JP2015523082A5 - - Google Patents
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- JP2015523082A5 JP2015523082A5 JP2015521573A JP2015521573A JP2015523082A5 JP 2015523082 A5 JP2015523082 A5 JP 2015523082A5 JP 2015521573 A JP2015521573 A JP 2015521573A JP 2015521573 A JP2015521573 A JP 2015521573A JP 2015523082 A5 JP2015523082 A5 JP 2015523082A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- sequence
- target rna
- change
- seq
- 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
- 108091034117 Oligonucleotide Proteins 0.000 claims 36
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 15
- 230000008859 change Effects 0.000 claims 12
- 125000003729 nucleotide group Chemical group 0.000 claims 11
- 239000011780 sodium chloride Substances 0.000 claims 11
- 239000002773 nucleotide Substances 0.000 claims 10
- 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 8
- 239000002777 nucleoside Substances 0.000 claims 8
- 210000004027 cell Anatomy 0.000 claims 7
- 108091027075 5S-rRNA precursor Proteins 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 4
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 208000035475 disorder Diseases 0.000 claims 3
- 230000037431 insertion Effects 0.000 claims 3
- 238000003780 insertion Methods 0.000 claims 3
- 229960001855 mannitol Drugs 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- 210000003097 mucus Anatomy 0.000 claims 3
- 125000003835 nucleoside group Chemical group 0.000 claims 3
- 239000002243 precursor Substances 0.000 claims 3
- 239000002342 ribonucleoside Substances 0.000 claims 3
- 201000003883 Cystic fibrosis Diseases 0.000 claims 2
- 102000016911 Deoxyribonucleases Human genes 0.000 claims 2
- 108010053770 Deoxyribonucleases Proteins 0.000 claims 2
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims 2
- 241000322338 Loeseliastrum Species 0.000 claims 2
- 229930195725 Mannitol Natural products 0.000 claims 2
- 241001465754 Metazoa Species 0.000 claims 2
- 239000002202 Polyethylene glycol Substances 0.000 claims 2
- 229920002873 Polyethylenimine Polymers 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 235000010355 mannitol Nutrition 0.000 claims 2
- 239000000594 mannitol Substances 0.000 claims 2
- 239000002105 nanoparticle Substances 0.000 claims 2
- 229920001223 polyethylene glycol Polymers 0.000 claims 2
- 150000003384 small molecules Chemical class 0.000 claims 2
- 238000001890 transfection Methods 0.000 claims 2
- 208000035143 Bacterial infection Diseases 0.000 claims 1
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 claims 1
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 claims 1
- XRHVZWWRFMCBAZ-UHFFFAOYSA-L Endothal-disodium Chemical compound [Na+].[Na+].C1CC2C(C([O-])=O)C(C(=O)[O-])C1O2 XRHVZWWRFMCBAZ-UHFFFAOYSA-L 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 101000907783 Homo sapiens Cystic fibrosis transmembrane conductance regulator Proteins 0.000 claims 1
- 229930010555 Inosine Natural products 0.000 claims 1
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims 1
- 108010067035 Pancrelipase Proteins 0.000 claims 1
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims 1
- 229930182558 Sterol Natural products 0.000 claims 1
- 239000000443 aerosol Substances 0.000 claims 1
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims 1
- 230000004075 alteration Effects 0.000 claims 1
- 208000022362 bacterial infectious disease Diseases 0.000 claims 1
- 230000003115 biocidal effect Effects 0.000 claims 1
- 230000032770 biofilm formation Effects 0.000 claims 1
