JP2019500428A5 - - Google Patents
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- JP2019500428A5 JP2019500428A5 JP2018550880A JP2018550880A JP2019500428A5 JP 2019500428 A5 JP2019500428 A5 JP 2019500428A5 JP 2018550880 A JP2018550880 A JP 2018550880A JP 2018550880 A JP2018550880 A JP 2018550880A JP 2019500428 A5 JP2019500428 A5 JP 2019500428A5
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- 108091034117 Oligonucleotide Proteins 0.000 claims description 69
- 201000011240 Frontotemporal dementia Diseases 0.000 claims description 14
- 206010012289 Dementia Diseases 0.000 claims description 12
- 201000010099 disease Diseases 0.000 claims description 12
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 12
- 239000002773 nucleotide Substances 0.000 claims description 12
- 125000003729 nucleotide group Chemical group 0.000 claims description 12
- 238000012986 modification Methods 0.000 claims description 11
- 150000007523 nucleic acids Chemical class 0.000 claims description 9
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 8
- 238000000034 method Methods 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 7
- 102000039446 nucleic acids Human genes 0.000 claims description 7
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 6
- 108020004999 messenger RNA Proteins 0.000 claims description 6
- 210000002682 neurofibrillary tangle Anatomy 0.000 claims description 6
- 102000004169 proteins and genes Human genes 0.000 claims description 6
- 108090000623 proteins and genes Proteins 0.000 claims description 6
- 208000024827 Alzheimer disease Diseases 0.000 claims description 4
- 208000005145 Cerebral amyloid angiopathy Diseases 0.000 claims description 4
- 208000004051 Chronic Traumatic Encephalopathy Diseases 0.000 claims description 4
- 208000020406 Creutzfeldt Jacob disease Diseases 0.000 claims description 4
- 208000003407 Creutzfeldt-Jakob Syndrome Diseases 0.000 claims description 4
- 208000010859 Creutzfeldt-Jakob disease Diseases 0.000 claims description 4
- 208000010577 Niemann-Pick disease type C Diseases 0.000 claims description 4
- 206010034010 Parkinsonism Diseases 0.000 claims description 4
- 208000000609 Pick Disease of the Brain Diseases 0.000 claims description 4
- 208000007930 Type C Niemann-Pick Disease Diseases 0.000 claims description 4
- 229940104302 cytosine Drugs 0.000 claims description 4
- 208000017004 dementia pugilistica Diseases 0.000 claims description 4
- 201000002212 progressive supranuclear palsy Diseases 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 229960000549 4-dimethylaminophenol Drugs 0.000 claims description 2
- VHYFNPMBLIVWCW-UHFFFAOYSA-N 4-dimethylaminopyridine Substances CN(C)C1=CC=NC=C1 VHYFNPMBLIVWCW-UHFFFAOYSA-N 0.000 claims description 2
- 206010059245 Angiopathy Diseases 0.000 claims description 2
- 208000014644 Brain disease Diseases 0.000 claims description 2
- 208000009093 Diffuse Neurofibrillary Tangles with Calcification Diseases 0.000 claims description 2
- 201000010374 Down Syndrome Diseases 0.000 claims description 2
- 208000032274 Encephalopathy Diseases 0.000 claims description 2
- 208000002339 Frontotemporal Lobar Degeneration Diseases 0.000 claims description 2
- 208000003736 Gerstmann-Straussler-Scheinker Disease Diseases 0.000 claims description 2
- 206010072075 Gerstmann-Straussler-Scheinker syndrome Diseases 0.000 claims description 2
- 206010018341 Gliosis Diseases 0.000 claims description 2
- 208000023105 Huntington disease Diseases 0.000 claims description 2
- 206010061216 Infarction Diseases 0.000 claims description 2
- 208000032382 Ischaemic stroke Diseases 0.000 claims description 2
