RU2016150629A - Множественные олигонуклеотидные фрагменты на пептидном носителе - Google Patents
Множественные олигонуклеотидные фрагменты на пептидном носителе Download PDFInfo
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- RU2016150629A RU2016150629A RU2016150629A RU2016150629A RU2016150629A RU 2016150629 A RU2016150629 A RU 2016150629A RU 2016150629 A RU2016150629 A RU 2016150629A RU 2016150629 A RU2016150629 A RU 2016150629A RU 2016150629 A RU2016150629 A RU 2016150629A
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- aon
- compounds
- subunits
- conjugated
- phosphorodiamidate
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- 108090000765 processed proteins & peptides Proteins 0.000 title claims 5
- 108091034117 Oligonucleotide Proteins 0.000 title 1
- 239000012634 fragment Substances 0.000 title 1
- RTINQKRHLKCKBZ-UHFFFAOYSA-N diaminophosphinic acid;morpholine Chemical class NP(N)(O)=O.C1COCCN1 RTINQKRHLKCKBZ-UHFFFAOYSA-N 0.000 claims 6
- KDCGOANMDULRCW-UHFFFAOYSA-N 7H-purine Chemical compound N1=CNC2=NC=NC2=C1 KDCGOANMDULRCW-UHFFFAOYSA-N 0.000 claims 4
- 230000000295 complement effect Effects 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 239000002213 purine nucleotide Substances 0.000 claims 2
- 239000002719 pyrimidine nucleotide Substances 0.000 claims 2
- 150000003230 pyrimidines Chemical class 0.000 claims 2
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 150000001875 compounds Chemical class 0.000 claims 1
- -1 phosphorodiamidate morpholine compound Chemical class 0.000 claims 1
Classifications
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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
- 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
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
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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
- 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
-
- 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
- C12N2310/11—Antisense
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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
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
-
- 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
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Claims (14)
1. Конъюгированное с пептидом AON соединение, содержащее по меньшей мере два AON соединения, содержащих 15-30 субъединиц Формул (I), (II), (III), (IV), (V) или (VI):
где B представляет собой встречающееся в природе пуриновое или пиримидиновое нуклеотидное основание, выбранное из C, G, A или T;
где указанные по меньшей мере два AON соединения имеют последовательность нуклеотидных оснований, комплементарную по меньшей мере 75% оснований пре-мРНК-мишени или мРНК-мишени;
где указанные по меньшей мере два AON соединения содержат от 0 до 3 повторяющихся субъединиц, где В представляет собой G;
где указанные по меньшей мере два AON соединения содержат менее чем 60% указанных субъединиц, где В представляет собой С или G;
где указанные по меньшей мере два AON соединения содержат не комплементарную самой себе последовательность; и
где указанные по меньшей мере два AON соединения конъюгированы с поликатионным пептидом.
2. Конъюгированное с пептидом фосфородиамидатное морфолиновое соединение, содержащее по меньшей мере два фосфородиамидатные морфолиновые соединения, содержащие 15-30 субъединиц Формулы (I): 
где R представляет собой алкильную группу, и B представляет собой встречающееся в природе пуриновое или пиримидиновое нуклеотидное основание, выбранное из C, G, A или T;
