JP2018184423A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018184423A5 JP2018184423A5 JP2018128926A JP2018128926A JP2018184423A5 JP 2018184423 A5 JP2018184423 A5 JP 2018184423A5 JP 2018128926 A JP2018128926 A JP 2018128926A JP 2018128926 A JP2018128926 A JP 2018128926A JP 2018184423 A5 JP2018184423 A5 JP 2018184423A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- human subject
- composition according
- antisense compound
- administered
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 29
- 230000000692 anti-sense effect Effects 0.000 claims 16
- 150000001875 compounds Chemical class 0.000 claims 16
- 108091034117 Oligonucleotide Proteins 0.000 claims 13
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 10
- 239000002777 nucleoside Substances 0.000 claims 9
- 239000000074 antisense oligonucleotide Substances 0.000 claims 4
- 238000012230 antisense oligonucleotides Methods 0.000 claims 4
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 4
- 125000003835 nucleoside group Chemical group 0.000 claims 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 3
- 101150081851 SMN1 gene 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
- 210000002161 motor neuron Anatomy 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 230000004083 survival effect Effects 0.000 claims 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- -1 Methoxyethyl nucleoside Chemical class 0.000 claims 1
- 208000008425 Protein deficiency Diseases 0.000 claims 1
- 102100021947 Survival motor neuron protein Human genes 0.000 claims 1
- 230000037396 body weight Effects 0.000 claims 1
- 230000002068 genetic effect Effects 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 229920002477 rna polymer Polymers 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21803109P | 2009-06-17 | 2009-06-17 | |
| US61/218,031 | 2009-06-17 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016216200A Division JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019227997A Division JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018184423A JP2018184423A (ja) | 2018-11-22 |
| JP2018184423A5 true JP2018184423A5 (OSRAM) | 2019-05-16 |
| JP6637121B2 JP6637121B2 (ja) | 2020-01-29 |
Family
ID=43356760
Family Applications (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012516315A Active JP5707396B2 (ja) | 2009-06-17 | 2010-06-17 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2015040152A Active JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2016216200A Active JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2018128926A Active JP6637121B2 (ja) | 2009-06-17 | 2018-07-06 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2019227997A Pending JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2021180965A Pending JP2022025131A (ja) | 2009-06-17 | 2021-11-05 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2023185174A Pending JP2024012416A (ja) | 2009-06-17 | 2023-10-30 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012516315A Active JP5707396B2 (ja) | 2009-06-17 | 2010-06-17 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2015040152A Active JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2016216200A Active JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019227997A Pending JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2021180965A Pending JP2022025131A (ja) | 2009-06-17 | 2021-11-05 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2023185174A Pending JP2024012416A (ja) | 2009-06-17 | 2023-10-30 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Country Status (25)
| Country | Link |
|---|---|
| US (5) | US8980853B2 (OSRAM) |
| EP (4) | EP3449926B1 (OSRAM) |
| JP (7) | JP5707396B2 (OSRAM) |
| KR (8) | KR20120093138A (OSRAM) |
| CN (5) | CN115227709A (OSRAM) |
| AU (2) | AU2010262862C1 (OSRAM) |
| CA (1) | CA2765396C (OSRAM) |
| CY (2) | CY1120807T1 (OSRAM) |
| DK (2) | DK3449926T3 (OSRAM) |
| ES (2) | ES2761614T3 (OSRAM) |
| HK (1) | HK1246648B (OSRAM) |
| HR (2) | HRP20181712T1 (OSRAM) |
| HU (2) | HUE046966T2 (OSRAM) |
| IL (2) | IL217014A (OSRAM) |
| LT (2) | LT3305302T (OSRAM) |
| MX (3) | MX361732B (OSRAM) |
| NO (1) | NO2020008I1 (OSRAM) |
| NZ (2) | NZ624712A (OSRAM) |
| PL (2) | PL3449926T3 (OSRAM) |
| PT (2) | PT3305302T (OSRAM) |
| RU (2) | RU2683772C2 (OSRAM) |
| SI (2) | SI3305302T1 (OSRAM) |
| SM (2) | SMT202000020T1 (OSRAM) |
