JP2017518043A5 - - Google Patents
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- Publication number
- JP2017518043A5 JP2017518043A5 JP2016564998A JP2016564998A JP2017518043A5 JP 2017518043 A5 JP2017518043 A5 JP 2017518043A5 JP 2016564998 A JP2016564998 A JP 2016564998A JP 2016564998 A JP2016564998 A JP 2016564998A JP 2017518043 A5 JP2017518043 A5 JP 2017518043A5
- Authority
- JP
- Japan
- Prior art keywords
- complex
- polynucleotide
- antisense strand
- seq
- gene
- 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
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- 108091033319 polynucleotide Proteins 0.000 claims description 24
- 239000002157 polynucleotide Substances 0.000 claims description 24
- 102000040430 polynucleotide Human genes 0.000 claims description 24
- 230000000692 anti-sense effect Effects 0.000 claims description 14
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- 229920001184 polypeptide Polymers 0.000 claims description 10
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 10
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 10
- 235000001014 amino acid Nutrition 0.000 claims description 8
- 150000001413 amino acids Chemical class 0.000 claims description 8
- 210000003169 central nervous system Anatomy 0.000 claims description 8
- 230000000295 complement effect Effects 0.000 claims description 8
- 108020004999 messenger RNA Proteins 0.000 claims description 8
- 101150081618 nox gene Proteins 0.000 claims description 8
- 239000002773 nucleotide Substances 0.000 claims description 8
- 125000003729 nucleotide group Chemical group 0.000 claims description 8
- 108090000623 proteins and genes Proteins 0.000 claims description 7
- 108010082699 NADPH Oxidase 4 Proteins 0.000 claims description 6
- 102100021872 NADPH oxidase 4 Human genes 0.000 claims description 6
- 201000010099 disease Diseases 0.000 claims description 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 6
- 238000001990 intravenous administration Methods 0.000 claims description 6
- 238000000034 method Methods 0.000 claims description 5
- 102100025621 Cytochrome b-245 heavy chain Human genes 0.000 claims description 4
- 102000004019 NADPH Oxidase 1 Human genes 0.000 claims description 4
- 108090000424 NADPH Oxidase 1 Proteins 0.000 claims description 4
- 108010082739 NADPH Oxidase 2 Proteins 0.000 claims description 4
- 108091081021 Sense strand Proteins 0.000 claims description 4
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 4
- 238000001802 infusion Methods 0.000 claims description 3
- 238000010254 subcutaneous injection Methods 0.000 claims description 3
- 239000007929 subcutaneous injection Substances 0.000 claims description 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 2
- 108091034117 Oligonucleotide Proteins 0.000 claims description 2
- 208000006011 Stroke Diseases 0.000 claims description 2
- 238000007792 addition Methods 0.000 claims description 2
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 208000014674 injury Diseases 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 208000028867 ischemia Diseases 0.000 claims description 2
- 208000004296 neuralgia Diseases 0.000 claims description 2
- 230000004770 neurodegeneration Effects 0.000 claims description 2
- 208000015122 neurodegenerative disease Diseases 0.000 claims description 2
- 208000021722 neuropathic pain Diseases 0.000 claims description 2
- 208000020016 psychiatric disease Diseases 0.000 claims description 2
- 230000001698 pyrogenic effect Effects 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 230000008733 trauma Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 3
- 239000002131 composite material Substances 0.000 claims 2
- 210000004899 c-terminal region Anatomy 0.000 claims 1
- 108091033409 CRISPR Proteins 0.000 description 1
- 238000010354 CRISPR gene editing Methods 0.000 description 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 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461987228P | 2014-05-01 | 2014-05-01 | |
