SA517381912B1 - "عناصر تنظيمية معدلة بعد الانتساخ لعلاج الالتهاب الكبدي pre - Google Patents
"عناصر تنظيمية معدلة بعد الانتساخ لعلاج الالتهاب الكبدي preInfo
- Publication number
- SA517381912B1 SA517381912B1 SA517381912A SA517381912A SA517381912B1 SA 517381912 B1 SA517381912 B1 SA 517381912B1 SA 517381912 A SA517381912 A SA 517381912A SA 517381912 A SA517381912 A SA 517381912A SA 517381912 B1 SA517381912 B1 SA 517381912B1
- Authority
- SA
- Saudi Arabia
- Prior art keywords
- gene
- regulatory elements
- transcriptional regulatory
- modified hepatitis
- modified pre
- Prior art date
Links
- 208000006454 hepatitis Diseases 0.000 title abstract 3
- 231100000283 hepatitis Toxicity 0.000 title abstract 3
- 230000001124 posttranscriptional effect Effects 0.000 title 1
- 230000001105 regulatory effect Effects 0.000 title 1
- 101150003160 X gene Proteins 0.000 abstract 4
- 108091033319 polynucleotide Proteins 0.000 abstract 3
- 102000040430 polynucleotide Human genes 0.000 abstract 3
- 239000002157 polynucleotide Substances 0.000 abstract 3
- 108020004705 Codon Proteins 0.000 abstract 1
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 abstract 1
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 abstract 1
- 230000015556 catabolic process Effects 0.000 abstract 1
- 238000006731 degradation reaction Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/14—Blood; Artificial blood
- A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/15—Natural-killer [NK] cells; Natural-killer T [NKT] cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
- A61K40/31—Chimeric antigen receptors [CAR]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
- A61K40/41—Vertebrate antigens
- A61K40/42—Cancer antigens
- A61K40/4202—Receptors, cell surface antigens or cell surface determinants
- A61K40/421—Immunoglobulin superfamily
- A61K40/4211—CD19 or B4
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
-
- 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
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N2730/00—Reverse transcribing DNA viruses
- C12N2730/00011—Details
- C12N2730/10011—Hepadnaviridae
- C12N2730/10041—Use of virus, viral particle or viral elements as a vector
- C12N2730/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Immunology (AREA)
- Virology (AREA)
- Molecular Biology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Cell Biology (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Developmental Biology & Embryology (AREA)
- Hematology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562102569P | 2015-01-12 | 2015-01-12 | |
| PCT/US2016/013109 WO2016115177A1 (en) | 2015-01-12 | 2016-01-12 | Modified hepatitis post-transcriptional regulatory elements |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SA517381912B1 true SA517381912B1 (ar) | 2021-12-30 |
Family
ID=55310922
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SA517381912A SA517381912B1 (ar) | 2015-01-12 | 2017-07-12 | "عناصر تنظيمية معدلة بعد الانتساخ لعلاج الالتهاب الكبدي pre |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US10363269B2 (enExample) |
| EP (1) | EP3245292B1 (enExample) |
| JP (1) | JP6868560B2 (enExample) |
| KR (1) | KR102554374B1 (enExample) |
| CN (1) | CN107429260B (enExample) |
| AU (1) | AU2016206868B2 (enExample) |
| BR (1) | BR112017014917A2 (enExample) |
| CA (1) | CA2973483A1 (enExample) |
| ES (1) | ES2877367T3 (enExample) |
| IL (1) | IL253399B2 (enExample) |