где указанные по меньшей мере два фосфородиамидатные морфолиновые соединения имеют последовательность нуклеотидных оснований, комплементарную по меньшей мере 8 смежных оснований пре-мРНК-мишени или мРНК-мишени;
где указанные по меньшей мере два фосфородиамидатные морфолиновые соединения содержат от 0 до 3 повторяющихся субъединиц, где В представляет собой G;
где указанные по меньшей мере два фосфородиамидатные морфолиновые соединения содержат менее чем 60% указанных субъединиц, где В представляет собой С или G;
где указанные по меньшей мере два фосфородиамидатные морфолиновые соединения содержат не комплементарную самой себе последовательность; и
где указанные по меньшей мере два фосфородиамидатные морфолиновые соединения конъюгированы с поликатионным пептидом.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462002296P | 2014-05-23 | 2014-05-23 | |
| US62/002,296 | 2014-05-23 | ||
| PCT/US2015/032142 WO2015179742A1 (en) | 2014-05-23 | 2015-05-22 | Multiple oligonucleotide moieties on peptide carrier |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020142530A Division RU2020142530A (ru) | 2014-05-23 | 2015-05-22 | Множественные олигонуклеотидные фрагменты на пептидном носителе |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2016150629A true RU2016150629A (ru) | 2018-06-25 |
| RU2016150629A3 RU2016150629A3 (ru) | 2018-12-24 |
| RU2739987C2 RU2739987C2 (ru) | 2020-12-30 |
Family
ID=53366294
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2016150629A RU2739987C2 (ru) | 2014-05-23 | 2015-05-22 | Множественные олигонуклеотидные фрагменты на пептидном носителе |
| RU2020142530A RU2020142530A (ru) | 2014-05-23 | 2015-05-22 | Множественные олигонуклеотидные фрагменты на пептидном носителе |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020142530A RU2020142530A (ru) | 2014-05-23 | 2015-05-22 | Множественные олигонуклеотидные фрагменты на пептидном носителе |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20170182171A1 (ru) |
| EP (1) | EP3151841B1 (ru) |
| JP (2) | JP6825914B2 (ru) |
| KR (1) | KR102487942B1 (ru) |
| CN (1) | CN106459975B (ru) |
| AR (1) | AR101542A1 (ru) |
| AU (1) | AU2015263963B2 (ru) |
| BR (1) | BR112016027236A2 (ru) |
| CA (1) | CA2949104C (ru) |
| IL (1) | IL249064B (ru) |
| MX (1) | MX2016015156A (ru) |
| RU (2) | RU2739987C2 (ru) |
| SG (2) | SG11201608880VA (ru) |
| TW (1) | TWI726844B (ru) |
| WO (1) | WO2015179742A1 (ru) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN106459975B (zh) | 2014-05-23 | 2020-03-27 | 建新公司 | 肽载体上的多个寡核苷酸部分 |
| JP7441455B2 (ja) * | 2017-09-22 | 2024-03-01 | ザ リージェンツ オブ ザ ユニバーシティ オブ コロラド，ア ボディー コーポレイト | 筋ジストロフィーの処置のためのチオモルホリノオリゴヌクレオチド |
| WO2019170731A1 (en) | 2018-03-07 | 2019-09-12 | Sanofi | Nucleotide precursors, nucleotide analogs and oligomeric compounds containing the same |
| US10765760B2 (en) * | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP4025582A1 (en) * | 2019-09-05 | 2022-07-13 | Sanofi | Oligonucleotides containing nucleotide analogs |
| WO2021211572A1 (en) * | 2020-04-14 | 2021-10-21 | Oregon State University | Antisense therapeutics for the treatment of coronavirus |
| JP2024536721A (ja) * | 2021-09-03 | 2024-10-08 | サレプタ セラピューティクス， インコーポレイテッド | 鏡像ペプチドによるアンチセンスオリゴマーの送達 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1995006056A1 (en) * | 1993-08-20 | 1995-03-02 | University Of Medicine & Dentistry Of New Jersey | Bridged polycationic polymer-oligonucleotide conjugates and methods for preparing same |
| DE19935302A1 (de) * | 1999-07-28 | 2001-02-08 | Aventis Pharma Gmbh | Konjugate und Verfahren zu deren Herstellung sowie deren Verwendung zum Transport von Molekülen über biologische Membranen |
