JP2018150344A5 - - Google Patents
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- JP2018150344A5 JP2018150344A5 JP2018095820A JP2018095820A JP2018150344A5 JP 2018150344 A5 JP2018150344 A5 JP 2018150344A5 JP 2018095820 A JP2018095820 A JP 2018095820A JP 2018095820 A JP2018095820 A JP 2018095820A JP 2018150344 A5 JP2018150344 A5 JP 2018150344A5
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- 108091034117 Oligonucleotide Proteins 0.000 claims description 114
- 150000001875 compounds Chemical class 0.000 claims description 88
- 238000000034 method Methods 0.000 claims description 83
- 239000002777 nucleoside Substances 0.000 claims description 83
- 125000003835 nucleoside group Chemical group 0.000 claims description 59
- 241001465754 Metazoa Species 0.000 claims description 56
- 102000018658 Myotonin-Protein Kinase Human genes 0.000 claims description 51
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 claims description 51
- 230000000295 complement effect Effects 0.000 claims description 32
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 24
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 22
- 206010061533 Myotonia Diseases 0.000 claims description 16
- 239000000203 mixture Substances 0.000 claims description 13
- 206010068871 Myotonic dystrophy Diseases 0.000 claims description 12
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 9
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 6
- 125000002619 bicyclic group Chemical group 0.000 claims description 5
- 230000001419 dependent effect Effects 0.000 claims description 5
- 101000583839 Homo sapiens Muscleblind-like protein 1 Proteins 0.000 claims description 2
- 102100030965 Muscleblind-like protein 1 Human genes 0.000 claims description 2
- 239000003085 diluting agent Substances 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 201000010099 disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 208000024891 symptom Diseases 0.000 claims 4
- 159000000000 sodium salts Chemical class 0.000 claims 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 30
- 239000000074 antisense oligonucleotide Substances 0.000 description 29
- 238000012230 antisense oligonucleotides Methods 0.000 description 29
- 239000003795 chemical substances by application Substances 0.000 description 12
- 102100022437 Myotonin-protein kinase Human genes 0.000 description 7
- 230000003252 repetitive effect Effects 0.000 description 6
- 108010083644 Ribonucleases Proteins 0.000 description 4
- 102000006382 Ribonucleases Human genes 0.000 description 4
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 4
- 108020000948 Antisense Oligonucleotides Proteins 0.000 description 3
- 101150086792 CLCN1 gene Proteins 0.000 description 3
- 101100107064 Drosophila melanogaster Zasp52 gene Proteins 0.000 description 3
- 108091026890 Coding region Proteins 0.000 description 2
- 229940104302 cytosine Drugs 0.000 description 2
- 238000001990 intravenous administration Methods 0.000 description 2
- -1 intron Proteins 0.000 description 2
- 210000003205 muscle Anatomy 0.000 description 2
- 238000007911 parenteral administration Methods 0.000 description 2
- 239000008194 pharmaceutical composition Substances 0.000 description 2
- 238000007920 subcutaneous administration Methods 0.000 description 2
- 238000007910 systemic administration Methods 0.000 description 2
- 108020005345 3' Untranslated Regions Proteins 0.000 description 1
- 108020003589 5' Untranslated Regions Proteins 0.000 description 1
- 101000659173 Homo sapiens Tetratricopeptide repeat protein 39B Proteins 0.000 description 1
- 102100025290 Ribonuclease H1 Human genes 0.000 description 1
- 102100036125 Tetratricopeptide repeat protein 39B Human genes 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000007913 intrathecal administration Methods 0.000 description 1
- 238000007914 intraventricular administration Methods 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 108020004707 nucleic acids Proteins 0.000 description 1
- 102000039446 nucleic acids Human genes 0.000 description 1
- 150000007523 nucleic acids Chemical class 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 108010052833 ribonuclease HI Proteins 0.000 description 1
- 150000003839 salts Chemical class 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US36577510P | 2010-07-19 | 2010-07-19 | |
| US36576210P | 2010-07-19 | 2010-07-19 | |
| US61/365,762 | 2010-07-19 | ||
| US61/365,775 | 2010-07-19 | ||
| US201161478021P | 2011-04-21 | 2011-04-21 | |
| US61/478,021 | 2011-04-21 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016147984A Division JP6343308B2 (ja) | 2010-07-19 | 2016-07-28 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020078906A Division JP2020183379A (ja) | 2010-07-19 | 2020-04-28 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018150344A JP2018150344A (ja) | 2018-09-27 |
| JP2018150344A5 true JP2018150344A5 (enExample) | 2019-01-17 |
| JP6698740B2 JP6698740B2 (ja) | 2020-05-27 |
Family
ID=45497419
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013520822A Active JP6059141B2 (ja) | 2010-07-19 | 2011-07-19 | 核内繋留ｒｎａの調節 |
| JP2013520815A Active JP5981428B2 (ja) | 2010-07-19 | 2011-07-19 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
| JP2016147984A Active JP6343308B2 (ja) | 2010-07-19 | 2016-07-28 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
| JP2018095820A Active JP6698740B2 (ja) | 2010-07-19 | 2018-05-18 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
| JP2020078906A Pending JP2020183379A (ja) | 2010-07-19 | 2020-04-28 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013520822A Active JP6059141B2 (ja) | 2010-07-19 | 2011-07-19 | 核内繋留ｒｎａの調節 |
| JP2013520815A Active JP5981428B2 (ja) | 2010-07-19 | 2011-07-19 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
| JP2016147984A Active JP6343308B2 (ja) | 2010-07-19 | 2016-07-28 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020078906A Pending JP2020183379A (ja) | 2010-07-19 | 2020-04-28 | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
Country Status (13)
| Country | Link |
|---|---|
| US (7) | US20130237585A1 (enExample) |
| EP (5) | EP3489360A3 (enExample) |
| JP (5) | JP6059141B2 (enExample) |
| KR (2) | KR20180105730A (enExample) |
| CN (2) | CN106434648A (enExample) |
| AU (6) | AU2011282243B2 (enExample) |
| CA (2) | CA2805791A1 (enExample) |
| DK (1) | DK3031920T3 (enExample) |
| ES (1) | ES2756326T3 (enExample) |
| HR (1) | HRP20191753T1 (enExample) |
| IL (2) | IL223962B (enExample) |
| PL (1) | PL3031920T3 (enExample) |
| WO (2) | WO2012012467A2 (enExample) |
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| DK2638163T3 (en) | 2010-11-12 | 2017-07-24 | Massachusetts Gen Hospital | POLYCOMB-ASSOCIATED NON-CODING RNAs |
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2011
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- 2011-07-19 US US13/811,181 patent/US20130237585A1/en not_active Abandoned
- 2011-07-19 EP EP18199910.3A patent/EP3489360A3/en active Pending
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- 2011-07-19 WO PCT/US2011/044583 patent/WO2012012467A2/en not_active Ceased
- 2011-07-19 AU AU2011282243A patent/AU2011282243B2/en not_active Expired - Fee Related
- 2011-07-19 EP EP20110810291 patent/EP2595663A4/en not_active Withdrawn
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- 2011-07-19 EP EP16153949.9A patent/EP3031920B1/en not_active Revoked
- 2011-07-19 CN CN201180035573.2A patent/CN103167883B/zh active Active
- 2011-07-19 DK DK16153949.9T patent/DK3031920T3/da active
- 2011-07-19 US US13/810,879 patent/US20130225659A1/en not_active Abandoned
- 2011-07-19 PL PL16153949T patent/PL3031920T3/pl unknown
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2012
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