CA2768598A1 - Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes - Google Patents
Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes Download PDFInfo
- Publication number
- CA2768598A1 CA2768598A1 CA2768598A CA2768598A CA2768598A1 CA 2768598 A1 CA2768598 A1 CA 2768598A1 CA 2768598 A CA2768598 A CA 2768598A CA 2768598 A CA2768598 A CA 2768598A CA 2768598 A1 CA2768598 A1 CA 2768598A1
- Authority
- CA
- Canada
- Prior art keywords
- seq
- module
- conjugate
- covalently linked
- cell
- Prior art date
- Legal status (The legal status 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 status listed.)
- Abandoned
Links
Classifications
-
- 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
- C12N15/11—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
- A61K47/6415—Toxins or lectins, e.g. clostridial toxins or Pseudomonas exotoxins
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
- A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3517—Marker; Tag
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Toxicology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US22766909P | 2009-07-22 | 2009-07-22 | |
| US61/227669 | 2009-07-22 | ||
| PCT/EP2010/004512 WO2011009624A1 (en) | 2009-07-22 | 2010-07-22 | Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2768598A1 true CA2768598A1 (en) | 2011-01-27 |
Family
ID=42731966
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2768598A Abandoned CA2768598A1 (en) | 2009-07-22 | 2010-07-22 | Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20120258104A1 (https=) |
| EP (1) | EP2456470A1 (https=) |
| JP (1) | JP2012533587A (https=) |
| CA (1) | CA2768598A1 (https=) |
| WO (1) | WO2011009624A1 (https=) |
Families Citing this family (53)
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| US11246915B2 (en) | 2010-09-15 | 2022-02-15 | Applied Molecular Transport Inc. | Cholix toxin-derived fusion molecules for oral delivery of biologically active cargo |
| RU2593715C2 (ru) | 2010-09-15 | 2016-08-10 | Рэндэлл Дж. МРСНИ | Системы и способы доставки биоактивных средств с применением транспортных последовательностей, происходящих от бактериального токсина |
| CA2818173C (en) | 2010-11-30 | 2022-05-03 | Genentech, Inc. | Low affinity blood brain barrier receptor antibodies and uses therefor |
| US20140065172A1 (en) * | 2011-01-26 | 2014-03-06 | Cenix Bioscience Gmbh | Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes |
| WO2012113413A1 (en) * | 2011-02-21 | 2012-08-30 | Curevac Gmbh | Vaccine composition comprising complexed immunostimulatory nucleic acids and antigens packaged with disulfide-linked polyethyleneglycol/peptide conjugates |
| US9045750B2 (en) * | 2011-03-18 | 2015-06-02 | Yuelong Ma | Humanized lewis-Y specific antibody-based delivery of dicer substrate siRNA (D-siRNA) against STAT3 |
| AU2012308320C1 (en) * | 2011-09-14 | 2018-08-23 | Translate Bio Ma, Inc. | Multimeric oligonucleotide compounds |
| CN104039961A (zh) * | 2012-01-07 | 2014-09-10 | 株式会社博纳克 | 具有氨基酸骨架的单链核酸分子 |
| WO2013127829A1 (en) * | 2012-02-27 | 2013-09-06 | Universitat De Barcelona | Protease-resistant compounds useful as shuttles through the blood-brain barrier and shuttle-cargo constructs |
| SG11201407483YA (en) | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating smn gene family expression |
| EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| EA201492122A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии utrn |
| AU2013262699A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating ATP2A2 expression |
| BR112014028631A2 (pt) | 2012-05-16 | 2017-10-17 | Rana Therapeutics Inc | composições e métodos para modulação da expressão da família de genes da hemoglobina |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| WO2013180038A1 (ja) | 2012-05-26 | 2013-12-05 | 株式会社ボナック | デリバリー機能を有する遺伝子発現制御用の一本鎖核酸分子 |
| US20150247141A1 (en) | 2012-09-14 | 2015-09-03 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| EP3666795A1 (en) | 2013-03-12 | 2020-06-17 | Molecular Templates, Inc. | Cytotoxic proteins comprising cell-targeting binding regions and shiga toxin a subunit regions for selective killing of specific cell types |
| EP3035969B1 (en) | 2013-08-19 | 2022-08-03 | S.M. Discovery Holdings Inc. | Peptidic nanodelivery composition targeting two receptors |
| BR112016003361A2 (pt) | 2013-08-21 | 2017-11-21 | Curevac Ag | vacina do vírus sincicial respiratório (rsv) |
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| WO2015099122A1 (ja) | 2013-12-26 | 2015-07-02 | 学校法人東京医科大学 | 遺伝子発現制御のための人工ミミックmiRNAおよびその用途 |
| CN106068324B (zh) | 2013-12-27 | 2020-12-29 | 株式会社博纳克 | 控制基因表达的人工匹配型miRNA及其用途 |
| CN112851769A (zh) | 2014-01-27 | 2021-05-28 | 分子模板公司 | 用于哺乳动物中的去免疫化志贺毒素a亚基效应子多肽 |
| US11142584B2 (en) | 2014-03-11 | 2021-10-12 | Molecular Templates, Inc. | CD20-binding proteins comprising Shiga toxin A subunit effector regions for inducing cellular internalization and methods using same |
| JP6935195B2 (ja) * | 2014-03-11 | 2021-09-15 | モレキュラー テンプレーツ, インク.Molecular Templates, Inc. | 結合領域、志賀毒素aサブユニットのエフェクター領域、及びカルボキシ末端小胞体局在化シグナルモチーフを含むタンパク質 |
| EP3126501A1 (en) | 2014-03-24 | 2017-02-08 | RiboxX GmbH | Double-stranded rna conjugates and their use |
| CN106470707A (zh) | 2014-05-07 | 2017-03-01 | 应用分子运输有限责任公司 | 用于口服递送生物活性货物的cholix毒素衍生的融合分子 |
| CN106604934A (zh) | 2014-06-11 | 2017-04-26 | 分子模板公司 | 耐受蛋白酶切割的志贺毒素a亚基效应子多肽和包含其的细胞靶向分子 |
| JP2017534598A (ja) * | 2014-09-30 | 2017-11-24 | ザ メディカル カレッジ オブ ウィスコンシン インクThe Medical College Of Wisconsin, Inc. | 神経系疾患を治療する標的療法のための汎用的なプラットフォーム |
| US20170333571A1 (en) * | 2014-11-04 | 2017-11-23 | The Regents Of The University Of California | Targeted Delivery Platform for Delivery of Therapeutics |
| KR20170098914A (ko) | 2014-12-27 | 2017-08-30 | 가부시키가이샤 보낙 | 유전자 발현 제어를 위한 천연형 miRNA 및 그의 용도 |
| EP3253799B1 (en) | 2015-02-05 | 2020-12-02 | Molecular Templates, Inc. | Multivalent cd20-binding molecules comprising shiga toxin a subunit effector regions and enriched compositions thereof |
| WO2016158809A1 (ja) | 2015-03-27 | 2016-10-06 | 株式会社ボナック | デリバリー機能と遺伝子発現制御能を有する一本鎖核酸分子 |
| EP3303373B1 (en) | 2015-05-30 | 2020-04-08 | Molecular Templates, Inc. | De-immunized, shiga toxin a subunit scaffolds and cell-targeting molecules comprising the same |
| KR20180056750A (ko) * | 2015-10-05 | 2018-05-29 | 신젠타 파티서페이션즈 아게 | 리보핵산의 생물학적 활성을 보존하는 방법 |
| JP2019513371A (ja) * | 2016-04-01 | 2019-05-30 | アビディティー バイオサイエンシーズ エルエルシー | 核酸ポリペプチド組成物とその使用 |
| WO2018106895A1 (en) | 2016-12-07 | 2018-06-14 | Molecular Templates, Inc. | Shiga toxin a subunit effector polypeptides, shiga toxin effector scaffolds, and cell-targeting molecules for site-specific conjugation |