| TR (1) | TR201816256T4 (OSRAM) |
| WO (1) | WO2010148249A1 (OSRAM) |
Families Citing this family (114)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| EP3470072A1 (en) | 2005-06-23 | 2019-04-17 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| KR20120093138A (ko) * | 2009-06-17 | 2012-08-22 | 콜드스프링하버러보러토리 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| NZ603606A (en) | 2010-05-28 | 2015-06-26 | Sarepta Therapeutics Inc | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| WO2012031243A2 (en) | 2010-09-03 | 2012-03-08 | Avi Biopharma, Inc. | dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EP2638163B1 (en) | 2010-11-12 | 2017-05-17 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| JP6478632B2 (ja) | 2011-05-05 | 2019-03-06 | サレプタ セラピューティクス， インコーポレイテッド | ペプチドオリゴヌクレオチドコンジュゲート |
| EP2714037B1 (en) | 2011-05-25 | 2016-07-13 | Université Paris Descartes | Erk inhibitors for use in treating spinal muscular atrophy |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| CA2855241A1 (en) | 2011-11-11 | 2013-05-16 | Santaris Pharma A/S | Compounds for the modulation of smn2 splicing |
| AU2012340390B2 (en) | 2011-11-18 | 2016-11-24 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| EP2788087A4 (en) * | 2011-12-06 | 2015-08-26 | Ohio State Innovation Foundation | Non-ionic, low-osmolar contrast agents for the administration of antisense oligonucleotides and the treatment of diseases |
| AU2013202595B2 (en) | 2012-03-30 | 2016-04-21 | Biogen Ma Inc. | Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| WO2014110291A1 (en) * | 2013-01-09 | 2014-07-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| WO2014113540A1 (en) * | 2013-01-16 | 2014-07-24 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| KR20150130430A (ko) | 2013-03-14 | 2015-11-23 | 아이시스 파마수티컬즈 인코포레이티드 | 타우 발현을 조절하는 조성물 및 방법 |
| US9885040B2 (en) | 2013-04-12 | 2018-02-06 | The Curators Of The University Of Missouri | SMN2 element 1 antisense compositions and methods and uses thereof |
| 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 |
| ES2750608T3 (es) | 2013-07-25 | 2020-03-26 | Exicure Inc | Construcciones esféricas a base de ácido nucleico como agentes inmunoestimulantes para uso profiláctico y terapéutico |
| US10568898B2 (en) | 2013-08-13 | 2020-02-25 | Northwestern University | Lipophilic nanoparticles for drug delivery |
| WO2015023941A1 (en) | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Oligonucleotides targeting euchromatin regions of genes |
| CA2930859C (en) | 2013-09-04 | 2022-05-03 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
| US20150197534A1 (en) | 2013-09-05 | 2015-07-16 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| US10144933B2 (en) | 2014-01-15 | 2018-12-04 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| WO2015161170A2 (en) | 2014-04-17 | 2015-10-22 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN104163871A (zh) * | 2014-08-12 | 2014-11-26 | 大连医科大学 | 抗肿瘤的特异性剪接因子及制备方法 |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| WO2016070060A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| US10517924B2 (en) | 2014-11-24 | 2019-12-31 | Northwestern University | High density lipoprotein nanoparticles for inflammation |
| WO2016149455A2 (en) | 2015-03-17 | 2016-09-22 | The General Hospital Corporation | The rna interactome of polycomb repressive complex 1 (prc1) |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| WO2016164896A2 (en) | 2015-04-10 | 2016-10-13 | Ionis Pharmaceuticals, Inc. | Modulation of smn expression |
| US10849917B2 (en) | 2015-06-01 | 2020-12-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type VII collagen |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| EP3359668A4 (en) | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF |
| EP3359685A1 (en) | 2015-10-09 | 2018-08-15 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| WO2017087486A1 (en) | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
| JP7054675B2 (ja) | 2015-12-14 | 2022-04-14 | コールド スプリング ハーバー ラボラトリー | 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 |
| CA3005090A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of liver diseases |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| WO2017106382A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of central nervous system diseases |
| CA3005245A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of alagille syndrome |