- 208000001089 Multiple system atrophy Diseases 0.000 claims description 2
- 206010068871 Myotonic dystrophy Diseases 0.000 claims description 2
- 102000029797 Prion Human genes 0.000 claims description 2
- 108091000054 Prion Proteins 0.000 claims description 2
- 208000037065 Subacute sclerosing leukoencephalitis Diseases 0.000 claims description 2
- 206010042297 Subacute sclerosing panencephalitis Diseases 0.000 claims description 2
- 208000026911 Tuberous sclerosis complex Diseases 0.000 claims description 2
- 230000003213 activating effect Effects 0.000 claims description 2
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims description 2
- 210000000349 chromosome Anatomy 0.000 claims description 2
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- 239000003937 drug carrier Substances 0.000 claims description 2
- 206010014599 encephalitis Diseases 0.000 claims description 2
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- 238000000338 in vitro Methods 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 201000008319 inclusion body myositis Diseases 0.000 claims description 2
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- 150000004713 phosphodiesters Chemical class 0.000 claims description 2
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- 208000009999 tuberous sclerosis Diseases 0.000 claims description 2
- 208000024571 Pick disease Diseases 0.000 claims 2
- 208000011580 syndromic disease Diseases 0.000 claims 2
- 208000032612 Glial tumor Diseases 0.000 claims 1
- 206010018338 Glioma Diseases 0.000 claims 1
- 208000027089 Parkinsonian disease Diseases 0.000 claims 1
- 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 1
- 210000003710 cerebral cortex Anatomy 0.000 claims 1
- -1 dimethylaminoethyloxyethyl Chemical group 0.000 claims 1
- 201000005649 gangliocytoma Diseases 0.000 claims 1
- 201000008361 ganglioneuroma Diseases 0.000 claims 1
- 210000005036 nerve Anatomy 0.000 claims 1
- 239000002777 nucleoside Substances 0.000 description 4
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- 230000000295 complement effect Effects 0.000 description 2
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- HWPZZUQOWRWFDB-UHFFFAOYSA-N 1-methylcytosine Chemical compound CN1C=CC(N)=NC1=O HWPZZUQOWRWFDB-UHFFFAOYSA-N 0.000 description 1
- 201000007547 Dravet syndrome Diseases 0.000 description 1
- 208000036572 Myoclonic epilepsy Diseases 0.000 description 1
- 206010028980 Neoplasm Diseases 0.000 description 1
- 108091028043 Nucleic acid sequence Proteins 0.000 description 1
- 229910019142 PO4 Inorganic materials 0.000 description 1
- 206010073677 Severe myoclonic epilepsy of infancy Diseases 0.000 description 1
- BQCADISMDOOEFD-UHFFFAOYSA-N Silver Chemical compound [Ag] BQCADISMDOOEFD-UHFFFAOYSA-N 0.000 description 1
- 230000002490 cerebral effect Effects 0.000 description 1
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- 238000007917 intracranial administration Methods 0.000 description 1
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- 239000010452 phosphate Substances 0.000 description 1
- 229910052709 silver Inorganic materials 0.000 description 1
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- 238000007920 subcutaneous administration Methods 0.000 description 1
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Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021076622A JP2021118732A (ja) | 2015-12-21 | 2021-04-28 | タウ発現を減少させるための組成物および方法 |
| JP2022021494A JP7594557B2 (ja) | 2015-12-21 | 2022-02-15 | タウ発現を減少させるための組成物および方法 |