- 239000002775 capsule Substances 0.000 claims 1
- 230000004700 cellular uptake Effects 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 150000001875 compounds Chemical class 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 210000001842 enterocyte Anatomy 0.000 claims 1
- 229940088598 enzyme Drugs 0.000 claims 1
- 229940124747 fibrosis therapeutics Drugs 0.000 claims 1
- 238000009472 formulation Methods 0.000 claims 1
- 102000056427 human CFTR Human genes 0.000 claims 1
- 238000009396 hybridization Methods 0.000 claims 1
- 230000006872 improvement Effects 0.000 claims 1
- 229960003786 inosine Drugs 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 210000004072 lung Anatomy 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 230000007246 mechanism Effects 0.000 claims 1
- YACKEPLHDIMKIO-UHFFFAOYSA-N methylphosphonic acid Chemical compound CP(O)(O)=O YACKEPLHDIMKIO-UHFFFAOYSA-N 0.000 claims 1
- 239000006199 nebulizer Substances 0.000 claims 1
- 229940045258 pancrelipase Drugs 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 210000002729 polyribosome Anatomy 0.000 claims 1
- -1 polysome Substances 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 238000011084 recovery Methods 0.000 claims 1
- 210000002345 respiratory system Anatomy 0.000 claims 1
- 150000003432 sterols Chemical class 0.000 claims 1
- 235000003702 sterols Nutrition 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 238000013268 sustained release Methods 0.000 claims 1
- 239000012730 sustained-release form Substances 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 229940124597 therapeutic agent Drugs 0.000 claims 1
- 230000008719 thickening Effects 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
- 239000003981 vehicle Substances 0.000 claims 1
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261670681P | 2012-07-12 | 2012-07-12 | |
| US61/670,681 | 2012-07-12 | ||
| US201261718801P | 2012-10-26 | 2012-10-26 | |
| US61/718,801 | 2012-10-26 | ||
| EP13166465 | 2013-05-03 | ||
| EP13166465.8 | 2013-05-03 | ||
| EP13172515 | 2013-06-18 | ||
| EP13172515.2 | 2013-06-18 | ||
| EP13173818 | 2013-06-26 | ||
| EP13173818.9 | 2013-06-26 | ||
| PCT/NL2013/050534 WO2014011053A1 (en) | 2012-07-12 | 2013-07-12 | Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015523082A JP2015523082A (ja) | 2015-08-13 |
| JP2015523082A5 true JP2015523082A5 (enExample) | 2016-07-14 |
| JP6373833B2 JP6373833B2 (ja) | 2018-08-15 |
Family
ID=49916369
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015521573A Expired - Fee Related JP6373833B2 (ja) | 2012-07-12 | 2013-07-12 | 生細胞に存在する標的ｒｎａ分子の配列を変化させるためのオリゴヌクレオチド |
Country Status (23)
| Country | Link |
|---|---|
| US (2) | US9605255B2 (enExample) |
| EP (2) | EP3103872B1 (enExample) |
| JP (1) | JP6373833B2 (enExample) |
| KR (1) | KR20150037968A (enExample) |
| CN (1) | CN104640986B (enExample) |
| AU (1) | AU2013287353B2 (enExample) |
| BR (1) | BR112015000710A8 (enExample) |
| CA (1) | CA2878934A1 (enExample) |
| CY (1) | CY1117858T1 (enExample) |
| DK (2) | DK3103872T3 (enExample) |
| ES (2) | ES2584856T3 (enExample) |
| HR (1) | HRP20160854T1 (enExample) |
| HU (1) | HUE029977T2 (enExample) |
| IL (1) | IL236654B (enExample) |
| MX (1) | MX356625B (enExample) |
| NZ (1) | NZ703824A (enExample) |
| PL (1) | PL2852668T3 (enExample) |
| PT (1) | PT2852668T (enExample) |
| RS (1) | RS54944B1 (enExample) |
| RU (1) | RU2663110C2 (enExample) |
| SI (1) | SI2852668T1 (enExample) |
| SM (1) | SMT201600234B (enExample) |