| US11083745B2 (en) | 2015-12-14 | 2021-08-10 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome |
| WO2017184529A1 (en) | 2016-04-18 | 2017-10-26 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| EP3452598A4 (en) | 2016-05-06 | 2020-04-29 | Exicure, Inc. | LIPOSOMAL SPHERICAL NUCLEIC ACID (ANS) CONSTRUCTS HAVING ANTISENSE OLIGONUCLEOTIDES (ASO) FOR THE SPECIFIC INACTIVATION OF INTERLEUKIN 17 RECEPTOR RNA |
| BR112018075667B1 (pt) | 2016-06-14 | 2024-02-20 | Biogen Ma, Inc | Métodos para separar um oligonucleotídeo alvo de uma mistura contendo o oligonucleotídeo alvo e uma impureza relacionada com o produto |
| EP3471779A4 (en) | 2016-06-16 | 2020-07-08 | Ionis Pharmaceuticals, Inc. | COMBINATIONS FOR MODULATING SMN EXPRESSION |
| KR102722183B1 (ko) | 2016-06-24 | 2024-10-24 | 바이오젠 엠에이 인코포레이티드 | 캡핑 단계 없이 티올화된 올리고뉴클레오타이드의 합성 |
| US11364304B2 (en) | 2016-08-25 | 2022-06-21 | Northwestern University | Crosslinked micellar spherical nucleic acids |
| JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| KR102520654B1 (ko) | 2017-03-10 | 2023-04-10 | 고쿠리츠켄큐카이하츠호진 고쿠리츠 세이쿠이료켄큐센타 | 안티센스 올리고뉴클레오티드 및 당원병 Ia형 예방 또는 치료용 조성물 |
| JP7679175B2 (ja) | 2017-04-20 | 2025-05-19 | エータイアー ファーマ， インコーポレイテッド | 肺の炎症を治療するための組成物および方法 |
| WO2018201090A1 (en) | 2017-04-28 | 2018-11-01 | Exicure, Inc. | Synthesis of spherical nucleic acids using lipophilic moieties |
| AU2018289930B2 (en) | 2017-06-19 | 2024-03-21 | Sumitomo Metal Mining Co., Ltd. | Near-infrared-curable ink composition, method for producing same, near-infrared-cured film, and optical shaping method |
| LT3652187T (lt) | 2017-07-10 | 2022-01-25 | Geron Corporation | Patobulintas imetelstato gamybos būdas |
| CN111499615B (zh) | 2017-08-04 | 2024-02-02 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| SMT202400071T1 (it) | 2017-08-25 | 2024-03-13 | Stoke Therapeutics Inc | Oligomeri antisenso per il trattamento di condizioni e malattie |
| WO2019075357A1 (en) * | 2017-10-12 | 2019-04-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| EP3697910A4 (en) | 2017-10-18 | 2021-07-14 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMER COMPOUNDS |
| HRP20250322T1 (hr) | 2017-10-23 | 2025-06-06 | Stoke Therapeutics, Inc. | Protusmisleni oligomeri, namijenjeni liječenju stanja i bolesti uzrokovanih raspadanjem rna posredovanim besmislenim mutacijama |
| MX2020004830A (es) | 2017-11-08 | 2020-11-11 | Avexis Inc | Medios y metodo para preparar vectores virales y usos de los mismos. |
| SG11202006528XA (en) | 2018-01-12 | 2020-08-28 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| CN120310791A (zh) | 2018-01-12 | 2025-07-15 | 百时美施贵宝公司 | 靶向α-突触核蛋白的反义寡核苷酸及其用途 |
| EP3737755B1 (en) * | 2018-01-12 | 2024-08-28 | Camena Bioscience Limited | Method for template-free geometric enzymatic nucleic acid synthesis |
| BR112020015082A2 (pt) | 2018-01-25 | 2021-01-05 | Biogen Ma, Inc. | Métodos de tratamento da atrofia muscular espinhal |
| WO2019168558A1 (en) * | 2018-02-28 | 2019-09-06 | Exicure, Inc. | Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors |
| US20210002640A1 (en) * | 2018-02-28 | 2021-01-07 | Exicure, Inc. | Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors |
| KR102398295B1 (ko) | 2018-03-09 | 2022-05-17 | 다이이찌 산쿄 가부시키가이샤 | 당원병 Ia형 치료약 |
| WO2019181946A1 (ja) | 2018-03-19 | 2019-09-26 | 国立大学法人東京医科歯科大学 | 毒性が軽減した核酸 |
| JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス，インク． | コレステリルエステル蓄積症の処置のための方法及び組成物 |
| CN120718958A (zh) | 2018-06-08 | 2025-09-30 | 诺华股份有限公司 | 用于测量药物产品效力的基于细胞的测定 |
| SG11202013088YA (en) | 2018-06-27 | 2021-02-25 | Reborna Biosciences Inc | Prophylactic or therapeutic agent for spinal muscular atrophy |
| US20210308281A1 (en) * | 2018-08-15 | 2021-10-07 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
| AU2019371814A1 (en) | 2018-10-29 | 2021-06-17 | Biogen Ma Inc. | Humanized and stabilized fc5 variants for enhancement of blood brain barrier transport |
| EP3886919A1 (en) | 2018-11-30 | 2021-10-06 | Novartis AG | Aav viral vectors and uses thereof |
| EP3890752A1 (en) | 2018-12-06 | 2021-10-13 | Biogen MA Inc. | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
| WO2020157760A1 (en) | 2019-01-31 | 2020-08-06 | Bar Ilan University | Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy |
