JP2018526015A5 - - Google Patents
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- JP2018526015A5 JP2018526015A5 JP2018512546A JP2018512546A JP2018526015A5 JP 2018526015 A5 JP2018526015 A5 JP 2018526015A5 JP 2018512546 A JP2018512546 A JP 2018512546A JP 2018512546 A JP2018512546 A JP 2018512546A JP 2018526015 A5 JP2018526015 A5 JP 2018526015A5
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- 230000009261 transgenic effect Effects 0.000 claims description 104
- 238000012239 gene modification Methods 0.000 claims description 73
- 230000005017 genetic modification Effects 0.000 claims description 73
- 235000013617 genetically modified food Nutrition 0.000 claims description 73
- 238000000034 method Methods 0.000 claims description 71
- 108090000623 proteins and genes Proteins 0.000 claims description 57
- 108700019146 Transgenes Proteins 0.000 claims description 39
- 239000012634 fragment Substances 0.000 claims description 29
- 101000856513 Homo sapiens Inactive N-acetyllactosaminide alpha-1,3-galactosyltransferase Proteins 0.000 claims description 25
- 102100025509 Inactive N-acetyllactosaminide alpha-1,3-galactosyltransferase Human genes 0.000 claims description 25
- 238000003780 insertion Methods 0.000 claims description 24
- 230000037431 insertion Effects 0.000 claims description 24
- 230000010354 integration Effects 0.000 claims description 22
- 108010047303 von Willebrand Factor Proteins 0.000 claims description 21
- 238000006467 substitution reaction Methods 0.000 claims description 15
- 210000004072 lung Anatomy 0.000 claims description 14
- 238000012217 deletion Methods 0.000 claims description 13
- 230000037430 deletion Effects 0.000 claims description 13
- 239000002773 nucleotide Substances 0.000 claims description 13
- 125000003729 nucleotide group Chemical group 0.000 claims description 13
- 108020004414 DNA Proteins 0.000 claims description 12
- 239000002955 immunomodulating agent Substances 0.000 claims description 12
- 229940121354 immunomodulator Drugs 0.000 claims description 12
- 108060003306 Galactosyltransferase Proteins 0.000 claims description 11
- 101150045640 VWF gene Proteins 0.000 claims description 11
- 230000002584 immunomodulator Effects 0.000 claims description 11
- 210000000056 organ Anatomy 0.000 claims description 11
- 238000003209 gene knockout Methods 0.000 claims description 9
- 230000035772 mutation Effects 0.000 claims description 9
- 101100156611 Homo sapiens VWF gene Proteins 0.000 claims description 8
- 102100034343 Integrase Human genes 0.000 claims description 8
- 108010061833 Integrases Proteins 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 8
- 239000004074 complement inhibitor Substances 0.000 claims description 8
- 230000001120 cytoprotective effect Effects 0.000 claims description 8
- 230000004048 modification Effects 0.000 claims description 8
- 238000012986 modification Methods 0.000 claims description 8
- 102000030902 Galactosyltransferase Human genes 0.000 claims description 7
- 101000902205 Homo sapiens Inactive cytidine monophosphate-N-acetylneuraminic acid hydroxylase Proteins 0.000 claims description 7
- 102100022247 Inactive cytidine monophosphate-N-acetylneuraminic acid hydroxylase Human genes 0.000 claims description 7
- -1 ROSA26 Proteins 0.000 claims description 7
- 229940124073 Complement inhibitor Drugs 0.000 claims description 6