| ES2500921T3 (es) | 2003-04-29 | 2014-10-01 | Sarepta Therapeutics, Inc. | Composiciones para potenciar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| CA2691673A1 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
| WO2009144481A2 (en) | 2008-05-30 | 2009-12-03 | Isis Innovation Limited | Conjugates for delivery of biologically active compounds |
| US8575305B2 (en) | 2008-06-04 | 2013-11-05 | Medical Research Council | Cell penetrating peptides |
| US8871918B2 (en) | 2008-10-24 | 2014-10-28 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for DMD |
| WO2012177639A2 (en) * | 2011-06-22 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Bioprocessing and bioproduction using avian cell lines |
| EP2756080B1 (en) * | 2011-09-14 | 2019-02-20 | Translate Bio MA, Inc. | Multimeric oligonucleotide compounds |
| SG11201501544PA (en) * | 2012-09-25 | 2015-03-30 | Genzyme Corp | Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy |
| US10358643B2 (en) * | 2014-01-30 | 2019-07-23 | Hoffmann-La Roche, Inc. | Poly oligomer compound with biocleavable conjugates |
| CN106459975B (zh) | 2014-05-23 | 2020-03-27 | 建新公司 | 肽载体上的多个寡核苷酸部分 |
-
2015
- 2015-05-22 CN CN201580027369.4A patent/CN106459975B/zh active Active
- 2015-05-22 SG SG11201608880VA patent/SG11201608880VA/en unknown
- 2015-05-22 RU RU2016150629A patent/RU2739987C2/ru active
- 2015-05-22 AR ARP150101604A patent/AR101542A1/es unknown
- 2015-05-22 KR KR1020167035607A patent/KR102487942B1/ko active IP Right Grant
- 2015-05-22 JP JP2016567889A patent/JP6825914B2/ja active Active
- 2015-05-22 TW TW104116447A patent/TWI726844B/zh active
- 2015-05-22 AU AU2015263963A patent/AU2015263963B2/en active Active
- 2015-05-22 RU RU2020142530A patent/RU2020142530A/ru unknown
- 2015-05-22 CA CA2949104A patent/CA2949104C/en active Active
- 2015-05-22 BR BR112016027236A patent/BR112016027236A2/pt not_active Application Discontinuation
- 2015-05-22 US US15/313,405 patent/US20170182171A1/en not_active Abandoned
- 2015-05-22 WO PCT/US2015/032142 patent/WO2015179742A1/en active Application Filing
- 2015-05-22 EP EP15727795.5A patent/EP3151841B1/en active Active
- 2015-05-22 MX MX2016015156A patent/MX2016015156A/es unknown
- 2015-05-22 SG SG10202004611SA patent/SG10202004611SA/en unknown
-
2016
- 2016-11-20 IL IL249064A patent/IL249064B/en unknown
-
2019
- 2019-02-01 US US16/265,883 patent/US11103587B2/en active Active
-
2021
- 2021-01-13 JP JP2021003149A patent/JP7269968B2/ja active Active
Also Published As
| Publication number | Publication date |
|---|---|
| MX2016015156A (es) | 2017-03-27 |
| KR20170005118A (ko) | 2017-01-11 |
| IL249064A0 (en) | 2017-01-31 |
| IL249064B (en) | 2021-07-29 |
| JP6825914B2 (ja) | 2021-02-03 |
| JP7269968B2 (ja) | 2023-05-09 |
| RU2016150629A3 (ru) | 2018-12-24 |
| CA2949104C (en) | 2023-09-26 |
| AU2015263963B2 (en) | 2021-02-18 |
| AR101542A1 (es) | 2016-12-28 |
| US11103587B2 (en) | 2021-08-31 |
| JP2017517253A (ja) | 2017-06-29 |
| SG10202004611SA (en) | 2020-06-29 |
| US20190388547A1 (en) | 2019-12-26 |
| US20170182171A1 (en) | 2017-06-29 |
| CN106459975A (zh) | 2017-02-22 |
| RU2739987C2 (ru) | 2020-12-30 |
| RU2020142530A (ru) | 2021-04-26 |
| SG11201608880VA (en) | 2016-11-29 |
| EP3151841A1 (en) | 2017-04-12 |
| TW201617373A (zh) | 2016-05-16 |
| JP2021063128A (ja) | 2021-04-22 |
| BR112016027236A2 (pt) | 2017-10-17 |
| AU2015263963A1 (en) | 2016-12-01 |
| TWI726844B (zh) | 2021-05-11 |
| EP3151841B1 (en) | 2022-10-19 |
| KR102487942B1 (ko) | 2023-01-11 |
| CN106459975B (zh) | 2020-03-27 |
| WO2015179742A1 (en) | 2015-11-26 |
| CA2949104A1 (en) | 2015-11-26 |
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