| CN114007613A (zh) | 2019-02-05 | 2022-02-01 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| JP7603595B2 (ja) | 2019-02-06 | 2024-12-20 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングを調節するための方法および組成物 |
| WO2020191212A1 (en) | 2019-03-20 | 2020-09-24 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| US12458657B2 (en) | 2019-04-18 | 2025-11-04 | Brown University | Neurogenesis |
| WO2020227618A2 (en) | 2019-05-08 | 2020-11-12 | Biogen Ma Inc. | Convergent liquid phase syntheses of oligonucleotides |
| BR112022000870A2 (pt) | 2019-07-19 | 2022-03-22 | Biogen Ma Inc | Métodos de tratamento ou prevenção da atrofia muscular espinhal |
| WO2021030766A1 (en) * | 2019-08-15 | 2021-02-18 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
| US20220387624A1 (en) * | 2019-11-06 | 2022-12-08 | Association Institut De Myologie | Combined therapy for muscular diseases |
| RU2738093C9 (ru) * | 2019-12-23 | 2020-12-29 | Общество с ограниченной ответственностью "Гелеспон" | Способ синтеза 2'-о-(2-метоксиэтил) тиофосфатного олигонуклеотида |
| JP7446443B2 (ja) * | 2020-02-28 | 2024-03-08 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | Ｓｍｎ２を調節するための化合物及び方法 |
| CN113444722B (zh) * | 2020-03-24 | 2024-09-24 | 中国科学院脑科学与智能技术卓越创新中心 | 单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用 |
| CA3173647A1 (en) | 2020-05-11 | 2021-11-18 | Isabel AZNAREZ | Opa1 antisense oligomers for treatment of conditions and diseases |
| IL300287A (en) * | 2020-07-31 | 2023-04-01 | Ractigen Therapeutics | Combined treatment of SMA with SARNA and mRNA modulators |
| AR125239A1 (es) | 2021-04-02 | 2023-06-28 | Kumiai Chemical Industry Co | Compuesto heterocíclico y uso del mismo |
| US20250235554A1 (en) | 2021-10-25 | 2025-07-24 | Novartis Ag | Methods for improving adeno-associated virus (aav) delivery |
| CA3241399A1 (en) | 2021-12-20 | 2023-06-29 | Marco PREUSSNER | Methods and agents for increasing rbm3 expression |
| WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| WO2025035143A1 (en) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
Family Cites Families (125)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS5927900A (ja) | 1982-08-09 | 1984-02-14 | Wakunaga Seiyaku Kk | 固定化オリゴヌクレオチド |
| FR2540122B1 (fr) | 1983-01-27 | 1985-11-29 | Centre Nat Rech Scient | Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application |
| US4605735A (en) | 1983-02-14 | 1986-08-12 | Wakunaga Seiyaku Kabushiki Kaisha | Oligonucleotide derivatives |
| US4948882A (en) | 1983-02-22 | 1990-08-14 | Syngene, Inc. | Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis |
| US4824941A (en) | 1983-03-10 | 1989-04-25 | Julian Gordon | Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems |
| US4587044A (en) | 1983-09-01 | 1986-05-06 | The Johns Hopkins University | Linkage of proteins to nucleic acids |
| US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
| US5118802A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside |
| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
| US5430136A (en) | 1984-10-16 | 1995-07-04 | Chiron Corporation | Oligonucleotides having selectably cleavable and/or abasic sites |
| US5258506A (en) | 1984-10-16 | 1993-11-02 | Chiron Corporation | Photolabile reagents for incorporation into oligonucleotide chains |
| US4828979A (en) | 1984-11-08 | 1989-05-09 | Life Technologies, Inc. | Nucleotide analogs for nucleic acid labeling and detection |
| US4762779A (en) | 1985-06-13 | 1988-08-09 | Amgen Inc. | Compositions and methods for functionalizing nucleic acids |
| US5317098A (en) | 1986-03-17 | 1994-05-31 | Hiroaki Shizuya | Non-radioisotope tagging of fragments |
| JPS638396A (ja) | 1986-06-30 | 1988-01-14 | Wakunaga Pharmaceut Co Ltd | ポリ標識化オリゴヌクレオチド誘導体 |
| US4904582A (en) | 1987-06-11 | 1990-02-27 | Synthetic Genetics | Novel amphiphilic nucleic acid conjugates |
| US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
| US5525465A (en) | 1987-10-28 | 1996-06-11 | Howard Florey Institute Of Experimental Physiology And Medicine | Oligonucleotide-polyamide conjugates and methods of production and applications of the same |
| DE3738460A1 (de) | 1987-11-12 | 1989-05-24 | Max Planck Gesellschaft | Modifizierte oligonukleotide |
| US5082830A (en) | 1988-02-26 | 1992-01-21 | Enzo Biochem, Inc. | End labeled nucleotide probe |
| US5109124A (en) | 1988-06-01 | 1992-04-28 | Biogen, Inc. | Nucleic acid probe linked to a label having a terminal cysteine |