- 101000961414 Homo sapiens Membrane cofactor protein Proteins 0.000 claims description 6
- 230000002776 aggregation Effects 0.000 claims description 6
- 238000004220 aggregation Methods 0.000 claims description 6
- 238000004519 manufacturing process Methods 0.000 claims description 6
- 230000001404 mediated effect Effects 0.000 claims description 6
- 238000000520 microinjection Methods 0.000 claims description 6
- 238000010374 somatic cell nuclear transfer Methods 0.000 claims description 6
- 230000002269 spontaneous effect Effects 0.000 claims description 6
- 238000001890 transfection Methods 0.000 claims description 6
- 101001000998 Homo sapiens Protein phosphatase 1 regulatory subunit 12C Proteins 0.000 claims description 5
- 102100035620 Protein phosphatase 1 regulatory subunit 12C Human genes 0.000 claims description 5
- 238000010348 incorporation Methods 0.000 claims description 5
- 239000003550 marker Substances 0.000 claims description 5
- 101150113197 CMAH gene Proteins 0.000 claims description 4
- 201000003883 Cystic fibrosis Diseases 0.000 claims description 4
- 102100031780 Endonuclease Human genes 0.000 claims description 4
- 108010042407 Endonucleases Proteins 0.000 claims description 4
- 102000004190 Enzymes Human genes 0.000 claims description 4
- 108090000790 Enzymes Proteins 0.000 claims description 4
- 102100028970 HLA class I histocompatibility antigen, alpha chain E Human genes 0.000 claims description 4
- 101000986085 Homo sapiens HLA class I histocompatibility antigen, alpha chain E Proteins 0.000 claims description 4
- 208000019693 Lung disease Diseases 0.000 claims description 4
- 102100039373 Membrane cofactor protein Human genes 0.000 claims description 4
- 241000282887 Suidae Species 0.000 claims description 4
- 239000003146 anticoagulant agent Substances 0.000 claims description 4
- 229940127219 anticoagulant drug Drugs 0.000 claims description 4
- 239000003814 drug Substances 0.000 claims description 4
- 238000010362 genome editing Methods 0.000 claims description 4
- 210000002216 heart Anatomy 0.000 claims description 4
- 230000001506 immunosuppresive effect Effects 0.000 claims description 4
- 210000003734 kidney Anatomy 0.000 claims description 4
- 210000000496 pancreas Anatomy 0.000 claims description 4
- 108091033319 polynucleotide Proteins 0.000 claims description 4
- 239000002157 polynucleotide Substances 0.000 claims description 4
- 102000040430 polynucleotide Human genes 0.000 claims description 4
- 230000000750 progressive effect Effects 0.000 claims description 4
- 229940124597 therapeutic agent Drugs 0.000 claims description 4
- 241001465754 Metazoa Species 0.000 claims description 3
- 108010052160 Site-specific recombinase Proteins 0.000 claims description 3
- 230000002779 inactivation Effects 0.000 claims description 3
- 230000000392 somatic effect Effects 0.000 claims description 3
- 102100022002 CD59 glycoprotein Human genes 0.000 claims description 2
- 108010013423 CMPacetylneuraminate monooxygenase Proteins 0.000 claims description 2
- 108091033409 CRISPR Proteins 0.000 claims description 2
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 claims description 2
- 102100025680 Complement decay-accelerating factor Human genes 0.000 claims description 2
- 108010051219 Cre recombinase Proteins 0.000 claims description 2
- 102100029722 Ectonucleoside triphosphate diphosphohydrolase 1 Human genes 0.000 claims description 2