| MX2019008840A (es) | 2017-01-25 | 2019-09-09 | Molecular Templates Inc | Moleculas con direccion hacia las celulas que comprenden efectores de la sub-unidad a de la toxina shiga desinmunizados y epitopos de las celulas t cd8+. |
| AU2018378812B2 (en) | 2017-12-06 | 2025-05-22 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
| CN121513216A (zh) | 2018-03-08 | 2026-02-13 | 应用分子转运公司 | 用于经口递送的毒素衍生的递送构建体 |
| WO2019183600A1 (en) * | 2018-03-23 | 2019-09-26 | The Regents Of The University Of California | Methods and compounds for targeted autophagy |
| CN110612117B (zh) | 2018-04-17 | 2024-04-12 | 分子模板公司 | 包含去免疫化的志贺毒素a亚基支架的her2靶向分子 |
| EP3818167A4 (en) * | 2018-07-05 | 2022-04-13 | The Regents of The University of California | COMPOSITIONS AND METHODS FOR DELIVERING RNA TO A CELL |
| WO2020096695A1 (en) | 2018-11-07 | 2020-05-14 | Applied Molecular Transport Inc. | Cholix-derived carriers for oral delivery of heterologous payload |
| EP4083058A3 (en) | 2018-11-07 | 2023-01-11 | Applied Molecular Transport Inc. | Delivery constructs for transcytosis and related methods |
| MX2022001975A (es) | 2019-08-16 | 2022-03-11 | Applied Molecular Transport Inc | Composiciones, formulaciones y produccion y purificacion de interleucinas. |
| EP3868882A1 (en) * | 2020-02-21 | 2021-08-25 | European Molecular Biology Laboratory | Archaeal pyrrolysyl trna synthetases for orthogonal use |
| WO2021188390A1 (en) | 2020-03-19 | 2021-09-23 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| WO2021216997A2 (en) * | 2020-04-23 | 2021-10-28 | Cornell University | Nanotherapy targeting rhamm-positive tumors |
| CN118265544A (zh) | 2021-09-16 | 2024-06-28 | 艾维迪提生物科学公司 | 治疗面肩肱型肌营养不良的组合物和方法 |
| WO2023076820A1 (en) * | 2021-10-26 | 2023-05-04 | Navicure Biopharmaceuticals Limited | Immunogenic fusion proteins against coronavirus |
| CN121378391B (zh) * | 2025-12-24 | 2026-03-27 | 中国医学科学院药用植物研究所 | 具有抗肿瘤作用的龟源环肽化合物及其制备方法 |
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| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
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| WO1997013529A1 (en) * | 1995-10-13 | 1997-04-17 | The Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services | Immunotoxin containing a disulfide-stabilized antibody fragment |
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| WO2005084158A2 (en) | 2003-06-20 | 2005-09-15 | The Regents Of The University Of California | Polypeptide transduction and fusogenic peptides |
| US20100240597A1 (en) * | 2007-06-15 | 2010-09-23 | Arkansas State University | Methods of delivery of molecules to cells using a ricin subunit and compositions relating to same |
| WO2009045457A2 (en) | 2007-10-02 | 2009-04-09 | Rxi Pharmaceuticals Corp. | Tripartite rnai constructs |
-
2010
- 2010-07-22 JP JP2012520958A patent/JP2012533587A/ja active Pending
- 2010-07-22 WO PCT/EP2010/004512 patent/WO2011009624A1/en not_active Ceased
- 2010-07-22 US US13/386,347 patent/US20120258104A1/en not_active Abandoned
- 2010-07-22 EP EP10737781A patent/EP2456470A1/en not_active Withdrawn
- 2010-07-22 CA CA2768598A patent/CA2768598A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| US20120258104A1 (en) | 2012-10-11 |
| EP2456470A1 (en) | 2012-05-30 |
| JP2012533587A (ja) | 2012-12-27 |
| WO2011009624A1 (en) | 2011-01-27 |
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