| US5262536A (en) | 1988-09-15 | 1993-11-16 | E. I. Du Pont De Nemours And Company | Reagents for the preparation of 5'-tagged oligonucleotides |
| US5512439A (en) | 1988-11-21 | 1996-04-30 | Dynal As | Oligonucleotide-linked magnetic particles and uses thereof |
| US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
| US5599923A (en) | 1989-03-06 | 1997-02-04 | Board Of Regents, University Of Tx | Texaphyrin metal complexes having improved functionalization |
| US5457183A (en) | 1989-03-06 | 1995-10-10 | Board Of Regents, The University Of Texas System | Hydroxylated texaphyrins |
| FR2645345A1 (fr) | 1989-03-31 | 1990-10-05 | Thomson Csf | Procede de modulation dirigee de la composition ou du dopage de semi-conducteurs, notamment pour la realisation de composants electroniques monolithiques de type planar, utilisation et produits correspondants |
| US5391723A (en) | 1989-05-31 | 1995-02-21 | Neorx Corporation | Oligonucleotide conjugates |
| US5256775A (en) | 1989-06-05 | 1993-10-26 | Gilead Sciences, Inc. | Exonuclease-resistant oligonucleotides |
| US4958013A (en) | 1989-06-06 | 1990-09-18 | Northwestern University | Cholesteryl modified oligonucleotides |
| US5451463A (en) | 1989-08-28 | 1995-09-19 | Clontech Laboratories, Inc. | Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides |
| US5254469A (en) | 1989-09-12 | 1993-10-19 | Eastman Kodak Company | Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures |
| US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
| DE69034150T2 (de) | 1989-10-24 | 2005-08-25 | Isis Pharmaceuticals, Inc., Carlsbad | 2'-Modifizierte Oligonukleotide |
| US5292873A (en) | 1989-11-29 | 1994-03-08 | The Research Foundation Of State University Of New York | Nucleic acids labeled with naphthoquinone probe |
| US5486603A (en) | 1990-01-08 | 1996-01-23 | Gilead Sciences, Inc. | Oligonucleotide having enhanced binding affinity |
| US6753423B1 (en) | 1990-01-11 | 2004-06-22 | Isis Pharmaceuticals, Inc. | Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals |
| US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
| US5578718A (en) | 1990-01-11 | 1996-11-26 | Isis Pharmaceuticals, Inc. | Thiol-derivatized nucleosides |
| US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
| AU7579991A (en) | 1990-02-20 | 1991-09-18 | Gilead Sciences, Inc. | Pseudonucleosides and pseudonucleotides and their polymers |
| US5214136A (en) | 1990-02-20 | 1993-05-25 | Gilead Sciences, Inc. | Anthraquinone-derivatives oligonucleotides |
| GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
| DK0455905T3 (da) | 1990-05-11 | 1998-12-07 | Microprobe Corp | Dipsticks til nukleinsyrehybridiseringsassays og fremgangsmåde til kovalent immobilisering af oligonukleotider |
| US5218105A (en) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| US5138045A (en) | 1990-07-27 | 1992-08-11 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| US5688941A (en) | 1990-07-27 | 1997-11-18 | Isis Pharmaceuticals, Inc. | Methods of making conjugated 4' desmethyl nucleoside analog compounds |
| US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
| US5245022A (en) | 1990-08-03 | 1993-09-14 | Sterling Drug, Inc. | Exonuclease resistant terminally substituted oligonucleotides |
| US5512667A (en) | 1990-08-28 | 1996-04-30 | Reed; Michael W. | Trifunctional intermediates for preparing 3'-tailed oligonucleotides |
| ATE198598T1 (de) | 1990-11-08 | 2001-01-15 | Hybridon Inc | Verbindung von mehrfachreportergruppen auf synthetischen oligonukleotiden |
| US6582908B2 (en) | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
| US5371241A (en) | 1991-07-19 | 1994-12-06 | Pharmacia P-L Biochemicals Inc. | Fluorescein labelled phosphoramidites |
| EP0538194B1 (de) | 1991-10-17 | 1997-06-04 | Novartis AG | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
| US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
| US5565552A (en) | 1992-01-21 | 1996-10-15 | Pharmacyclics, Inc. | Method of expanded porphyrin-oligonucleotide conjugate synthesis |
| US5595726A (en) | 1992-01-21 | 1997-01-21 | Pharmacyclics, Inc. | Chromophore probe for detection of nucleic acid |
| FR2687679B1 (fr) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | Oligothionucleotides. |
| EP0577558A2 (de) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte |
| US5272250A (en) | 1992-07-10 | 1993-12-21 | Spielvogel Bernard F | Boronated phosphoramidate compounds |