- 102000009839 Endothelial Protein C Receptor Human genes 0.000 claims description 2
- 108010009900 Endothelial Protein C Receptor Proteins 0.000 claims description 2
- 108010046276 FLP recombinase Proteins 0.000 claims description 2
- 208000004248 Familial Primary Pulmonary Hypertension Diseases 0.000 claims description 2
- 101150042360 GGTA1 gene Proteins 0.000 claims description 2
- 108010091714 Globoside alpha-N-acetylgalactosaminyltransferase Proteins 0.000 claims description 2
- 101000897400 Homo sapiens CD59 glycoprotein Proteins 0.000 claims description 2
- 101000856022 Homo sapiens Complement decay-accelerating factor Proteins 0.000 claims description 2
- 101001012447 Homo sapiens Ectonucleoside triphosphate diphosphohydrolase 1 Proteins 0.000 claims description 2
- 101000868279 Homo sapiens Leukocyte surface antigen CD47 Proteins 0.000 claims description 2
- 101000972282 Homo sapiens Mucin-5AC Proteins 0.000 claims description 2
- 201000009794 Idiopathic Pulmonary Fibrosis Diseases 0.000 claims description 2
- 102100037872 Intercellular adhesion molecule 2 Human genes 0.000 claims description 2
- 101710148794 Intercellular adhesion molecule 2 Proteins 0.000 claims description 2
- 102100032913 Leukocyte surface antigen CD47 Human genes 0.000 claims description 2
- 102100022496 Mucin-5AC Human genes 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims description 2
- 206010064911 Pulmonary arterial hypertension Diseases 0.000 claims description 2
- 238000010459 TALEN Methods 0.000 claims description 2
- 102100030951 Tissue factor pathway inhibitor Human genes 0.000 claims description 2
- 108010010574 Tn3 resolvase Proteins 0.000 claims description 2
- 241000700605 Viruses Species 0.000 claims description 2
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 2
- 102000015395 alpha 1-Antitrypsin Human genes 0.000 claims description 2
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims description 2
- 229940024142 alpha 1-antitrypsin Drugs 0.000 claims description 2
- 239000003242 anti bacterial agent Substances 0.000 claims description 2
- 239000002260 anti-inflammatory agent Substances 0.000 claims description 2
- 229940121363 anti-inflammatory agent Drugs 0.000 claims description 2
- 230000003409 anti-rejection Effects 0.000 claims description 2
- 239000003443 antiviral agent Substances 0.000 claims description 2
- 210000002459 blastocyst Anatomy 0.000 claims description 2
- 239000003795 chemical substances by application Substances 0.000 claims description 2
- 230000001086 cytosolic effect Effects 0.000 claims description 2
- 201000010099 disease Diseases 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 2
- 230000008030 elimination Effects 0.000 claims description 2
- 238000003379 elimination reaction Methods 0.000 claims description 2
- 210000002889 endothelial cell Anatomy 0.000 claims description 2
- 108010089843 gamma delta resolvase Proteins 0.000 claims description 2
- 102000044446 human CD46 Human genes 0.000 claims description 2
- 239000003018 immunosuppressive agent Substances 0.000 claims description 2
- 229940125721 immunosuppressive agent Drugs 0.000 claims description 2
- 230000001939 inductive effect Effects 0.000 claims description 2
- 208000036971 interstitial lung disease 2 Diseases 0.000 claims description 2
- 108010013555 lipoprotein-associated coagulation inhibitor Proteins 0.000 claims description 2
