JP2020510827A5 - - Google Patents
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- JP2020510827A5 JP2020510827A5 JP2019546343A JP2019546343A JP2020510827A5 JP 2020510827 A5 JP2020510827 A5 JP 2020510827A5 JP 2019546343 A JP2019546343 A JP 2019546343A JP 2019546343 A JP2019546343 A JP 2019546343A JP 2020510827 A5 JP2020510827 A5 JP 2020510827A5
- Authority
- JP
- Japan
- Prior art keywords
- rodent
- gene
- symptoms
- locus
- nucleic acid
- 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.)
- Granted
Links
- 241000283984 Rodentia Species 0.000 claims 53
- 238000000034 method Methods 0.000 claims 40
- 101150071258 C3 gene Proteins 0.000 claims 22
- 208000024891 symptom Diseases 0.000 claims 20
- 208000017169 kidney disease Diseases 0.000 claims 18
- 150000007523 nucleic acids Chemical group 0.000 claims 18
- 101100328540 Homo sapiens C3 gene Proteins 0.000 claims 16
- 239000003814 drug Substances 0.000 claims 16
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 14
- 229940079593 drug Drugs 0.000 claims 13
- 239000003795 chemical substances by application Substances 0.000 claims 11
- 238000011808 rodent model Methods 0.000 claims 10
- 108090000623 proteins and genes Proteins 0.000 claims 9
- 230000000295 complement effect Effects 0.000 claims 8
- 230000001105 regulatory effect Effects 0.000 claims 8
- 208000019425 cirrhosis of liver Diseases 0.000 claims 7
- 238000006467 substitution reaction Methods 0.000 claims 6
- 230000002401 inhibitory effect Effects 0.000 claims 5
- 108700024394 Exon Proteins 0.000 claims 4
- 101100328542 Mus musculus C3 gene Proteins 0.000 claims 4
- 101100440312 Mus musculus C5 gene Proteins 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 4
- 102000039446 nucleic acids Human genes 0.000 claims 4
- 230000001225 therapeutic effect Effects 0.000 claims 4
- 206010019668 Hepatic fibrosis Diseases 0.000 claims 3
- 210000002966 serum Anatomy 0.000 claims 3
- 229940124597 therapeutic agent Drugs 0.000 claims 3
- 238000011282 treatment Methods 0.000 claims 3
- 102100036475 Alanine aminotransferase 1 Human genes 0.000 claims 2
- 108010082126 Alanine transaminase Proteins 0.000 claims 2
- 102000002260 Alkaline Phosphatase Human genes 0.000 claims 2
- 108020004774 Alkaline Phosphatase Proteins 0.000 claims 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 2
- 108091023037 Aptamer Proteins 0.000 claims 2
- 108010003415 Aspartate Aminotransferases Proteins 0.000 claims 2
- 102000004625 Aspartate Aminotransferases Human genes 0.000 claims 2
- 102000053642 Catalytic RNA Human genes 0.000 claims 2
- 108090000994 Catalytic RNA Proteins 0.000 claims 2
- 206010018364 Glomerulonephritis Diseases 0.000 claims 2
- 108091034117 Oligonucleotide Proteins 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- 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 2
- 239000000074 antisense oligonucleotide Substances 0.000 claims 2
- 238000012230 antisense oligonucleotides Methods 0.000 claims 2
- 230000008021 deposition Effects 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 238000001727 in vivo Methods 0.000 claims 2
- 210000003734 kidney Anatomy 0.000 claims 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 108091092562 ribozyme Proteins 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 150000003384 small molecules Chemical class 0.000 claims 2
- 239000000126 substance Substances 0.000 claims 2
- 101710131943 40S ribosomal protein S3a Proteins 0.000 claims 1
- 108010088751 Albumins Proteins 0.000 claims 1
- 102000009027 Albumins Human genes 0.000 claims 1
- 108010034753 Complement Membrane Attack Complex Proteins 0.000 claims 1
- 102000012192 Cystatin C Human genes 0.000 claims 1
- 108010061642 Cystatin C Proteins 0.000 claims 1
- 102000015779 HDL Lipoproteins Human genes 0.000 claims 1
- 108010010234 HDL Lipoproteins Proteins 0.000 claims 1
- 206010020880 Hypertrophy Diseases 0.000 claims 1
- 206010061218 Inflammation Diseases 0.000 claims 1
- 102000004882 Lipase Human genes 0.000 claims 1
- 108090001060 Lipase Proteins 0.000 claims 1
- 239000004367 Lipase Substances 0.000 claims 1
- PNNCWTXUWKENPE-UHFFFAOYSA-N [N].NC(N)=O Chemical compound [N].NC(N)=O PNNCWTXUWKENPE-UHFFFAOYSA-N 0.000 claims 1
- 210000000577 adipose tissue Anatomy 0.000 claims 1
- 210000004369 blood Anatomy 0.000 claims 1