| WO (1) | WO2014011053A1 (enExample) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT1993360T (pt) | 2005-12-28 | 2017-05-25 | Vertex Pharma | Formas sólidas de n-[2,4-bis(1,1-dimetiletil)-5-hidroxifenil]-1,4-di-hidro-4-oxoquinolina-3-carboxamida |
| US8563573B2 (en) | 2007-11-02 | 2013-10-22 | Vertex Pharmaceuticals Incorporated | Azaindole derivatives as CFTR modulators |
| US8802868B2 (en) | 2010-03-25 | 2014-08-12 | Vertex Pharmaceuticals Incorporated | Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide |
| RU2569678C2 (ru) | 2010-04-22 | 2015-11-27 | Вертекс Фармасьютикалз Инкорпорейтед | Способ получения циклоалкилкарбоксамидо-индольных соединений |
| WO2014011053A1 (en) | 2012-07-12 | 2014-01-16 | Proqr Therapeutics B.V. | Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell |
| PT3131582T (pt) | 2014-04-15 | 2018-10-08 | Vertex Pharma | Composições farmacêuticas para o tratamento de doenças mediadas pelo regulador de condutância transmembranar da fibrose quística |
| RU2771104C2 (ru) | 2014-05-14 | 2022-04-26 | Таргиммьюн Терапьютикс Аг | Улучшенные полиэтилениминовые полиэтиленгликолевые векторы |
| CN107250113B (zh) | 2014-10-07 | 2019-03-29 | 弗特克斯药品有限公司 | 囊性纤维化跨膜传导调节蛋白的调节剂的共晶 |
| GB201418892D0 (en) * | 2014-10-23 | 2014-12-10 | Proqr Therapeutics B V | DNA editing |
| CN107532162A (zh) * | 2014-12-12 | 2018-01-02 | 托德·M·伍尔夫 | 用于利用寡核苷酸编辑细胞中核酸的组合物和方法 |
| DK3234134T3 (da) * | 2014-12-17 | 2020-07-27 | Proqr Therapeutics Ii Bv | Målrettet rna-redigering |
| US20210401869A1 (en) * | 2015-02-20 | 2021-12-30 | Rosalind Franklin University Of Medicine And Science | Antisense Compounds Targeting Genes Associated with Cystic Fibrosis |
| GB201507926D0 (en) * | 2015-05-08 | 2015-06-24 | Proqr Therapeutics N V | Improved treatments using oligonucleotides |
| KR20180053753A (ko) | 2015-10-05 | 2018-05-23 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 삼핵산 반복 확장과 관련된 유전질환의 예방 또는 치료용 단일 가닥 안티센스 올리고뉴클레오타이드의 용도 |
| EP3985116A1 (en) | 2015-10-15 | 2022-04-20 | City of Hope | Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use 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 |
| ES2837076T3 (es) | 2016-09-01 | 2021-06-29 | Proqr Therapeutics Ii Bv | Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| JP2020508685A (ja) | 2017-03-03 | 2020-03-26 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
| 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 |
| EP3697904A1 (en) | 2017-10-17 | 2020-08-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Combination treatment for cystic fibrosis |
| CN110573184B (zh) * | 2018-02-09 | 2025-05-13 | 俄亥俄州立创新基金会 | Rna纳米结构，其制备方法和用途 |
| AU2019243946B2 (en) | 2018-03-27 | 2025-03-06 | Proqr Therapeutics Ii B.V. | Nucleic acid molecules for pseudouridylation |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| WO2020154343A1 (en) * | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| CA3159944A1 (en) | 2019-12-02 | 2021-06-10 | David HUSS | Therapeutic editing |
| CN113397996A (zh) * | 2021-07-23 | 2021-09-17 | 河南省人民医院 | 一种抗菌漱口水及其制备方法 |
| CN114917227B (zh) * | 2022-05-19 | 2023-10-31 | 中国人民解放军海军军医大学 | 依伐卡托在制备抗蜱传脑炎病毒、西尼罗病毒、黄热病毒和基孔肯雅热病毒感染药中的应用 |
| US20250014676A1 (en) | 2023-03-15 | 2025-01-09 | Shape Therapeutics Inc. | Generative sequence screening with conditional gans, diffusion models, and denoising diffusion conditional gans |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5616483A (en) * | 1992-06-11 | 1997-04-01 | Aktiebolaget Astra | Genomic DNA sequences encoding human BSSL/CEL |