| MA (1) | MA41346A (enExample) |
| MX (1) | MX391694B (enExample) |
| MY (1) | MY187559A (enExample) |
| RU (1) | RU2734594C2 (enExample) |
| SA (1) | SA517381912B1 (enExample) |
| SG (1) | SG11201705692SA (enExample) |
| WO (1) | WO2016115177A1 (enExample) |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA40318A (fr) | 2014-11-05 | 2017-09-13 | Juno Therapeutics Inc | Procédés de transduction et de traitement de cellules |
| EP3331905B1 (en) | 2015-08-06 | 2022-10-05 | Dana-Farber Cancer Institute, Inc. | Targeted protein degradation to attenuate adoptive t-cell therapy associated adverse inflammatory responses |
| AU2017254665C1 (en) | 2016-04-20 | 2021-03-11 | Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. | Compositions and methods for enhanced gene expression of PKLR |
| AU2017345430B2 (en) | 2016-10-20 | 2024-09-05 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of Fabry disease |
| CN107058315B (zh) * | 2016-12-08 | 2019-11-08 | 上海优卡迪生物医药科技有限公司 | 敲减人PD-1的siRNA、重组表达CAR-T载体及其构建方法和应用 |
| JP7227912B2 (ja) * | 2017-02-08 | 2023-02-24 | ダナ－ファーバー キャンサー インスティテュート，インコーポレイテッド | キメラ抗原受容体の調節 |
| US11713468B2 (en) * | 2017-08-02 | 2023-08-01 | Dna Twopointo Inc. | DNA vectors and elements for sustained gene expression in eukaryotic cells |
| JP7654240B2 (ja) | 2017-09-19 | 2025-04-01 | マサチューセッツ インスティテュート オブ テクノロジー | キメラ抗原受容体ｔ細胞治療のための組成物およびその使用 |
| BR112020021903A2 (pt) | 2018-04-27 | 2021-03-02 | Rocket Pharmaceuticals, Ltd. | terapia genética para degeneração do snc |
| AU2019346335B2 (en) | 2018-09-28 | 2024-07-25 | Massachusetts Institute Of Technology | Collagen-localized immunomodulatory molecules and methods thereof |
| KR20210152515A (ko) | 2019-04-12 | 2021-12-15 | 씨4 테라퓨틱스, 인코포레이티드 | 이카로스 및 아이올로스의 트리시클릭 분해제 |
| CN111825769B (zh) * | 2019-04-16 | 2022-03-25 | 上海科技大学 | 一种泛素化缺失的嵌合抗原受体及其用途 |
| JP2022536692A (ja) | 2019-06-11 | 2022-08-18 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | 調節不全の血漿カリクレインによって媒介される疾患の治療のための抗体のアデノ随伴ウイルスベクター送達 |
| EP3990491A1 (en) | 2019-06-26 | 2022-05-04 | Massachusetts Institute of Technology | Immunomodulatory fusion protein-metal hydroxide complexes and methods thereof |
| WO2021061648A1 (en) | 2019-09-23 | 2021-04-01 | Massachusetts Institute Of Technology | Methods and compositions for stimulation of endogenous t cell responses |
| EP4048800A1 (en) | 2019-10-23 | 2022-08-31 | Shire Human Genetic Therapies, Inc. | Adeno-associated virus vectors based gene therapy for hereditary angioedema |
| CA3173527A1 (en) | 2020-03-10 | 2021-09-16 | Massachusetts Institute Of Technology | Methods for generating engineered memory-like nk cells and compositions thereof |
| JP2023517889A (ja) | 2020-03-10 | 2023-04-27 | マサチューセッツ インスティテュート オブ テクノロジー | ＮＰＭ１ｃ陽性がんの免疫療法のための組成物および方法 |
| DE102020111571A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| DE102020106710A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| CA3181339A1 (en) | 2020-04-27 | 2021-11-04 | Juno Therapeutics, Inc. | Stabilization of polyethyleneimine-deoxyribonucleic acid complex size and activity |
| US12433954B2 (en) | 2020-05-01 | 2025-10-07 | Massachusetts Institute Of Technology | Methods of activating anti-CD19 chimeric antigen receptor (CAR) T cells using amphiphilic ligand conjugates comprising CAR-targeting protein sequence motifs |
| US20210340524A1 (en) | 2020-05-01 | 2021-11-04 | Massachusetts Institute Of Technology | Methods for identifying chimeric antigen receptor-targeting ligands and uses thereof |