- 239000008280 blood Substances 0.000 claims 1
- 230000037182 bone density Effects 0.000 claims 1
- 230000001434 glomerular Effects 0.000 claims 1
- 230000004054 inflammatory process Effects 0.000 claims 1
- 235000019421 lipase Nutrition 0.000 claims 1
- 239000011159 matrix material Substances 0.000 claims 1
- 235000018102 proteins Nutrition 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 210000005239 tubule Anatomy 0.000 claims 1
- 230000002485 urinary effect Effects 0.000 claims 1
- 230000004580 weight loss Effects 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762464262P | 2017-02-27 | 2017-02-27 | |
| US62/464,262 | 2017-02-27 | ||
| US201762529916P | 2017-07-07 | 2017-07-07 | |
| US62/529,916 | 2017-07-07 | ||
| US201762583780P | 2017-11-09 | 2017-11-09 | |
| US62/583,780 | 2017-11-09 | ||
| PCT/US2018/019651 WO2018157027A1 (en) | 2017-02-27 | 2018-02-26 | Humanized model of kidney and liver disorders |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020510827A JP2020510827A (ja) | 2020-04-09 |
| JP2020510827A5 true JP2020510827A5 (enExample) | 2021-04-01 |
| JP7169979B2 JP7169979B2 (ja) | 2022-11-11 |
Family
ID=61617150
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019546343A Active JP7169979B2 (ja) | 2017-02-27 | 2018-02-26 | 補体関連腎症および肝線維症の候補治療剤の治療有効性を評価する方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US10765762B2 (enExample) |
| EP (1) | EP3585445B1 (enExample) |
| JP (1) | JP7169979B2 (enExample) |
| KR (1) | KR102602199B1 (enExample) |
| CN (1) | CN110494168B (enExample) |
| AU (1) | AU2018225745B2 (enExample) |
| CA (1) | CA3054167A1 (enExample) |
| ES (1) | ES2975416T3 (enExample) |
| IL (1) | IL268748B2 (enExample) |
| MX (1) | MX2019010219A (enExample) |
| SG (2) | SG11201907606XA (enExample) |
| WO (1) | WO2018157027A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3585162B1 (en) | 2017-09-29 | 2023-08-30 | Regeneron Pharmaceuticals, Inc. | Rodents comprising a humanized ttr locus and methods of use |
| SMT202400300T1 (it) | 2018-07-16 | 2024-09-16 | Regeneron Pharma | Modelli di roditori della sindrome ditra e loro utilizzo |
| US20220053741A1 (en) * | 2018-09-13 | 2022-02-24 | Regeneron Pharmaceuticals, Inc. | Complement Factor H Gene Knockout Rat as a Model of C3 Glomerulopathy |
| CN117178959A (zh) | 2019-04-04 | 2023-12-08 | 瑞泽恩制药公司 | 包括人源化凝血因子12基因座的非人动物 |
| AU2020286382A1 (en) | 2019-06-04 | 2021-11-04 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized TTR locus with a beta-slip mutation and methods of use |
| MX2021015122A (es) | 2019-06-07 | 2022-04-06 | Regeneron Pharma | Animales no humanos que comprenden un locus de albumina humanizado. |
| WO2021112235A1 (ja) * | 2019-12-05 | 2021-06-10 | 国立大学法人宮崎大学 | Ｃ３腎症を予防又は治療するための医薬、医薬組成物及び補体Ｃ３ｂ分解促進剤 |
| IL315694A (en) * | 2020-10-01 | 2024-11-01 | Regeneron Pharma | Rodent animals expressing human cr1 |
| WO2025122669A1 (en) | 2023-12-05 | 2025-06-12 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a humanized complement factor b gene |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1984003506A1 (en) | 1983-03-08 | 1984-09-13 | Commw Serum Lab Commission | Antigenically active amino acid sequences |
| NZ207394A (en) | 1983-03-08 | 1987-03-06 | Commw Serum Lab Commission | Detecting or determining sequence of amino acids |
| US4708871A (en) | 1983-03-08 | 1987-11-24 | Commonwealth Serum Laboratories Commission | Antigenically active amino acid sequences |
| AU4434585A (en) | 1985-03-30 | 1986-10-23 | Marc Ballivet | Method for obtaining dna, rna, peptides, polypeptides or proteins by means of a dna recombinant technique |
| NZ215865A (en) | 1985-04-22 | 1988-10-28 | Commw Serum Lab Commission | Method of determining the active site of a receptor-binding analogue |
| US5763192A (en) | 1986-11-20 | 1998-06-09 | Ixsys, Incorporated | Process for obtaining DNA, RNA, peptides, polypeptides, or protein, by recombinant DNA technique |
| DE3883899T3 (de) | 1987-03-18 | 1999-04-22 | Sb2, Inc., Danville, Calif. | Geänderte antikörper. |
| US5266684A (en) | 1988-05-02 | 1993-11-30 | The Reagents Of The University Of California | Peptide mixtures |
| US5571689A (en) | 1988-06-16 | 1996-11-05 | Washington University | Method of N-acylating peptide and proteins with diheteroatom substituted analogs of myristic acid |
