-03-03 | 2017-11-01 | Quark Pharmaceuticals, Inc. | Compositions and methods for treating lung disease and injury |
| US9796979B2 (en) | 2011-03-03 | 2017-10-24 | Quark Pharmaceuticals Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| EP2773758B1 (en) | 2011-11-03 | 2017-06-07 | Quark Pharmaceuticals, Inc. | Compositions for use in neuroprotection |
| US20140323549A1 (en) | 2011-11-08 | 2014-10-30 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the nervous system |
| JP2015502365A (ja) * | 2011-12-12 | 2015-01-22 | オンコイミューニン,インコーポレイティド | オリゴヌクレオチドのイン−ビボ送達 |
| WO2013098813A1 (en) | 2012-01-01 | 2013-07-04 | Qbi Enterprises Ltd. | Endo180-targeted particles for selective delivery of therapeutic and diagnostic agents |
| WO2014043289A2 (en) | 2012-09-12 | 2014-03-20 | Quark Pharmaceuticals, Inc. | Double-stranded oligonucleotide molecules to ddit4 and methods of use thereof |
| WO2014043291A1 (en) | 2012-09-12 | 2014-03-20 | Quark Pharmaceuticals, Inc. | Double-stranded nucleic acid compounds |
| EP2895608B1 (en) | 2012-09-12 | 2018-12-05 | Quark Pharmaceuticals, Inc. | Double-stranded oligonucleotide molecules to p53 and methods of use thereof |
| EP3027223A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| EP3027222A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Sphingolipid-polyalkylamine-oligonucleotide compounds |
| KR101696704B1 (ko) | 2013-12-17 | 2017-01-16 | 주식회사 인코드젠 | 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도 |
| KR101627404B1 (ko) | 2014-04-08 | 2016-06-03 | 성균관대학교산학협력단 | Dab2 유전자가 넉 다운된 수지상 세포를 포함하는 암 예방 또는 치료용 약학적 조성물 |
| DK3149172T3 (en) | 2014-05-29 | 2018-08-20 | Quark Pharmaceuticals Inc | METHODS AND COMPOSITIONS FOR THE PREVENTION OF ISKEMIC REPERFUSION LESSON IN BODIES |
| US20160089391A1 (en) * | 2014-09-30 | 2016-03-31 | Temple University-Of The Commonwealth System Of Higher Education | Molecular Targets for Cardiovascular Disease |
| WO2017173297A1 (en) * | 2016-04-01 | 2017-10-05 | Avidity Biosciences Llc | Beta-catenin nucleic acids and uses thereof |
| US20180193270A1 (en) | 2016-11-29 | 2018-07-12 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| MX2020001912A (es) * | 2017-09-14 | 2020-03-24 | Arrowhead Pharmaceuticals Inc | Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso. |
| WO2019165267A1 (en) * | 2018-02-22 | 2019-08-29 | The Regents Of The University Of California | Methods for treating brain injury |
| CN112331258B (zh) * | 2020-11-06 | 2024-02-23 | 大连大学 | 基于dna笼状结构的人工神经元计算模型构建方法 |
| KR102404883B1 (ko) * | 2020-11-30 | 2022-06-07 | (주)이노보테라퓨틱스 | 벤즈브로마론을 포함하는 켈로이드 또는 비대흉터 예방 또는 치료용 약학 조성물 |
| CA3226019A1 (en) | 2021-07-20 | 2023-01-26 | Ags Therapeutics Sas | Extracellular vesicles from microalgae, their preparation, and uses |
| WO2023144127A1 (en) | 2022-01-31 | 2023-08-03 | Ags Therapeutics Sas | Extracellular vesicles from microalgae, their biodistribution upon administration, and uses |
| WO2023232976A1 (en) | 2022-06-03 | 2023-12-07 | Ags Therapeutics Sas | Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses |
| JP2025535944A (ja) | 2022-10-24 | 2025-10-30 | エイジーエス・セラピューティクス・ソシエテ・パール・アクシオン・サンプリフィエ | 微細藻類由来の細胞外小胞、鼻腔内投与時のそれらの生体内分布、及びそれらの使用 |
| WO2025176843A1 (en) | 2024-02-21 | 2025-08-28 | Ags Therapeutics Sas | Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof |
| CN120574835B (zh) * | 2025-07-31 | 2025-10-03 | 羿美诚健(上海)生物医药有限公司 | 一种靶向rankl基因的小核酸及其在治疗骨科疾病中的应用 |
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-
2010
- 2010-11-25 TW TW099141067A patent/TW201124160A/zh unknown
- 2010-11-25 JP JP2012541211A patent/JP2013511990A/ja active Pending
- 2010-11-25 WO PCT/US2010/058123 patent/WO2011066475A1/en not_active Ceased
- 2010-11-25 CN CN201080049901XA patent/CN102597239A/zh active Pending
- 2010-11-25 AU AU2010324658A patent/AU2010324658A1/en not_active Abandoned
- 2010-11-25 NZ NZ599237A patent/NZ599237A/en unknown
- 2010-11-25 US US13/511,111 patent/US8796239B2/en active Active
- 2010-11-25 ES ES10785571T patent/ES2759003T3/es active Active
- 2010-11-25 EP EP10785571.0A patent/EP2504435B1/en not_active Not-in-force
- 2010-11-25 DK DK10785571T patent/DK2504435T3/da active
- 2010-11-25 CA CA2776568A patent/CA2776568A1/en not_active Abandoned
- 2010-11-25 KR KR1020127012259A patent/KR20120102630A/ko not_active Withdrawn
-
2012
- 2012-04-01 IL IL218965A patent/IL218965A0/en unknown
-
2014
- 2014-08-04 US US14/451,233 patent/US9701960B2/en not_active Expired - Fee Related
-
2017
- 2017-07-06 US US15/642,581 patent/US10494631B2/en not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| US20180030441A1 (en) | 2018-02-01 |
| US9701960B2 (en) | 2017-07-11 |
| CN102597239A (zh) | 2012-07-18 |
| US10494631B2 (en) | 2019-12-03 |
| US20150152412A1 (en) | 2015-06-04 |
| US20120283309A1 (en) | 2012-11-08 |
| TW201124160A (en) | 2011-07-16 |
| AU2010324658A1 (en) | 2012-05-03 |
| WO2011066475A1 (en) | 2011-06-03 |
| NZ599237A (en) | 2014-03-28 |
| EP2504435A1 (en) | 2012-10-03 |
| JP2013511990A (ja) | 2013-04-11 |
| EP2504435B1 (en) | 2019-11-13 |
| IL218965A0 (en) | 2012-07-31 |
| ES2759003T3 (es) | 2020-05-07 |
| KR20120102630A (ko) | 2012-09-18 |
| US8796239B2 (en) | 2014-08-05 |
| CA2776568A1 (en) | 2011-06-03 |
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