| US20230323390A1 (en) * | 2020-06-11 | 2023-10-12 | Sangamo Therapeutics, Inc. | Methods and compositions for expressing phenylalanine hydroxylase |
| EP4172330A1 (en) * | 2020-06-30 | 2023-05-03 | The University Court Of The University of Edinburgh | Transgene expression system |
| CN116194154A (zh) | 2020-08-07 | 2023-05-30 | 太空飞船七有限责任公司 | 使用aav载体的plakophilin-2(pkp2)基因疗法 |
| KR20220035693A (ko) * | 2020-09-14 | 2022-03-22 | 주식회사 제너로스 | 표적화된 유전자 전달을 위한 아데노-부속 바이러스 벡터 |
| IL301747A (en) * | 2020-09-29 | 2023-05-01 | Neuexcell Therapeutics Inc | Dlx2 vector |
| CN116829595A (zh) | 2020-12-16 | 2023-09-29 | 武田药品工业株式会社 | 用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送 |
| US20230049217A1 (en) * | 2021-05-21 | 2023-02-16 | Novartis Ag | Compositions and methods for enhancing visual function |
| WO2023081715A1 (en) | 2021-11-03 | 2023-05-11 | Viracta Therapeutics, Inc. | Combination of car t-cell therapy with btk inhibitors and methods of use thereof |
| CA3236331A1 (en) * | 2021-11-04 | 2023-05-11 | Mayo Foundation For Medical Education And Research | Methods and materials for treating cancer |
| KR20250033266A (ko) | 2022-07-07 | 2025-03-07 | 사이언스 코포레이션 | 신경 인터페이스 디바이스 |
| EP4598572A1 (en) * | 2022-10-04 | 2025-08-13 | Emergent Product Development Gaithersburg Inc. | Novel vsv virus formulations |
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-
2016
- 2016-01-11 MA MA041346A patent/MA41346A/fr unknown
- 2016-01-12 KR KR1020177022738A patent/KR102554374B1/ko active Active
- 2016-01-12 RU RU2017126975A patent/RU2734594C2/ru active
- 2016-01-12 MX MX2017009113A patent/MX391694B/es unknown
- 2016-01-12 AU AU2016206868A patent/AU2016206868B2/en active Active
- 2016-01-12 BR BR112017014917A patent/BR112017014917A2/pt not_active Application Discontinuation
- 2016-01-12 EP EP16703392.7A patent/EP3245292B1/en active Active
- 2016-01-12 SG SG11201705692SA patent/SG11201705692SA/en unknown
- 2016-01-12 US US14/994,114 patent/US10363269B2/en active Active
- 2016-01-12 CN CN201680015106.6A patent/CN107429260B/zh active Active
- 2016-01-12 MY MYPI2017001032A patent/MY187559A/en unknown
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2017
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- 2017-07-12 SA SA517381912A patent/SA517381912B1/ar unknown
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| Publication number | Publication date |
|---|---|
| EP3245292A1 (en) | 2017-11-22 |
| IL253399A0 (en) | 2017-09-28 |
| RU2017126975A (ru) | 2019-02-20 |
| US10363269B2 (en) | 2019-07-30 |
| MY187559A (en) | 2021-09-29 |
| RU2734594C2 (ru) | 2020-10-20 |
| AU2016206868B2 (en) | 2022-03-17 |
| SG11201705692SA (en) | 2017-08-30 |
| HK1246347A1 (en) | 2018-09-07 |
| CN107429260A (zh) | 2017-12-01 |
| RU2017126975A3 (enExample) | 2019-09-04 |
| BR112017014917A2 (pt) | 2018-03-13 |
| ES2877367T3 (es) | 2021-11-16 |
| KR102554374B1 (ko) | 2023-07-11 |
| KR20170131362A (ko) | 2017-11-29 |
| US20160199412A1 (en) | 2016-07-14 |
| MX391694B (es) | 2025-03-21 |
| JP6868560B2 (ja) | 2021-05-12 |
| IL253399B2 (en) | 2023-06-01 |
| MA41346A (fr) | 2017-11-21 |
| EP3245292B1 (en) | 2021-06-09 |
| WO2016115177A1 (en) | 2016-07-21 |
| CA2973483A1 (en) | 2016-07-21 |
| JP2018502583A (ja) | 2018-02-01 |
| AU2016206868A1 (en) | 2017-07-27 |
| CN107429260B (zh) | 2021-12-31 |
| MX2017009113A (es) | 2017-11-15 |
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