JP2018506297A5 - - Google Patents
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- JP2018506297A5 JP2018506297A5 JP2017550092A JP2017550092A JP2018506297A5 JP 2018506297 A5 JP2018506297 A5 JP 2018506297A5 JP 2017550092 A JP2017550092 A JP 2017550092A JP 2017550092 A JP2017550092 A JP 2017550092A JP 2018506297 A5 JP2018506297 A5 JP 2018506297A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- pharmaceutical composition
- editing
- disease
- modification
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 108091034117 Oligonucleotide Proteins 0.000 claims 77
- 239000008194 pharmaceutical composition Substances 0.000 claims 54
- 238000000034 method Methods 0.000 claims 43
- 230000004048 modification Effects 0.000 claims 36
- 238000012986 modification Methods 0.000 claims 36
- 108020004707 nucleic acids Proteins 0.000 claims 35
- 102000039446 nucleic acids Human genes 0.000 claims 35
- 150000007523 nucleic acids Chemical class 0.000 claims 35
- 108020004705 Codon Proteins 0.000 claims 30
- 239000002773 nucleotide Substances 0.000 claims 20
- 125000003729 nucleotide group Chemical group 0.000 claims 20
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 17
- 230000000295 complement effect Effects 0.000 claims 16
- 108090000623 proteins and genes Proteins 0.000 claims 15
- 102000004169 proteins and genes Human genes 0.000 claims 15
- 201000010099 disease Diseases 0.000 claims 11
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 11
- 101710163270 Nuclease Proteins 0.000 claims 9
- 229910052736 halogen Inorganic materials 0.000 claims 8
- 150000002367 halogens Chemical class 0.000 claims 8
- 230000035772 mutation Effects 0.000 claims 7
- 208000007056 sickle cell anemia Diseases 0.000 claims 7
- 201000003883 Cystic fibrosis Diseases 0.000 claims 6
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 6
- 108020005187 Oligonucleotide Probes Proteins 0.000 claims 6
- 239000002751 oligonucleotide probe Substances 0.000 claims 6
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 4
- 102000008300 Mutant Proteins Human genes 0.000 claims 4
- 108010021466 Mutant Proteins Proteins 0.000 claims 4
- 101710128896 Tyrosine-protein phosphatase non-receptor type 1 Proteins 0.000 claims 4
- 150000001413 amino acids Chemical class 0.000 claims 4
- 229910052794 bromium Inorganic materials 0.000 claims 4
- 229910052801 chlorine Inorganic materials 0.000 claims 4
- 239000003937 drug carrier Substances 0.000 claims 4
- 230000000694 effects Effects 0.000 claims 4
- 230000008451 emotion Effects 0.000 claims 4
- 229910052731 fluorine Inorganic materials 0.000 claims 4
- 229910052740 iodine Inorganic materials 0.000 claims 4
- 208000011580 syndromic disease Diseases 0.000 claims 4
- 208000001072 type 2 diabetes mellitus Diseases 0.000 claims 4
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- 208000024827 Alzheimer disease Diseases 0.000 claims 2
- 206010061666 Autonomic neuropathy Diseases 0.000 claims 2
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims 2
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 claims 2
- 201000004569 Blindness Diseases 0.000 claims 2
- 206010006187 Breast cancer Diseases 0.000 claims 2
- 208000026310 Breast neoplasm Diseases 0.000 claims 2
- 208000022526 Canavan disease Diseases 0.000 claims 2
- 206010007509 Cardiac amyloidosis Diseases 0.000 claims 2
- 208000010693 Charcot-Marie-Tooth Disease Diseases 0.000 claims 2
- 102100022641 Coagulation factor IX Human genes 0.000 claims 2
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 2
- 206010010099 Combined immunodeficiency Diseases 0.000 claims 2
- 208000024720 Fabry Disease Diseases 0.000 claims 2
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 2
- 208000034846 Familial Amyloid Neuropathies Diseases 0.000 claims 2
- 208000024412 Friedreich ataxia Diseases 0.000 claims 2
- 208000001905 GM2 Gangliosidoses Diseases 0.000 claims 2
- 201000008905 GM2 gangliosidosis Diseases 0.000 claims 2
