TW201124160A - SiRNA compounds comprising terminal substitutions - Google Patents
SiRNA compounds comprising terminal substitutions Download PDFInfo
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- TW201124160A TW201124160A TW099141067A TW99141067A TW201124160A TW 201124160 A TW201124160 A TW 201124160A TW 099141067 A TW099141067 A TW 099141067A TW 99141067 A TW99141067 A TW 99141067A TW 201124160 A TW201124160 A TW 201124160A
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- nucleic acid
- double
- acid molecule
- modified
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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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
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- A—HUMAN NECESSITIES
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- A61P11/16—Central respiratory analeptics
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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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- A—HUMAN NECESSITIES
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- 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
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- A—HUMAN NECESSITIES
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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
- 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
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
- A61P27/06—Antiglaucoma agents or miotics
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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
- A61P27/16—Otologicals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
- 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
- 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
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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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- 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
- C12N15/11—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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- 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]
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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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- 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
- C12N2310/31—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
-
- C—CHEMISTRY; METALLURGY
- 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)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (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)
- Urology & Nephrology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Epidemiology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Vascular Medicine (AREA)
- Dermatology (AREA)
Applications Claiming Priority (3)
| 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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| TW201124160A true TW201124160A (en) | 2011-07-16 |
Family
ID=43589717
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW099141067A TW201124160A (en) | 2009-11-26 | 2010-11-25 | SiRNA compounds comprising terminal substitutions |
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 |
|---|---|---|---|---|
| JP5830460B2 (ja) | 2009-03-23 | 2015-12-09 | クォーク ファーマシューティカルズ インコーポレーティッドQuark Pharmaceuticals,Inc. | 癌および線維性疾患を治療する化合物、組成物、および方法 |
| EP2504435B1 (en) * | 2009-11-26 | 2019-11-13 | Quark Pharmaceuticals, Inc. | Sirna compounds comprising terminal substitutions |
| TWI611021B (zh) * | 2009-12-09 | 2018-01-11 | 日東電工股份有限公司 | Hsp47表現之調節 |
| WO2011084193A1 (en) | 2010-01-07 | 2011-07-14 | Quark Pharmaceuticals, Inc. | Oligonucleotide compounds comprising non-nucleotide overhangs |
| AU2011338682B2 (en) | 2010-12-06 | 2017-04-27 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising threose modifications |
| CN103492572A (zh) | 2011-03-03 | 2014-01-01 | 夸克医药公司 | 用于治疗肺疾病和损伤的组合物和方法 |
| WO2012118911A1 (en) | 2011-03-03 | 2012-09-07 | Quark Pharmaceuticals, Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| US9796979B2 (en) | 2011-03-03 | 2017-10-24 | Quark Pharmaceuticals Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| BR112014010769B1 (pt) | 2011-11-03 | 2021-08-03 | Quark Pharmaceuticals, Inc | Uso de um composto de rna de fita dupla para preparar um medicamento para tratar doença de ménière |
| WO2013070821A1 (en) | 2011-11-08 | 2013-05-16 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the nervous system |
| CA2895077A1 (en) * | 2011-12-12 | 2013-06-20 | Beverly Packard | In vivo delivery of oligonucleotides |
| JP2015509085A (ja) | 2012-01-01 | 2015-03-26 | キュービーアイ エンタープライゼズ リミテッドQbi Enterprises Ltd. | 治療剤および診断剤の選択的送達のためのendo180を標的とする粒子 |
| DK2895607T3 (da) | 2012-09-12 | 2021-05-25 | Quark Pharmaceuticals Inc | Dobbeltstrengede oligonukleotidmolekyler til ddit4 og fremgangsmåder til anvendelse deraf |
| WO2014043292A1 (en) | 2012-09-12 | 2014-03-20 | Quark Pharmaceuticals, Inc. | Double-stranded oligonucleotide molecules to p53 and methods of use thereof |
| WO2014043291A1 (en) | 2012-09-12 | 2014-03-20 | Quark Pharmaceuticals, Inc. | Double-stranded nucleic acid compounds |
| US9889200B2 (en) | 2013-07-31 | 2018-02-13 | Qbi Enterprises Ltd. | Sphingolipid-polyalkylamine-oligonucleotide compounds |
| WO2015015498A1 (en) | 2013-07-31 | 2015-02-05 | Qbi Enterprises Ltd. | Methods of use of 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 |
| DK3444349T3 (da) | 2014-05-29 | 2021-04-12 | Quark Pharmaceuticals Inc | Fremgangsmåder og sammensætninger til forebyggelse af iskæmsik reperfusionslæsion i organer |
| US20160089391A1 (en) * | 2014-09-30 | 2016-03-31 | Temple University-Of The Commonwealth System Of Higher Education | Molecular Targets for Cardiovascular Disease |
| MA45469A (fr) * | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques de bêta-caténine et leurs utilisations |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| CA3074320A1 (en) * | 2017-09-14 | 2019-03-21 | Arrowhead Pharmaceuticals, Inc. | Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use |
| 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 |
| EP4608424A1 (en) | 2022-10-24 | 2025-09-03 | AGS Therapeutics SAS | Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof |
| 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 EP EP10785571.0A patent/EP2504435B1/en not_active Not-in-force
- 2010-11-25 KR KR1020127012259A patent/KR20120102630A/ko not_active Withdrawn
- 2010-11-25 NZ NZ599237A patent/NZ599237A/en unknown
- 2010-11-25 US US13/511,111 patent/US8796239B2/en active Active
- 2010-11-25 DK DK10785571T patent/DK2504435T3/da active
- 2010-11-25 CA CA2776568A patent/CA2776568A1/en not_active Abandoned
- 2010-11-25 CN CN201080049901XA patent/CN102597239A/zh active Pending
- 2010-11-25 TW TW099141067A patent/TW201124160A/zh unknown
- 2010-11-25 JP JP2012541211A patent/JP2013511990A/ja active Pending
- 2010-11-25 ES ES10785571T patent/ES2759003T3/es active Active
- 2010-11-25 AU AU2010324658A patent/AU2010324658A1/en not_active Abandoned
- 2010-11-25 WO PCT/US2010/058123 patent/WO2011066475A1/en not_active Ceased
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2012
- 2012-04-01 IL IL218965A patent/IL218965A0/en unknown
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2014
- 2014-08-04 US US14/451,233 patent/US9701960B2/en not_active Expired - Fee Related
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- 2017-07-06 US US15/642,581 patent/US10494631B2/en not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| WO2011066475A1 (en) | 2011-06-03 |
| US20120283309A1 (en) | 2012-11-08 |
| US20180030441A1 (en) | 2018-02-01 |
| JP2013511990A (ja) | 2013-04-11 |
| US10494631B2 (en) | 2019-12-03 |
| EP2504435A1 (en) | 2012-10-03 |
| KR20120102630A (ko) | 2012-09-18 |
| EP2504435B1 (en) | 2019-11-13 |
| ES2759003T3 (es) | 2020-05-07 |
| US9701960B2 (en) | 2017-07-11 |
| CA2776568A1 (en) | 2011-06-03 |
| CN102597239A (zh) | 2012-07-18 |
| US8796239B2 (en) | 2014-08-05 |
| NZ599237A (en) | 2014-03-28 |
| AU2010324658A1 (en) | 2012-05-03 |
| IL218965A0 (en) | 2012-07-31 |
| US20150152412A1 (en) | 2015-06-04 |
| DK2504435T3 (da) | 2019-12-09 |
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