- 210000004185 liver Anatomy 0.000 claims description 2
- 230000013011 mating Effects 0.000 claims description 2
- 201000008312 primary pulmonary hypertension Diseases 0.000 claims description 2
- 230000009467 reduction Effects 0.000 claims description 2
- 239000000126 substance Substances 0.000 claims description 2
- 238000010361 transduction Methods 0.000 claims description 2
- 230000026683 transduction Effects 0.000 claims description 2
- 230000009466 transformation Effects 0.000 claims description 2
- 108700026220 vif Genes Proteins 0.000 claims description 2
- 230000010100 anticoagulation Effects 0.000 claims 2
- 108010091086 Recombinases Proteins 0.000 claims 1
- 102000018120 Recombinases Human genes 0.000 claims 1
- 230000001850 reproductive effect Effects 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- 239000003130 blood coagulation factor inhibitor Substances 0.000 description 2
- 210000004602 germ cell Anatomy 0.000 description 1
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021068874A JP2021101741A (ja) | 2015-09-09 | 2021-04-15 | 異種移植のための多トランスジェニックブタ |
| JP2023113055A JP2023119041A (ja) | 2015-09-09 | 2023-07-10 | 異種移植のための多トランスジェニックブタ |
| JP2025052613A JP2025092572A (ja) | 2015-09-09 | 2025-03-26 | 異種移植のための多トランスジェニックブタ |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562216225P | 2015-09-09 | 2015-09-09 | |
| US62/216,225 | 2015-09-09 | ||
| US201562256068P | 2015-11-16 | 2015-11-16 | |
| US62/256,068 | 2015-11-16 | ||
| PCT/US2016/051126 WO2017044864A1 (en) | 2015-09-09 | 2016-09-09 | Multi-transgenic pig for xenotransplantation |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021068874A Division JP2021101741A (ja) | 2015-09-09 | 2021-04-15 | 異種移植のための多トランスジェニックブタ |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018526015A JP2018526015A (ja) | 2018-09-13 |
| JP2018526015A5 true JP2018526015A5 (https=) | 2019-10-24 |
| JP7050665B2 JP7050665B2 (ja) | 2022-04-08 |
Family
ID=58240236
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018512546A Active JP7050665B2 (ja) | 2015-09-09 | 2016-09-09 | 異種移植のための多トランスジェニックブタ |
| JP2021068874A Pending JP2021101741A (ja) | 2015-09-09 | 2021-04-15 | 異種移植のための多トランスジェニックブタ |
| JP2023113055A Withdrawn JP2023119041A (ja) | 2015-09-09 | 2023-07-10 | 異種移植のための多トランスジェニックブタ |
| JP2025052613A Pending JP2025092572A (ja) | 2015-09-09 | 2025-03-26 | 異種移植のための多トランスジェニックブタ |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021068874A Pending JP2021101741A (ja) | 2015-09-09 | 2021-04-15 | 異種移植のための多トランスジェニックブタ |
| JP2023113055A Withdrawn JP2023119041A (ja) | 2015-09-09 | 2023-07-10 | 異種移植のための多トランスジェニックブタ |
| JP2025052613A Pending JP2025092572A (ja) | 2015-09-09 | 2025-03-26 | 異種移植のための多トランスジェニックブタ |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20180249688A1 (https=) |
| EP (1) | EP3347458A4 (https=) |
| JP (4) | JP7050665B2 (https=) |
| KR (2) | KR20250161669A (https=) |
| CN (2) | CN108473963A (https=) |
| AU (3) | AU2016319131B2 (https=) |
| CA (1) | CA2998187A1 (https=) |
| WO (1) | WO2017044864A1 (https=) |
Families Citing this family (56)
| Publication number | Priority date | Publication date | Assignee | Title |