| US5223409A (en) | 1988-09-02 | 1993-06-29 | Protein Engineering Corp. | Directed evolution of novel binding proteins |
| US5663143A (en) | 1988-09-02 | 1997-09-02 | Dyax Corp. | Engineered human-derived kunitz domains that inhibit human neutrophil elastase |
| US5427908A (en) | 1990-05-01 | 1995-06-27 | Affymax Technologies N.V. | Recombinant library screening methods |
| US5723286A (en) | 1990-06-20 | 1998-03-03 | Affymax Technologies N.V. | Peptide library and screening systems |
| US5698426A (en) | 1990-09-28 | 1997-12-16 | Ixsys, Incorporated | Surface expression libraries of heteromeric receptors |
| US5770434A (en) | 1990-09-28 | 1998-06-23 | Ixsys Incorporated | Soluble peptides having constrained, secondary conformation in solution and method of making same |
| ATE176239T1 (de) | 1990-11-21 | 1999-02-15 | Iterex Pharma Lp | Synthese äquimolarer mischungen vielzähliger oligomere, speziell oligopeptidmischungen |
| CA2095633C (en) | 1990-12-03 | 2003-02-04 | Lisa J. Garrard | Enrichment method for variant proteins with altered binding properties |
| US5270170A (en) | 1991-10-16 | 1993-12-14 | Affymax Technologies N.V. | Peptide library and screening method |
| US5644048A (en) | 1992-01-10 | 1997-07-01 | Isis Pharmaceuticals, Inc. | Process for preparing phosphorothioate oligonucleotides |
| WO1994029351A2 (en) | 1993-06-16 | 1994-12-22 | Celltech Limited | Antibodies |
| GB9521943D0 (en) | 1995-10-26 | 1996-01-03 | Univ Hertfordshire | Coded particles for process sequence tracking in combinatorial compound library preparation |
| PT1068241E (pt) | 1998-04-02 | 2007-11-19 | Genentech Inc | Variantes de anticorpos e respectivos fragmentos |
| US6335155B1 (en) | 1998-06-26 | 2002-01-01 | Sunesis Pharmaceuticals, Inc. | Methods for rapidly identifying small organic molecule ligands for binding to biological target molecules |
| US6280946B2 (en) | 1998-08-07 | 2001-08-28 | Boston Probes, Inc. | PNA probes, probe sets, methods and kits pertaining to the universal detection of bacteria and eucarya |
| EP1141708A1 (en) | 1998-12-28 | 2001-10-10 | Sunesis Pharmaceuticals Inc. | Identifying small organic molecule ligands for binding |
| US20090032592A1 (en) | 2006-01-11 | 2009-02-05 | Novo Nordisk A/S | Spherical encoded beads |
| BRPI0812767A2 (pt) * | 2007-06-07 | 2014-12-02 | Genentech Inc | Anticorpos para c3b e métodos para prevenção e tratamento de distúrbios associados ao complemento |
| CA2742802C (en) | 2008-11-10 | 2019-11-26 | Alexion Pharmaceuticals, Inc. | Methods and compositions for treating complement-associated disorders |
| US9243060B2 (en) * | 2012-04-03 | 2016-01-26 | Novelmed Therapeutics, Inc. | Humanized anti-factor C3b antibodies and uses thereof |
| JP6155332B2 (ja) * | 2012-06-28 | 2017-06-28 | ノバルティス アーゲー | ピロリジン誘導体、および補体経路調節因子としてのその使用 |
| AU2014306002B2 (en) * | 2013-08-07 | 2017-05-25 | Alexion Pharmaceuticals, Inc. | Atypical hemolytic uremic syndrome (aHUS) biomarker proteins |
| US9659904B2 (en) * | 2013-12-12 | 2017-05-23 | Intel Corporation | Distributed on-package millimeter-wave radio |
| EP3110804A4 (en) * | 2014-02-25 | 2017-11-22 | Achillion Pharmaceuticals, Inc. | Carbamate, ester, and ketone compounds for treatment of complement mediated disorders |
| MX2016014504A (es) | 2014-05-05 | 2017-05-23 | Regeneron Pharma | Animales c5 y c3 humanizados. |
-
2018
- 2018-02-26 JP JP2019546343A patent/JP7169979B2/ja active Active
- 2018-02-26 US US15/904,808 patent/US10765762B2/en active Active
- 2018-02-26 KR KR1020197027971A patent/KR102602199B1/ko active Active
- 2018-02-26 CN CN201880021486.3A patent/CN110494168B/zh active Active
- 2018-02-26 CA CA3054167A patent/CA3054167A1/en active Pending
- 2018-02-26 ES ES18710242T patent/ES2975416T3/es active Active
- 2018-02-26 SG SG11201907606XA patent/SG11201907606XA/en unknown
- 2018-02-26 IL IL268748A patent/IL268748B2/en unknown
- 2018-02-26 SG SG10202111663PA patent/SG10202111663PA/en unknown
- 2018-02-26 WO PCT/US2018/019651 patent/WO2018157027A1/en not_active Ceased
- 2018-02-26 AU AU2018225745A patent/AU2018225745B2/en active Active
- 2018-02-26 EP EP18710242.1A patent/EP3585445B1/en active Active
- 2018-02-26 MX MX2019010219A patent/MX2019010219A/es unknown
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