| US5574142A (en) | 1992-12-15 | 1996-11-12 | Microprobe Corporation | Peptide linkers for improved oligonucleotide delivery |
| ES2107205T3 (es) | 1993-03-30 | 1997-11-16 | Sanofi Sa | Analogos de nucleosidos aciclicos y secuencias oligonucleotidas que los contienen. |
| DE4311944A1 (de) | 1993-04-10 | 1994-10-13 | Degussa | Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen |
| CA2162361C (en) | 1993-05-11 | 2008-10-21 | Ryszard Kole | Antisense oligonucleotides which combat aberrant splicing and methods of using the same |
| US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
| US5446137B1 (en) | 1993-12-09 | 1998-10-06 | Behringwerke Ag | Oligonucleotides containing 4'-substituted nucleotides |
| US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
| JPH09510702A (ja) | 1994-02-28 | 1997-10-28 | アグリテック テクノロジーズ，リミテッド． | インヒビン組成物およびそれらの使用方法 |
| US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
| US5597696A (en) | 1994-07-18 | 1997-01-28 | Becton Dickinson And Company | Covalent cyanine dye oligonucleotide conjugates |
| US5580731A (en) | 1994-08-25 | 1996-12-03 | Chiron Corporation | N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith |
| US5597909A (en) | 1994-08-25 | 1997-01-28 | Chiron Corporation | Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use |
| EP0708178A1 (en) * | 1994-10-19 | 1996-04-24 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy |
| US5792747A (en) | 1995-01-24 | 1998-08-11 | The Administrators Of The Tulane Educational Fund | Highly potent agonists of growth hormone releasing hormone |
| US7034009B2 (en) | 1995-10-26 | 2006-04-25 | Sirna Therapeutics, Inc. | Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R) |
| EP1108724B1 (en) | 1996-01-16 | 2007-09-19 | Sirna Therpeutics, Inc. | Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules |
| US20030228597A1 (en) | 1998-04-13 | 2003-12-11 | Cowsert Lex M. | Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation |
| US6210892B1 (en) | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
| US6214986B1 (en) | 1998-10-07 | 2001-04-10 | Isis Pharmaceuticals, Inc. | Antisense modulation of bcl-x expression |
| US6172216B1 (en) | 1998-10-07 | 2001-01-09 | Isis Pharmaceuticals Inc. | Antisense modulation of BCL-X expression |
| AU776362B2 (en) | 1999-05-04 | 2004-09-09 | Roche Innovation Center Copenhagen A/S | L-ribo-LNA analogues |
| US6656730B1 (en) | 1999-06-15 | 2003-12-02 | Isis Pharmaceuticals, Inc. | Oligonucleotides conjugated to protein-binding drugs |
| US6649411B2 (en) | 1999-07-30 | 2003-11-18 | The United States Of America As Represented By The Department Of Health And Human Services | Methods of inhibiting cancer cells with ADNF III antisense oligonucleotides |
| US6770633B1 (en) | 1999-10-26 | 2004-08-03 | Immusol, Inc. | Ribozyme therapy for the treatment of proliferative skin and eye diseases |
| US20050020525A1 (en) | 2002-02-20 | 2005-01-27 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| US6962906B2 (en) | 2000-03-14 | 2005-11-08 | Active Motif | Oligonucleotide analogues, methods of synthesis and methods of use |
| AU2001259706A1 (en) | 2000-05-09 | 2001-11-20 | Reliable Biopharmaceutical, Inc. | Polymeric compounds useful as prodrugs |
| CA2437942C (en) | 2000-11-09 | 2013-06-11 | Cold Spring Harbor Laboratory | Chimeric molecules to modulate gene expression |
| US6376508B1 (en) | 2000-12-13 | 2002-04-23 | Academia Sinica | Treatments for spinal muscular atrophy |
| EP1499627A2 (en) | 2001-07-03 | 2005-01-26 | ISIS Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| GB0219143D0 (en) | 2002-08-16 | 2002-09-25 | Univ Leicester | Modified tailed oligonucleotides |
| AU2003291753B2 (en) | 2002-11-05 | 2010-07-08 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| EP2213738B1 (en) | 2002-11-14 | 2012-10-10 | Dharmacon, Inc. | siRNA molecules targeting Bcl-2 |
| JP2004344072A (ja) * | 2003-05-22 | 2004-12-09 | Japan Science & Technology Agency | Ｓｍｎ遺伝子のスプライシング調節エレメント |
| WO2004113867A2 (en) | 2003-06-16 | 2004-12-29 | University Of Massachusetts | Exon analysis |
| ATE555118T1 (de) | 2003-08-28 | 2012-05-15 | Takeshi Imanishi | Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung |
| US20050074801A1 (en) | 2003-09-09 | 2005-04-07 | Monia Brett P. | Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry |