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| ES2680636T3 (es) | 2011-02-14 | 2018-09-10 | Revivicor Inc. | Cerdos genéticamente modificados para xenotrasplante de xenoinjertos vascularizados y derivados de los mismos |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| WO2015105955A1 (en) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| WO2016126831A1 (en) | 2015-02-03 | 2016-08-11 | Mayo Foundation For Medical Education And Research | Methods and materials for assessing and treating arthritis |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| CN107921148A (zh) | 2015-05-08 | 2018-04-17 | 哈佛学院校长同事会 | 通用供体干细胞和相关方法 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| WO2018041118A1 (en) | 2016-08-31 | 2018-03-08 | Beijing Biocytogen Co., Ltd | Genetically modified non-human animal with human or chimeric pd-l1 |
| EP3519570B1 (en) * | 2016-09-29 | 2022-06-08 | F. Hoffmann-La Roche AG | Method to analyze and optimize gene editing modules and delivery approaches |
| KR102746223B1 (ko) | 2017-04-20 | 2024-12-27 | 이제네시스, 인크. | 유전자 변형 동물의 생성 방법 |
| KR102363891B1 (ko) * | 2017-04-28 | 2022-02-17 | 서울대학교산학협력단 | ＨＯ-1 유전자 및 ＴＮＦＲ1-Ｆｃ 유전자를 동시에 발현하며 GGTA1 유전자 및 CMAH 유전자가 넉아웃된 형질전환 돼지 및 이의 용도 |
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| KR102439221B1 (ko) | 2017-12-14 | 2022-09-01 | 프로디자인 소닉스, 인크. | 음향 트랜스듀서 구동기 및 제어기 |
| CN108103098B (zh) | 2017-12-14 | 2020-07-28 | 华南理工大学 | 一种化合物皮肤致敏体外评估细胞模型及其构建方法 |
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| CN109045357B (zh) * | 2018-06-11 | 2019-09-13 | 武汉奥翔生物科技有限公司 | 种源皮肤及其制备方法 |
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| WO2020051396A1 (en) * | 2018-09-08 | 2020-03-12 | Blueallele, Llc | Methods and compositions for modifying the von willebrand factor gene |
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| MX2021003866A (es) | 2018-10-05 | 2021-09-08 | Xenotherapeutics Inc | Productos y metodos de xenotransplante. |
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| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
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| WO2021072778A1 (en) * | 2019-10-18 | 2021-04-22 | Egenesis, Inc. | Cells, tissues, organs, and/or animals having one or more modified genes for enhanced xenograft survival and/or tolerance |
| KR20220033468A (ko) * | 2019-05-16 | 2022-03-16 | 이제네시스, 인크. | 강화된 이종이식편 생존 및/또는 내성을 위한 하나 이상의 변형된 유전자를 갖는 세포, 조직, 장기 및/또는 동물 |
| CN110373392A (zh) * | 2019-06-10 | 2019-10-25 | 云南农业大学 | 一种适合肾脏异种移植供体猪的构建方法 |
| CN110373389A (zh) * | 2019-06-10 | 2019-10-25 | 云南农业大学 | 一种适合胰岛细胞及皮肤等组织异种移植供体猪的构建方法 |
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| CN110305872A (zh) * | 2019-07-17 | 2019-10-08 | 中国农业科学院北京畜牧兽医研究所 | 小型猪2型糖尿病模型的构建方法及应用 |
| KR102176161B1 (ko) * | 2019-07-23 | 2020-11-09 | 주식회사 옵티팜 | 돼지 내인성 레트로바이러스 Envlope C 음성, GGTA1, CMAH, iGb3s, β4GalNT2 유전자가 넉아웃되고, 인간 CD46 및 TBM 유전자를 발현하는 이종장기이식을 위한 형질전환 복제돼지 및 이의 제조방법 |
| EP3811777B1 (en) | 2019-10-27 | 2024-07-31 | Shanghai RAAS Blood Products Co., Ltd. | Genetically modified non-human animals humanised for protein c |
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| CN113429475A (zh) * | 2020-03-23 | 2021-09-24 | 成都中科奥格生物科技有限公司 | 一种胶原材料及其制备方法和用途 |
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| CN115232813B (zh) * | 2021-07-15 | 2025-11-11 | 南京启真基因工程有限公司 | 用于构建vWF基因突变的血管性血友病模型猪核移植供体细胞的基因编辑系统及其应用 |
| KR102641015B1 (ko) * | 2021-08-04 | 2024-02-27 | 대한민국 | 항염증 및 면역 억제 유전자 발현을 위한 구조체가 도입된 gtko/cmahko 세포주와 이의 용도 |
| CA3232376A1 (en) | 2021-09-20 | 2023-03-23 | Maria KOKKINAKI | Multitransgenic pigs comprising ten genetic modifications for xenotransplantation |