| JP2024203529A JP2025026976A (ja) | 2015-12-21 | 2024-11-21 | タウ発現を減少させるための組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562270165P | 2015-12-21 | 2015-12-21 | |
| US62/270,165 | 2015-12-21 | ||
| PCT/IB2016/057794 WO2017109679A1 (en) | 2015-12-21 | 2016-12-19 | Compositions and methods for decreasing tau expression |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021076622A Division JP2021118732A (ja) | 2015-12-21 | 2021-04-28 | タウ発現を減少させるための組成物および方法 |
| JP2022021494A Division JP7594557B2 (ja) | 2015-12-21 | 2022-02-15 | タウ発現を減少させるための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500428A JP2019500428A (ja) | 2019-01-10 |
| JP2019500428A5 true JP2019500428A5 (https=) | 2020-01-30 |
| JP7027328B2 JP7027328B2 (ja) | 2022-03-01 |
Family
ID=57758674
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018550880A Active JP7027328B2 (ja) | 2015-12-21 | 2016-12-19 | タウ発現を減少させるための組成物および方法 |
| JP2021076622A Pending JP2021118732A (ja) | 2015-12-21 | 2021-04-28 | タウ発現を減少させるための組成物および方法 |
| JP2022021494A Active JP7594557B2 (ja) | 2015-12-21 | 2022-02-15 | タウ発現を減少させるための組成物および方法 |
| JP2024203529A Pending JP2025026976A (ja) | 2015-12-21 | 2024-11-21 | タウ発現を減少させるための組成物および方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021076622A Pending JP2021118732A (ja) | 2015-12-21 | 2021-04-28 | タウ発現を減少させるための組成物および方法 |
| JP2022021494A Active JP7594557B2 (ja) | 2015-12-21 | 2022-02-15 | タウ発現を減少させるための組成物および方法 |
| JP2024203529A Pending JP2025026976A (ja) | 2015-12-21 | 2024-11-21 | タウ発現を減少させるための組成物および方法 |
Country Status (40)
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2013203395A1 (en) | 2012-03-30 | 2013-10-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| ES2963428T3 (es) | 2016-09-29 | 2024-03-27 | Biogen Ma Inc | Compuestos y métodos para reducir la expresión de Tau |
| KR20200131263A (ko) | 2018-03-13 | 2020-11-23 | 얀센 파마슈티카 엔.브이. | 변형 올리고뉴클레오티드 및 타우병증에서의 사용 방법 |
| SG11202012759XA (en) | 2018-07-03 | 2021-01-28 | Hoffmann La Roche | Oligonucleotides for modulating tau expression |
| AU2021246024A1 (en) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals, Inc. | Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof |
| CN113817728A (zh) * | 2020-06-19 | 2021-12-21 | 四川大学华西医院 | 一种有效干扰Tau蛋白表达的重组慢病毒及其应用 |
| EP4355878A4 (en) * | 2021-06-16 | 2025-04-09 | Empirico Inc. | TREATMENT OF MTRES1-RELATED DISEASES AND DISORDERS |
| WO2023288111A2 (en) * | 2021-07-16 | 2023-01-19 | Aptah Bio, Inc. | Polynucleotide compositions and methods for gene expression regulation |
| KR20240067943A (ko) | 2021-09-24 | 2024-05-17 | 알닐람 파마슈티칼스 인코포레이티드 | 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법 |
| WO2023102188A1 (en) * | 2021-12-03 | 2023-06-08 | Quralis Corporation | Gapmer antisense oligonucleotides with modified backbone chemistries |
| WO2024091874A1 (en) * | 2022-10-25 | 2024-05-02 | Empirico Inc. | Gene therapy treatment of mtres1 related diseases and disorders |
| EP4652278A1 (en) * | 2023-01-18 | 2025-11-26 | Aptah Bio, Inc. | Polynucleotide compositions and methods for treatment of cancer |
| JP2026504808A (ja) * | 2023-01-18 | 2026-02-10 | アプター バイオ，インク． | 神経変性疾患の処置のためのポリヌクレオチド組成物および方法 |
| WO2024220930A2 (en) * | 2023-04-20 | 2024-10-24 | Adarx Pharmaceuticals, Inc. | Mapt-modulating compositions and methods of use thereof |
| WO2025188990A1 (en) * | 2024-03-08 | 2025-09-12 | Eli Lilly And Company | Mapt antisense oligonucleotide |
| US12551569B2 (en) | 2024-03-31 | 2026-02-17 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use |
| WO2026025082A1 (en) * | 2024-07-25 | 2026-01-29 | Biogen Ma Inc. | Methods for treating alzheimer's disease |
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