| JP5379347B2 (ja) | 2003-09-18 | 2013-12-25 | アイシス ファーマシューティカルズ， インコーポレーテッド | ４’−チオヌクレオシドおよびオリゴマー化合物 |
| GB0425625D0 (en) | 2004-11-22 | 2004-12-22 | Royal College Of Surgeons Ie | Treatment of disease |
| US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| US7879992B2 (en) | 2005-01-31 | 2011-02-01 | Isis Pharmaceuticals, Inc. | Modification of MyD88 splicing using modified oligonucleotides |
| EP3470072A1 (en) | 2005-06-23 | 2019-04-17 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing |
| JP4223027B2 (ja) | 2005-06-30 | 2009-02-12 | シャープ株式会社 | 画像形成装置及び秘匿データ送信方法 |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| EP2535413A3 (en) | 2005-11-10 | 2016-04-13 | Roche Innovation Center Copenhagen A/S | Splice switching oligomers for the TNF superfamily receptors and their use in treatment of disease |
| WO2007089611A2 (en) * | 2006-01-26 | 2007-08-09 | Isis Pharmaceuticals Inc. | Compositions and their uses directed to huntingtin |
| EP1984381B1 (en) | 2006-01-27 | 2010-09-29 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| JP5213723B2 (ja) | 2006-01-27 | 2013-06-19 | アイシス ファーマシューティカルズ， インコーポレーテッド | マイクロｒｎａの調節に使用するためのオリゴマー化合物及び組成物 |
| ES2389737T3 (es) | 2006-05-11 | 2012-10-31 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos modificados en 5' |
| EP2125852B1 (en) | 2007-02-15 | 2016-04-06 | Ionis Pharmaceuticals, Inc. | 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom |
| US8278425B2 (en) | 2007-05-30 | 2012-10-02 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| DK2173760T4 (en) | 2007-06-08 | 2016-02-08 | Isis Pharmaceuticals Inc | Carbocyclic bicyclic nukleinsyreanaloge |
| US8278283B2 (en) | 2007-07-05 | 2012-10-02 | Isis Pharmaceuticals, Inc. | 6-disubstituted or unsaturated bicyclic nucleic acid analogs |
| WO2009120700A2 (en) | 2008-03-26 | 2009-10-01 | Families Of Spinal Muscular Atrophy | Inhibition of dcps |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| WO2010123594A2 (en) | 2009-01-15 | 2010-10-28 | Children's Medical Center Corporation | Device for filtration of fluids there through and accompanying method |
| WO2010091308A2 (en) * | 2009-02-06 | 2010-08-12 | Isis Pharmaceuticals, Inc. | Oligomeric compounds and methods |
| US20120149757A1 (en) | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
| KR20120093138A (ko) | 2009-06-17 | 2012-08-22 | 콜드스프링하버러보러토리 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| WO2011032109A1 (en) | 2009-09-11 | 2011-03-17 | Sma Foundation | Biomarkers for spinal muscular atrophy |
| US8183002B2 (en) | 2009-12-03 | 2012-05-22 | Abbott Laboratories | Autoantibody enhanced immunoassays and kits |
| US10653713B2 (en) | 2010-10-06 | 2020-05-19 | Medtronic, Inc. | Methods for distributing agents to areas of brain |
| AU2012282825B2 (en) | 2011-07-13 | 2016-05-26 | Merck Sharp & Dohme Corp. | Method for detection of amyloid beta oligomers in a fluid sample and uses thereof |
| WO2014110291A1 (en) | 2013-01-09 | 2014-07-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| US20140367278A1 (en) | 2013-06-12 | 2014-12-18 | PharmOptima, LLC | Methods for Detecting Survival Motor Neuron (SMN) Protein in Whole Blood or Cerebral Spinal Fluid |
-
2010
- 2010-06-17 KR KR1020127001244A patent/KR20120093138A/ko not_active Ceased
- 2010-06-17 TR TR2018/16256T patent/TR201816256T4/tr unknown
- 2010-06-17 RU RU2015139553A patent/RU2683772C2/ru active
- 2010-06-17 DK DK18185820T patent/DK3449926T3/da active
- 2010-06-17 KR KR1020197009268A patent/KR20190037378A/ko not_active Ceased
- 2010-06-17 CN CN202210540428.XA patent/CN115227709A/zh active Pending
- 2010-06-17 SI SI201031784T patent/SI3305302T1/sl unknown
- 2010-06-17 AU AU2010262862A patent/AU2010262862C1/en active Active
- 2010-06-17 KR KR1020247007557A patent/KR20240036132A/ko not_active Ceased
- 2010-06-17 MX MX2015008433A patent/MX361732B/es unknown
- 2010-06-17 MX MX2011014004A patent/MX2011014004A/es active IP Right Grant
- 2010-06-17 EP EP18185820.0A patent/EP3449926B1/en active Active
- 2010-06-17 EP EP17203913.3A patent/EP3305302B1/en active Active
- 2010-06-17 KR KR1020207012412A patent/KR20200047790A/ko not_active Ceased
- 2010-06-17 SM SM20200020T patent/SMT202000020T1/it unknown
- 2010-06-17 CN CN2010800368070A patent/CN102665731A/zh active Pending
- 2010-06-17 SM SM20180587T patent/SMT201800587T1/it unknown
- 2010-06-17 WO PCT/US2010/039077 patent/WO2010148249A1/en not_active Ceased