| KR102738812B1 (ko) * | 2021-12-16 | 2024-12-09 | 주식회사 옵티팜 | 돼지 GGTA1, CMAH, β4GalNT2 및 CD40 유전자가 결손된 이종장기이식을 위한 형질전환 복제돼지 및 이의 제조방법 |
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| KR20260050358A (ko) * | 2023-05-26 | 2026-04-14 | 에크하드 울프 | 유전자 변형된 돼지 세포, 유전자 변형된 돼지 및 이의 생산 방법 |
| CN117099743B (zh) * | 2023-07-11 | 2025-12-05 | 吉林大学 | 一种snx6基因不表达的异种移植供体及制备方法 |
| KR102722231B1 (ko) * | 2023-12-15 | 2024-10-28 | 주식회사 옵티팜 | 돼지 GGTA1, CMAH, iGb3s, β4GalNT2 유전자가 결손되고 인간 CD59 유전자가 넉인된 이종수혈 및 이종장기이식을 위한 형질전환 복제돼지 및 이의 제조방법 |
| WO2025235683A1 (en) | 2024-05-07 | 2025-11-13 | Eledon Pharmaceuticals, Inc. | Methods and compositions for prevention of transplant rejection |
| WO2025259640A1 (en) * | 2024-06-14 | 2025-12-18 | The Board Of Trustees Of The Leland Stanford Junior University | Systems and methods for bioengineering hypoimmunogenic organs, tissues and cells |
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|---|---|---|---|---|
| US6518482B2 (en) * | 1994-09-13 | 2003-02-11 | American National Red Cross | Transgenic non-human mammals expressing human coagulation factor VIII and von Willebrand factor |
| KR101149475B1 (ko) | 2007-11-30 | 2012-05-25 | 한국생명공학연구원 | 이종 장기 이식용 미니 복제돼지 생산을 위한 유전자 조작된 세포주 및 그의 생산방법 |
| ES2767881T3 (es) * | 2009-08-14 | 2020-06-18 | Revivicor Inc | Cerdos multi-transgénicos para el tratamiento de diabetes |
| ES2680636T3 (es) * | 2011-02-14 | 2018-09-10 | Revivicor Inc. | Cerdos genéticamente modificados para xenotrasplante de xenoinjertos vascularizados y derivados de los mismos |
| WO2013188358A1 (en) * | 2012-06-12 | 2013-12-19 | The General Hospital Corporation | Miniature swine transgenic for one or more coagulation factors |
| US20130347134A1 (en) * | 2012-06-21 | 2013-12-26 | Thomas Diacovo | Transgenic von willebrand factor animals and uses thereof |
| US20140115728A1 (en) * | 2012-10-24 | 2014-04-24 | A. Joseph Tector | Double knockout (gt/cmah-ko) pigs, organs and tissues |
| CA2965550A1 (en) * | 2014-10-22 | 2016-04-28 | Indiana University Research & Technology Corporation | Triple transgenic pigs suitable for xenograft |
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2016
- 2016-09-09 EP EP16845199.5A patent/EP3347458A4/en active Pending
- 2016-09-09 WO PCT/US2016/051126 patent/WO2017044864A1/en not_active Ceased
- 2016-09-09 KR KR1020257036936A patent/KR20250161669A/ko active Pending
- 2016-09-09 JP JP2018512546A patent/JP7050665B2/ja active Active
- 2016-09-09 KR KR1020187009894A patent/KR102883068B1/ko active Active
- 2016-09-09 AU AU2016319131A patent/AU2016319131B2/en active Active
- 2016-09-09 US US15/758,895 patent/US20180249688A1/en active Pending
- 2016-09-09 CA CA2998187A patent/CA2998187A1/en active Pending
- 2016-09-09 CN CN201680065556.6A patent/CN108473963A/zh active Pending
- 2016-09-09 CN CN202210920541.0A patent/CN115380872A/zh active Pending
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2021
- 2021-04-15 JP JP2021068874A patent/JP2021101741A/ja active Pending
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2022
- 2022-11-07 AU AU2022268274A patent/AU2022268274B2/en active Active
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2023
- 2023-07-10 JP JP2023113055A patent/JP2023119041A/ja not_active Withdrawn
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2025
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