MX2019014412A - Productos terapeuticos para la enfermedad de almacenamiento de glucogeno de tipo iii. - Google Patents
Productos terapeuticos para la enfermedad de almacenamiento de glucogeno de tipo iii.Info
- Publication number
- MX2019014412A MX2019014412A MX2019014412A MX2019014412A MX2019014412A MX 2019014412 A MX2019014412 A MX 2019014412A MX 2019014412 A MX2019014412 A MX 2019014412A MX 2019014412 A MX2019014412 A MX 2019014412A MX 2019014412 A MX2019014412 A MX 2019014412A
- Authority
- MX
- Mexico
- Prior art keywords
- agl
- alpha
- glucogen
- type iii
- storage disease
- Prior art date
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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1048—Glycosyltransferases (2.4)
- C12N9/1051—Hexosyltransferases (2.4.1)
-
- 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
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/45—Transferases (2)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2405—Glucanases
- C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
-
- 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
-
- 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
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
-
- 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
- C12N2310/335—Modified T or U
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/01—Hexosyltransferases (2.4.1)
- C12Y204/01025—4-Alpha-glucanotransferase (2.4.1.25)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01033—Amylo-alpha-1,6-glucosidase (3.2.1.33)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762513350P | 2017-05-31 | 2017-05-31 | |
| PCT/US2018/035477 WO2018222926A1 (en) | 2017-05-31 | 2018-05-31 | Therapeutics for glycogen storage disease type iii |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2019014412A true MX2019014412A (es) | 2020-02-10 |
Family
ID=64456114
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2019014412A MX2019014412A (es) | 2017-05-31 | 2018-05-31 | Productos terapeuticos para la enfermedad de almacenamiento de glucogeno de tipo iii. |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US11377643B2 (enExample) |
| EP (1) | EP3630964B1 (enExample) |
| JP (2) | JP7284101B2 (enExample) |
| KR (1) | KR102636537B1 (enExample) |
| CN (1) | CN110719954B (enExample) |
| AR (1) | AR112706A1 (enExample) |
| AU (1) | AU2018278315B2 (enExample) |
| BR (1) | BR112019025224A2 (enExample) |
| CO (1) | CO2019013332A2 (enExample) |
| ES (1) | ES3048960T3 (enExample) |
| MX (1) | MX2019014412A (enExample) |
| TW (1) | TWI794237B (enExample) |
| WO (1) | WO2018222926A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2923757T3 (es) | 2011-12-16 | 2022-09-30 | Modernatx Inc | Composiciones de ARNm modificado |
| US10383952B2 (en) | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| KR102636537B1 (ko) | 2017-05-31 | 2024-02-15 | 울트라제닉스 파마수티컬 인코포레이티드 | 글리코겐 축적 질환 유형 iii에 대한 치료제 |
| CA3122080A1 (en) | 2018-12-06 | 2020-06-11 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| CN110314407A (zh) * | 2019-08-01 | 2019-10-11 | 山东新希望六和集团有限公司 | 一种脂肪快速提取装置 |
| WO2021067598A1 (en) | 2019-10-04 | 2021-04-08 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| IT202000003371A1 (it) * | 2020-02-19 | 2021-08-19 | Enea Agenzia Naz Per Le Nuove Tecnologie Lenergia E Lo Sviluppo Economico Sostenibile | Composto per il trattamento di una glicogenosi |
| EP4114470A4 (en) | 2020-03-03 | 2024-04-17 | Arcturus Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY |
| JP2023517644A (ja) | 2020-03-09 | 2023-04-26 | アークトゥラス・セラピューティクス・インコーポレイテッド | コロナウイルスワクチン組成物及び方法 |
| MX2023001109A (es) | 2020-07-27 | 2023-04-24 | Anjarium Biosciences Ag | Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas. |
| US12178921B2 (en) | 2020-08-14 | 2024-12-31 | Arcturus Therapeutics, Inc. | Method of lyophilizing lipid nanoparticles |
| JP2024517427A (ja) * | 2021-04-20 | 2024-04-22 | アンジャリウム バイオサイエンシズ エージー | アミロ－α－１，６－グルコシダーゼ、４－α－グルカノトランスフェラーゼをコードするＤＮＡ分子の組成物、その作製の方法、及びその使用の方法 |
| CN118369335A (zh) * | 2022-01-06 | 2024-07-19 | 上海吉量医药工程有限公司 | N1位修饰假尿嘧啶核苷及其在mRNA合成中的应用 |
| CN115960926A (zh) * | 2022-12-30 | 2023-04-14 | 南方科技大学 | 一种mRNA及含有mRNA的药物 |
| EP4791878A1 (en) | 2023-10-13 | 2026-08-19 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations |
| AU2024397279A1 (en) | 2023-12-13 | 2026-07-02 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating conditions associated with ube3a overexpression |
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| ATE291925T1 (de) | 2001-06-05 | 2005-04-15 | Curevac Gmbh | Stabilisierte mrna mit erhöhtem g/c-gehalt und optimierter codon usage für die gentherapie |
| JP2006508939A (ja) | 2002-10-29 | 2006-03-16 | エンジーン， インコーポレイテッド | 癌処置のための組成物 |
| US8160205B2 (en) | 2004-04-06 | 2012-04-17 | Accuray Incorporated | Robotic arm for patient positioning assembly |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| EP2049665A2 (en) | 2006-07-28 | 2009-04-22 | Applera Corporation | Dinucleotide mrna cap analogs |