| US61/987,228 | 2014-05-01 | ||
| US201462033903P | 2014-08-06 | 2014-08-06 | |
| US62/033,903 | 2014-08-06 | ||
| US201562144598P | 2015-04-08 | 2015-04-08 | |
| US62/144,598 | 2015-04-08 | ||
| PCT/US2015/028743 WO2015168521A2 (en) | 2014-05-01 | 2015-05-01 | P97-polynucleotide conjugates |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017518043A JP2017518043A (ja) | 2017-07-06 |
| JP2017518043A5 true JP2017518043A5 (enExample) | 2018-06-14 |
| JP6847664B2 JP6847664B2 (ja) | 2021-03-24 |
Family
ID=53055138
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016564998A Expired - Fee Related JP6847664B2 (ja) | 2014-05-01 | 2015-05-01 | Ｐ９７−ポリヌクレオチド複合体 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US10058619B2 (enExample) |
| EP (1) | EP3137610B1 (enExample) |
| JP (1) | JP6847664B2 (enExample) |
| CN (1) | CN106413757B (enExample) |
| AU (1) | AU2015252906C1 (enExample) |
| CA (1) | CA2943890A1 (enExample) |
| WO (1) | WO2015168521A2 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003057179A2 (en) | 2002-01-11 | 2003-07-17 | Biomarin Pharmaceutical, Inc. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
| AU2012278944B2 (en) | 2011-07-05 | 2015-09-17 | Bioasis Technologies Inc. | p97-antibody conjugates and methods of use |
| WO2014022515A1 (en) | 2012-07-31 | 2014-02-06 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
| NZ711373A (en) | 2013-03-13 | 2020-07-31 | Bioasis Technologies Inc | Fragments of p97 and uses thereof |
| JP6603227B2 (ja) | 2014-02-03 | 2019-11-06 | バイオアシス テクノロジーズ インコーポレイテッド | Ｐ９７融合タンパク質 |
| AU2015219339B2 (en) | 2014-02-19 | 2020-03-05 | Bioasis Technologies Inc. | P97-IDS fusion proteins |
| BR112020005271A2 (pt) | 2017-10-02 | 2020-09-15 | Denali Therapeutics Inc. | proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica |
| KR20210024492A (ko) * | 2018-05-27 | 2021-03-05 | 바이오아시스 테크놀로지스 인크. | 고쉐병의 치료 |
| WO2020033331A1 (en) * | 2018-08-06 | 2020-02-13 | Martner Anna | Method and compositions for treating colon cancer and breast cancer |
| US20210355468A1 (en) * | 2020-05-18 | 2021-11-18 | Bioasis Technologies, Inc. | Compositions and methods for treating lewy body dementia |
| US20210393787A1 (en) * | 2020-06-17 | 2021-12-23 | Bioasis Technologies, Inc. | Compositions and methods for treating frontotemporal dementia |
| WO2023185946A1 (zh) * | 2022-03-30 | 2023-10-05 | 苏州瑞博生物技术股份有限公司 | 一种寡核苷酸缀合物、含有该寡核苷酸缀合物的组合物及制备方法和用途 |
| CN114948802A (zh) * | 2022-04-12 | 2022-08-30 | 露乐健康科技股份有限公司 | 一种改善睡眠不足人群皮肤不良状态的护肤组合物及其制备方法及护肤品 |
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| AU2012278944B2 (en) * | 2011-07-05 | 2015-09-17 | Bioasis Technologies Inc. | p97-antibody conjugates and methods of use |
| CA2842492A1 (en) | 2011-08-05 | 2013-02-14 | Bioasis Technologies, Inc. | P97 fragments with transfer activity |
| WO2014022515A1 (en) | 2012-07-31 | 2014-02-06 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
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| NZ711373A (en) | 2013-03-13 | 2020-07-31 | Bioasis Technologies Inc | Fragments of p97 and uses thereof |
| US20150093399A1 (en) | 2013-08-28 | 2015-04-02 | Bioasis Technologies, Inc. | Cns-targeted conjugates having modified fc regions and methods of use thereof |
| JP6603227B2 (ja) | 2014-02-03 | 2019-11-06 | バイオアシス テクノロジーズ インコーポレイテッド | Ｐ９７融合タンパク質 |
| AU2015219339B2 (en) | 2014-02-19 | 2020-03-05 | Bioasis Technologies Inc. | P97-IDS fusion proteins |
-
2015
- 2015-05-01 CA CA2943890A patent/CA2943890A1/en active Pending
- 2015-05-01 WO PCT/US2015/028743 patent/WO2015168521A2/en not_active Ceased
- 2015-05-01 US US15/306,978 patent/US10058619B2/en active Active
- 2015-05-01 CN CN201580022655.1A patent/CN106413757B/zh not_active Expired - Fee Related
- 2015-05-01 AU AU2015252906A patent/AU2015252906C1/en not_active Ceased
- 2015-05-01 EP EP15721106.1A patent/EP3137610B1/en not_active Not-in-force
- 2015-05-01 JP JP2016564998A patent/JP6847664B2/ja not_active Expired - Fee Related
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