- 2010-06-17 PL PL18185820T patent/PL3449926T3/pl unknown
- 2010-06-17 CN CN201710003678.9A patent/CN106983768B/zh active Active
- 2010-06-17 KR KR1020197003809A patent/KR101965868B1/ko active Active
- 2010-06-17 EP EP10790221.5A patent/EP2442816A4/en not_active Withdrawn
- 2010-06-17 CN CN202310217516.0A patent/CN116440150A/zh active Pending
- 2010-06-17 PT PT17203913T patent/PT3305302T/pt unknown
- 2010-06-17 ES ES18185820T patent/ES2761614T3/es active Active
- 2010-06-17 KR KR1020227019810A patent/KR20220084437A/ko not_active Ceased
- 2010-06-17 ES ES17203913T patent/ES2699827T3/es active Active
- 2010-06-17 EP EP19201392.8A patent/EP3643783A1/en active Pending
- 2010-06-17 LT LTEP17203913.3T patent/LT3305302T/lt unknown
- 2010-06-17 KR KR1020177011876A patent/KR20170054537A/ko not_active Withdrawn
- 2010-06-17 US US13/380,021 patent/US8980853B2/en active Active
- 2010-06-17 NZ NZ624712A patent/NZ624712A/en unknown
- 2010-06-17 HU HUE18185820A patent/HUE046966T2/hu unknown
- 2010-06-17 LT LTEP18185820.0T patent/LT3449926T/lt unknown
- 2010-06-17 RU RU2012101491A patent/RU2566724C9/ru active
- 2010-06-17 PL PL17203913T patent/PL3305302T3/pl unknown
- 2010-06-17 KR KR1020217014192A patent/KR20210057223A/ko not_active Ceased
- 2010-06-17 CA CA2765396A patent/CA2765396C/en active Active
- 2010-06-17 SI SI201031946T patent/SI3449926T1/sl unknown
- 2010-06-17 JP JP2012516315A patent/JP5707396B2/ja active Active
- 2010-06-17 NZ NZ597071A patent/NZ597071A/en unknown
- 2010-06-17 DK DK17203913.3T patent/DK3305302T3/en active
- 2010-06-17 PT PT181858200T patent/PT3449926T/pt unknown
- 2010-06-17 HU HUE17203913A patent/HUE039741T2/hu unknown
- 2010-06-17 CN CN202210541466.7A patent/CN115227710A/zh active Pending
-
2011
- 2011-12-15 IL IL217014A patent/IL217014A/en active Protection Beyond IP Right Term
- 2011-12-16 MX MX2018015635A patent/MX2018015635A/es unknown
-
2015
- 2015-02-09 US US14/617,388 patent/US9717750B2/en active Active
- 2015-03-02 JP JP2015040152A patent/JP6064139B2/ja active Active
-
2016
- 2016-01-21 AU AU2016200344A patent/AU2016200344B2/en active Active
- 2016-04-13 IL IL245096A patent/IL245096B/en active Protection Beyond IP Right Term
- 2016-11-04 JP JP2016216200A patent/JP6370860B2/ja active Active
-
2018
- 2018-02-09 US US15/892,560 patent/US20190030058A1/en not_active Abandoned
- 2018-05-10 HK HK18106100.4A patent/HK1246648B/en unknown
- 2018-07-06 JP JP2018128926A patent/JP6637121B2/ja active Active
- 2018-10-19 HR HRP20181712TT patent/HRP20181712T1/hr unknown
- 2018-11-01 CY CY181101134T patent/CY1120807T1/el unknown
-
2019
- 2019-10-31 HR HRP20191978TT patent/HRP20191978T1/hr unknown
- 2019-12-10 US US16/708,739 patent/US20200376018A1/en not_active Abandoned
- 2019-12-18 JP JP2019227997A patent/JP2020073498A/ja active Pending
- 2019-12-19 CY CY20191101339T patent/CY1122687T1/el unknown
-
2020
- 2020-04-01 NO NO2020008C patent/NO2020008I1/no unknown
-
2021
- 2021-11-05 JP JP2021180965A patent/JP2022025131A/ja active Pending
-
2023
- 2023-10-25 US US18/494,445 patent/US20240238326A1/en not_active Abandoned
- 2023-10-30 JP JP2023185174A patent/JP2024012416A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018184423A5 (OSRAM) | ||
| JP6924242B2 (ja) | Ｃ９ｏｒｆ７２発現を調節するための組成物 | |
| JP7354342B2 (ja) | タウ発現を調節するための組成物 | |
| JP7198298B2 (ja) | Ｓｏｄ－１発現を調節するための組成物 | |
| RU2566724C2 (ru) | Композиции и способы модуляции smn2 сплайсинга у субъекта | |
| KR102055331B1 (ko) | B형 간염 바이러스(hbv) 발현 조절 | |
| JP6902869B2 (ja) | アタキシン２の発現を調節するための組成物 | |
| CN105517556B (zh) | 前激肽释放酶(pkk)表达的调节 | |
| JP2011504362A5 (OSRAM) | ||
| JP2016513976A5 (OSRAM) | ||
| JP2019525918A5 (OSRAM) | ||
| CN113905744A (zh) | 经修饰的间隙子寡核苷酸及其使用方法 | |
| Bizot et al. | Current status of antisense oligonucleotide-based therapy in neuromuscular disorders | |
| JP2021524277A (ja) | Ｒｔｅｌ１発現の調節用のオリゴヌクレオチド | |
| CN114786683B (zh) | 一种siRNA、药物组合物以及使用其治疗糖尿病的方法 | |
| WO2017043639A1 (ja) | キメラデコイ | |
| JP2008541729A5 (OSRAM) | ||
| US20220002726A1 (en) | Methods of safe administration of an irf5 antisense oligonucleotide | |
| JP2024506371A (ja) | 低酸素症及び虚血関連障害を処置する薬剤、組成物及び方法 | |
| RU2019108578A (ru) | Композиции и способы модуляции smn2 сплайсинга у субъекта | |
| KR20250076429A (ko) | 금속 나노 입자-핵산 결합체를 기반으로 하는 운반체를 포함하는 대사성 질환 치료제 | |
| CN121152878A (zh) | 用于抑制补体因子B (CFB)表达的RNAi剂、其药物组合物和使用方法 |