- 208000015872 Gaucher disease Diseases 0.000 claims 2
- 208000020322 Gaucher disease type I Diseases 0.000 claims 2
- 208000018565 Hemochromatosis Diseases 0.000 claims 2
- 208000009292 Hemophilia A Diseases 0.000 claims 2
- 206010019889 Hereditary neuropathic amyloidosis Diseases 0.000 claims 2
- 108010053317 Hexosaminidase A Proteins 0.000 claims 2
- 102000016871 Hexosaminidase A Human genes 0.000 claims 2
- 101001045440 Homo sapiens Beta-hexosaminidase subunit alpha Proteins 0.000 claims 2
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 2
- 101001098868 Homo sapiens Proprotein convertase subtilisin/kexin type 9 Proteins 0.000 claims 2
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 claims 2
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims 2
- 201000011252 Phenylketonuria Diseases 0.000 claims 2
- 208000024777 Prion disease Diseases 0.000 claims 2
- 102100038955 Proprotein convertase subtilisin/kexin type 9 Human genes 0.000 claims 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 2
- 206010067584 Type 1 diabetes mellitus Diseases 0.000 claims 2
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims 2
- 201000009628 adenosine deaminase deficiency Diseases 0.000 claims 2
- 239000000443 aerosol Substances 0.000 claims 2
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 claims 2
- 206010002022 amyloidosis Diseases 0.000 claims 2
- 208000036556 autosomal recessive T cell-negative B cell-negative NK cell-negative due to adenosine deaminase deficiency severe combined immunodeficiency Diseases 0.000 claims 2
- 208000005980 beta thalassemia Diseases 0.000 claims 2
- 239000008280 blood Substances 0.000 claims 2
- 210000004369 blood Anatomy 0.000 claims 2
- 239000007975 buffered saline Substances 0.000 claims 2
- 201000002758 colorectal adenoma Diseases 0.000 claims 2
- 208000011664 congenital factor XI deficiency Diseases 0.000 claims 2
- 230000007812 deficiency Effects 0.000 claims 2
- 201000007219 factor XI deficiency Diseases 0.000 claims 2
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims 2
- 208000007345 glycogen storage disease Diseases 0.000 claims 2
- 230000036541 health Effects 0.000 claims 2
- 208000009429 hemophilia B Diseases 0.000 claims 2
- 238000010255 intramuscular injection Methods 0.000 claims 2
- 239000007927 intramuscular injection Substances 0.000 claims 2
- 239000007928 intraperitoneal injection Substances 0.000 claims 2
- 238000010253 intravenous injection Methods 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 210000001672 ovary Anatomy 0.000 claims 2
- 208000030761 polycystic kidney disease Diseases 0.000 claims 2
- 239000006215 rectal suppository Substances 0.000 claims 2
- 229940100618 rectal suppository Drugs 0.000 claims 2
- 239000011780 sodium chloride Substances 0.000 claims 2
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 2
- 238000010254 subcutaneous injection Methods 0.000 claims 2
- 239000007929 subcutaneous injection Substances 0.000 claims 2
- 230000037317 transdermal delivery Effects 0.000 claims 2
- 201000007905 transthyretin amyloidosis Diseases 0.000 claims 2
- 239000006216 vaginal suppository Substances 0.000 claims 2
- 229940120293 vaginal suppository Drugs 0.000 claims 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 2
- 208000027121 wild type ATTR amyloidosis Diseases 0.000 claims 2
- BOKOVLFWCAFYHP-UHFFFAOYSA-N dihydroxy-methoxy-sulfanylidene-$l^{5}-phosphane Chemical compound COP(O)(O)=S BOKOVLFWCAFYHP-UHFFFAOYSA-N 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- YACKEPLHDIMKIO-UHFFFAOYSA-L methylphosphonate(2-) Chemical compound CP([O-])([O-])=O YACKEPLHDIMKIO-UHFFFAOYSA-L 0.000 claims 1
- 150000004713 phosphodiesters Chemical class 0.000 claims 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