| WO2009058911A2 (en) | 2007-10-31 | 2009-05-07 | Applied Biosystems Inc. | Preparation and isolation of 5' capped mrna |
| US9006191B2 (en) | 2007-12-27 | 2015-04-14 | Protiva Biotherapeutics, Inc. | Silencing of polo-like kinase expression using interfering RNA |
| EP3100718B1 (en) | 2008-01-02 | 2019-11-27 | Arbutus Biopharma Corporation | Improved compositions and methods for the delivery of nucleic acids |
| EP2279254B1 (en) | 2008-04-15 | 2017-07-05 | Protiva Biotherapeutics Inc. | Novel lipid formulations for nucleic acid delivery |
| US9050333B2 (en) | 2008-07-08 | 2015-06-09 | Duke University | Method of treating glycogen storage disease type III with human acid α-glucosidase |
| WO2010037408A1 (en) | 2008-09-30 | 2010-04-08 | Curevac Gmbh | Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof |
| AU2009303345B2 (en) | 2008-10-09 | 2015-08-20 | Arbutus Biopharma Corporation | Improved amino lipids and methods for the delivery of nucleic acids |
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| EP3699172A3 (en) | 2008-11-10 | 2020-11-18 | Arbutus Biopharma Corporation | Novel lipids and compositions for the delivery of therapeutics |
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| CA3042927C (en) | 2009-05-05 | 2022-05-17 | Arbutus Biopharma Corporation | Lipid compositions for the delivery of therapeutic agents |
| KR20220038506A (ko) | 2009-06-10 | 2022-03-28 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| SI2506857T1 (en) | 2009-12-01 | 2018-08-31 | Translate Bio, Inc. | Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases |
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| KR20220025112A (ko) | 2011-06-08 | 2022-03-03 | 샤이어 휴먼 지네틱 테라피즈 인크. | Mrna 전달을 위한 지질 나노입자 조성물 및 방법 |
| PT3682905T (pt) | 2011-10-03 | 2022-04-07 | Modernatx Inc | Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações |
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| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
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| EP3154570A4 (en) | 2014-06-13 | 2017-12-27 | Valerion Therapeutics, LLC | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
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| IL264439B2 (en) | 2016-08-17 | 2024-08-01 | Factor Bioscience Inc | non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment |
| KR20190093816A (ko) | 2016-10-26 | 2019-08-26 | 큐어백 아게 | 지질 나노입자 mRNA 백신 |
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| US10383952B2 (en) | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| US10227302B2 (en) | 2017-02-09 | 2019-03-12 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| KR102636537B1 (ko) * | 2017-05-31 | 2024-02-15 | 울트라제닉스 파마수티컬 인코포레이티드 | 글리코겐 축적 질환 유형 iii에 대한 치료제 |
-
2018
- 2018-05-31 KR KR1020197036416A patent/KR102636537B1/ko active Active
- 2018-05-31 WO PCT/US2018/035477 patent/WO2018222926A1/en not_active Ceased
- 2018-05-31 US US16/617,431 patent/US11377643B2/en active Active
- 2018-05-31 EP EP18810235.4A patent/EP3630964B1/en active Active
- 2018-05-31 MX MX2019014412A patent/MX2019014412A/es unknown
- 2018-05-31 CN CN201880036551.XA patent/CN110719954B/zh active Active
- 2018-05-31 JP JP2019565348A patent/JP7284101B2/ja active Active
- 2018-05-31 BR BR112019025224-0A patent/BR112019025224A2/pt unknown
- 2018-05-31 ES ES18810235T patent/ES3048960T3/es active Active
- 2018-05-31 TW TW107118693A patent/TWI794237B/zh active
- 2018-05-31 AU AU2018278315A patent/AU2018278315B2/en active Active
- 2018-05-31 AR ARP180101448A patent/AR112706A1/es unknown
-
2019
- 2019-11-27 CO CONC2019/0013332A patent/CO2019013332A2/es unknown
-
2022
- 2022-05-04 US US17/736,798 patent/US12703858B2/en active Active
-
2023
- 2023-03-13 JP JP2023038257A patent/JP2023075248A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AR112706A1 (es) | 2019-12-04 |
| EP3630964A1 (en) | 2020-04-08 |
| CN110719954B (zh) | 2023-12-26 |
| EP3630964A4 (en) | 2021-03-03 |
| JP7284101B2 (ja) | 2023-05-30 |
| AU2018278315B2 (en) | 2024-01-18 |
| JP2023075248A (ja) | 2023-05-30 |
| CO2019013332A2 (es) | 2020-02-18 |
| KR20200014319A (ko) | 2020-02-10 |
| EP3630964C0 (en) | 2025-08-13 |
| US20200149017A1 (en) | 2020-05-14 |
| BR112019025224A2 (pt) | 2020-12-08 |
| AU2018278315A1 (en) | 2019-12-12 |
| US20220340886A1 (en) | 2022-10-27 |
| EP3630964B1 (en) | 2025-08-13 |
| JP2020522244A (ja) | 2020-07-30 |
| CN110719954A (zh) | 2020-01-21 |
| US11377643B2 (en) | 2022-07-05 |
| US12703858B2 (en) | 2026-08-11 |
| TWI794237B (zh) | 2023-03-01 |
| ES3048960T3 (en) | 2025-12-12 |
| KR102636537B1 (ko) | 2024-02-15 |
| TW201903151A (zh) | 2019-01-16 |
| CA3063907A1 (en) | 2018-12-06 |
| WO2018222926A1 (en) | 2018-12-06 |
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