RU2018144875A3 - - Google Patents
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- RU2018144875A3 RU2018144875A3 RU2018144875A RU2018144875A RU2018144875A3 RU 2018144875 A3 RU2018144875 A3 RU 2018144875A3 RU 2018144875 A RU2018144875 A RU 2018144875A RU 2018144875 A RU2018144875 A RU 2018144875A RU 2018144875 A3 RU2018144875 A3 RU 2018144875A3
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- 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
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/34—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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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- 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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- 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
- C12N2310/315—Phosphorothioates
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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
- C12N2310/32—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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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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
- C12N2310/336—Modified G
-
- 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
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP16177973 | 2016-07-05 | ||
| EP16177973.1 | 2016-07-05 | ||
| PCT/EP2017/066845 WO2018007475A1 (en) | 2016-07-05 | 2017-07-05 | Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2022107926A Division RU2022107926A (ru) | 2016-07-05 | 2017-07-05 | Олигонуклеотиды, производящие переключение или модулирование сплайсинга пре-рнк и содержащие бициклические каркасные фрагменты, с улучшенными характеристиками для лечения генетических заболеваний |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2018144875A RU2018144875A (ru) | 2020-08-05 |
| RU2018144875A3 true RU2018144875A3 (ru) | 2020-11-24 |
| RU2769249C2 RU2769249C2 (ru) | 2022-03-29 |
Family
ID=56360269
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2018144875A RU2769249C2 (ru) | 2016-07-05 | 2017-07-05 | Олигонуклеотиды, производящие переключение или модулирование сплайсинга пре-рнк и содержащие бициклические каркасные фрагменты, с улучшенными характеристиками для лечения генетических заболеваний |
| RU2022107926A RU2022107926A (ru) | 2016-07-05 | 2017-07-05 | Олигонуклеотиды, производящие переключение или модулирование сплайсинга пре-рнк и содержащие бициклические каркасные фрагменты, с улучшенными характеристиками для лечения генетических заболеваний |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2022107926A RU2022107926A (ru) | 2016-07-05 | 2017-07-05 | Олигонуклеотиды, производящие переключение или модулирование сплайсинга пре-рнк и содержащие бициклические каркасные фрагменты, с улучшенными характеристиками для лечения генетических заболеваний |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20180028554A1 (ru) |
| EP (2) | EP3481955A1 (ru) |
| JP (3) | JP2019522972A (ru) |
| KR (3) | KR20230175312A (ru) |
| CN (1) | CN109757108A (ru) |
| AU (2) | AU2017291960B2 (ru) |
| BR (1) | BR112019000061A2 (ru) |
| CA (1) | CA3029772A1 (ru) |
| IL (2) | IL301091A (ru) |
| MX (2) | MX2018016253A (ru) |
| RU (2) | RU2769249C2 (ru) |
| WO (1) | WO2018007475A1 (ru) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| CN108137492B (zh) | 2015-10-09 | 2021-12-21 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| JP7074345B2 (ja) | 2016-06-22 | 2022-05-24 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | 一本鎖ｒｎａ編集オリゴヌクレオチド |
| WO2018039629A2 (en) | 2016-08-25 | 2018-03-01 | Northwestern University | Micellar spherical nucleic acids from thermoresponsive, traceless templates |
| CA3035293A1 (en) | 2016-09-01 | 2018-03-08 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| EP3694530A4 (en) * | 2017-10-12 | 2021-06-30 | Wave Life Sciences Ltd. | COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES |
| US11866689B2 (en) | 2018-03-26 | 2024-01-09 | Duke University | Splice-switching oligonucleotides and methods of use |
| TWI844541B (zh) * | 2018-05-11 | 2024-06-11 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組成物及其使用方法 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CN112955153A (zh) | 2018-08-02 | 2021-06-11 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗肌养蛋白病的用途 |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| CN112912499A (zh) | 2018-08-02 | 2021-06-04 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 |
| SG11202103938UA (en) | 2018-11-02 | 2021-05-28 | Biomarin Tech Bv | Bispecific antisense oligonucleotides for dystrophin exon skipping |
| JP2022513719A (ja) * | 2018-12-06 | 2022-02-09 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物及びその方法 |
| CN113994000B (zh) * | 2019-06-13 | 2025-03-25 | ProQR治疗上市公司Ⅱ | 包括胞苷类似物的反义rna编辑寡核苷酸 |
| US20220348917A1 (en) * | 2019-09-04 | 2022-11-03 | Exicure Operating Company | Liposomal spherical nucleic acid (sna) constructs for splice modulation |
| RU2738093C9 (ru) * | 2019-12-23 | 2020-12-29 | Общество с ограниченной ответственностью "Гелеспон" | Способ синтеза 2'-о-(2-метоксиэтил) тиофосфатного олигонуклеотида |
| JP7765837B2 (ja) | 2019-12-23 | 2025-11-07 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | シュタルガルト病の処置におけるヌクレオチド脱アミノ化のためのアンチセンスオリゴヌクレオチド |
| AR121446A1 (es) | 2020-02-28 | 2022-06-08 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular smn2 |
| US20220098586A1 (en) | 2020-09-30 | 2022-03-31 | Biomarin Technologies B.V. | Oligonucleotide |
| WO2022232478A1 (en) | 2021-04-30 | 2022-11-03 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| IL309936A (en) | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of dystrophinopathy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| IL315280A (en) | 2022-03-17 | 2024-10-01 | Sarepta Therapeutics Inc | Phosphorodiamidate morpholino oligomer conjugates |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| CN115806984B (zh) * | 2022-10-18 | 2023-10-10 | 昆明理工大学 | 环状rna及载体和载体的应用 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| TW202435897A (zh) | 2022-11-24 | 2024-09-16 | 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ | 用於治療遺傳性ｈｆｅ血色沉著病之反義寡核苷酸 |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| WO2024121373A1 (en) | 2022-12-09 | 2024-06-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of cardiovascular disease |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| WO2024175550A1 (en) | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| WO2024206175A1 (en) | 2023-03-24 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| AU2024246572A1 (en) | 2023-03-27 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| WO2024256620A1 (en) | 2023-06-16 | 2024-12-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurodegenerative disease |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2025056035A1 (en) * | 2023-09-13 | 2025-03-20 | Edigene Therapeutics (Beijing) Inc. | Compositions and methods for treating duchenne muscular dystrophy |
| WO2025104239A1 (en) | 2023-11-16 | 2025-05-22 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of classic galactosemia |
| WO2025132708A1 (en) | 2023-12-20 | 2025-06-26 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of huntington's disease |
| GB202404661D0 (en) | 2024-04-02 | 2024-05-15 | Proqr Therapeutics Ii Bv | Antisense oligoncleotides for the treatment of liver disease |
| GB202405143D0 (en) | 2024-04-11 | 2024-05-29 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of poly-q disease |
| WO2025224230A1 (en) | 2024-04-25 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of fatty liver disease |
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2017
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| KR20230175313A (ko) | 2023-12-29 |
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| IL264084A (en) | 2019-01-31 |
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| CN109757108A (zh) | 2019-05-14 |
| WO2018007475A1 (en) | 2018-01-11 |
| JP2019522972A (ja) | 2019-08-22 |
| BR112019000061A2 (pt) | 2019-11-26 |
| CA3029772A1 (en) | 2018-01-11 |
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