| PT698092E (pt) | 1993-05-11 | 2007-10-29 | Univ North Carolina | Oligonucleótidos complementares de uma cadeia codificadora que combatem splicing aberrante e métodos para a sua utilização |
| US6207455B1 (en) | 1997-05-01 | 2001-03-27 | Lung-Ji Chang | Lentiviral vectors |
| KR100556864B1 (ko) | 1997-05-13 | 2006-03-10 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 렌티바이러스-기원 유전자 전달 벡터 |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| US6218181B1 (en) | 1998-03-18 | 2001-04-17 | The Salk Institute For Biological Studies | Retroviral packaging cell line |
| US6683173B2 (en) | 1998-04-03 | 2004-01-27 | Epoch Biosciences, Inc. | Tm leveling methods |
| DE19909769A1 (de) | 1999-03-05 | 2000-09-07 | Bundesrepublik Deutschland Let | Von SIVagm abgeleitete lentivirale Vektoren, Verfahren zu ihrer Herstellung und ihre Verwendung zur Genübertragung in Säugerzellen |
| US8314226B2 (en) * | 2004-03-29 | 2012-11-20 | The General Hospital Corporation | Oligonucleotide complex compositions and methods of use as gene alteration tools |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| WO2011097641A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
| JP5857378B2 (ja) * | 2010-04-23 | 2016-02-10 | アローヘッド リサーチ コーポレイション | ベータ−ＥＮａＣ−関連疾患を処置するための有機組成物 |
| EP2576574A2 (en) | 2010-05-28 | 2013-04-10 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
| WO2014011053A1 (en) | 2012-07-12 | 2014-01-16 | Proqr Therapeutics B.V. | Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell |
| EP2872632A1 (en) | 2012-07-12 | 2015-05-20 | ProQR Therapeutics B.V. | Exon replacement with stabilized artificial rnas |
-
2013
- 2013-07-12 WO PCT/NL2013/050534 patent/WO2014011053A1/en not_active Ceased
- 2013-07-12 US US14/414,303 patent/US9605255B2/en not_active Expired - Fee Related
- 2013-07-12 KR KR1020157002757A patent/KR20150037968A/ko not_active Ceased
- 2013-07-12 RU RU2015104763A patent/RU2663110C2/ru not_active IP Right Cessation
- 2013-07-12 EP EP16166821.5A patent/EP3103872B1/en not_active Not-in-force
- 2013-07-12 RS RS20160489A patent/RS54944B1/sr unknown
- 2013-07-12 DK DK16166821.5T patent/DK3103872T3/en active
- 2013-07-12 MX MX2015000506A patent/MX356625B/es active IP Right Grant
- 2013-07-12 JP JP2015521573A patent/JP6373833B2/ja not_active Expired - Fee Related
- 2013-07-12 HU HUE13760133A patent/HUE029977T2/en unknown
- 2013-07-12 CA CA2878934A patent/CA2878934A1/en not_active Abandoned
- 2013-07-12 DK DK13760133.2T patent/DK2852668T3/en active
- 2013-07-12 CN CN201380047636.5A patent/CN104640986B/zh not_active Expired - Fee Related
- 2013-07-12 PL PL13760133.2T patent/PL2852668T3/pl unknown
- 2013-07-12 ES ES13760133.2T patent/ES2584856T3/es active Active
- 2013-07-12 PT PT137601332T patent/PT2852668T/pt unknown
- 2013-07-12 HR HRP20160854TT patent/HRP20160854T1/hr unknown
- 2013-07-12 AU AU2013287353A patent/AU2013287353B2/en not_active Ceased
- 2013-07-12 SI SI201330214A patent/SI2852668T1/sl unknown
- 2013-07-12 NZ NZ703824A patent/NZ703824A/en not_active IP Right Cessation
- 2013-07-12 EP EP13760133.2A patent/EP2852668B1/en active Active
- 2013-07-12 BR BR112015000710A patent/BR112015000710A8/pt active Search and Examination
- 2013-07-12 ES ES16166821T patent/ES2698522T3/es active Active
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2015
- 2015-01-11 IL IL236654A patent/IL236654B/en not_active IP Right Cessation
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2016
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2017
- 2017-02-10 US US15/430,069 patent/US9994856B2/en not_active Expired - Fee Related
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