- 0 C*COC(C1*)C(CO*)OC1[n]1c2ncnc(*)c2c(CNC(CCC(OCN(C)N=O)=O)=O)c1 Chemical compound C*COC(C1*)C(CO*)OC1[n]1c2ncnc(*)c2c(CNC(CCC(OCN(C)N=O)=O)=O)c1 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021159689A JP2022008651A (ja) | 2014-12-12 | 2021-09-29 | オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法 |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462091027P | 2014-12-12 | 2014-12-12 | |
| US62/091,027 | 2014-12-12 | ||
| US201562141077P | 2015-03-31 | 2015-03-31 | |
| US62/141,077 | 2015-03-31 | ||
| US201562180175P | 2015-06-16 | 2015-06-16 | |
| US62/180,175 | 2015-06-16 | ||
| US201562252693P | 2015-11-09 | 2015-11-09 | |
| US62/252,693 | 2015-11-09 | ||
| PCT/US2015/065348 WO2016094845A2 (en) | 2014-12-12 | 2015-12-11 | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021159689A Division JP2022008651A (ja) | 2014-12-12 | 2021-09-29 | オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018506297A JP2018506297A (ja) | 2018-03-08 |
| JP2018506297A5 true JP2018506297A5 (https=) | 2019-01-31 |
| JP6997623B2 JP6997623B2 (ja) | 2022-02-04 |
Family
ID=56108384
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017550092A Active JP6997623B2 (ja) | 2014-12-12 | 2015-12-11 | オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法 |
| JP2021159689A Pending JP2022008651A (ja) | 2014-12-12 | 2021-09-29 | オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021159689A Pending JP2022008651A (ja) | 2014-12-12 | 2021-09-29 | オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US12359197B2 (https=) |
| EP (2) | EP4372091A3 (https=) |
| JP (2) | JP6997623B2 (https=) |
| CN (1) | CN107532162A (https=) |
| CA (1) | CA2969464A1 (https=) |
| WO (1) | WO2016094845A2 (https=) |
Families Citing this family (62)
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| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| US12359197B2 (en) * | 2014-12-12 | 2025-07-15 | Etagen Pharma, Inc. | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides |
| EP3234134B1 (en) | 2014-12-17 | 2020-05-27 | ProQR Therapeutics II B.V. | Targeted rna editing |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| CA3022319A1 (en) * | 2016-05-06 | 2017-11-09 | Tod M. Woolf | Improved methods for genome editing with and without programmable nucleases |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| EP4056701A1 (en) * | 2016-08-12 | 2022-09-14 | Nexuspiral Inc. | Genome editing method |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| CN110352244B (zh) | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| KR20230125856A (ko) * | 2016-12-23 | 2023-08-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | Pcsk9의 유전자 편집 |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| CN110612353A (zh) | 2017-03-03 | 2019-12-24 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| CN107267516B (zh) * | 2017-07-28 | 2020-09-29 | 佛山科学技术学院 | 双sgRNA介导的基因精确修饰方法及应用 |
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| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| WO2019104094A2 (en) | 2017-11-21 | 2019-05-31 | The Regents Of The University Of California | Fusion proteins and methods for site-directed genome editing |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019136459A1 (en) | 2018-01-08 | 2019-07-11 | Iovance Biotherapeutics, Inc. | Processes for generating til products enriched for tumor antigen-specific t-cells |
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-
2015
- 2015-12-11 US US15/533,965 patent/US12359197B2/en active Active
- 2015-12-11 EP EP24153079.9A patent/EP4372091A3/en active Pending
- 2015-12-11 CN CN201580075918.5A patent/CN107532162A/zh active Pending
- 2015-12-11 WO PCT/US2015/065348 patent/WO2016094845A2/en not_active Ceased
- 2015-12-11 JP JP2017550092A patent/JP6997623B2/ja active Active
- 2015-12-11 EP EP15868062.9A patent/EP3230445B1/en active Active
- 2015-12-11 CA CA2969464A patent/CA2969464A1/en not_active Abandoned
-
2021
- 2021-09-29 JP JP2021159689A patent/JP2022008651A/ja active Pending
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