JP2016520310A5 - - Google Patents
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- Publication number
- JP2016520310A5 JP2016520310A5 JP2016514446A JP2016514446A JP2016520310A5 JP 2016520310 A5 JP2016520310 A5 JP 2016520310A5 JP 2016514446 A JP2016514446 A JP 2016514446A JP 2016514446 A JP2016514446 A JP 2016514446A JP 2016520310 A5 JP2016520310 A5 JP 2016520310A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- antisense oligonucleotide
- lna
- oligonucleotide according
- antisense
- 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.)
- Pending
Links
- 108091034117 Oligonucleotide Proteins 0.000 claims 27
- 239000000074 antisense oligonucleotide Substances 0.000 claims 24
- 238000012230 antisense oligonucleotides Methods 0.000 claims 24
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 10
- 125000003729 nucleotide group Chemical group 0.000 claims 6
- 208000007502 anemia Diseases 0.000 claims 5
- 208000035475 disorder Diseases 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 101000903703 Homo sapiens B-cell lymphoma/leukemia 11A Proteins 0.000 claims 4
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 4
- 108020004414 DNA Proteins 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 208000007056 sickle cell anemia Diseases 0.000 claims 3
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 2
- 102100022976 B-cell lymphoma/leukemia 11A Human genes 0.000 claims 2
- 208000018020 Sickle cell-beta-thalassemia disease syndrome Diseases 0.000 claims 2
- 206010043391 Thalassaemia beta Diseases 0.000 claims 2
- 229940104302 cytosine Drugs 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 108010029485 Protein Isoforms Proteins 0.000 claims 1
- 102000001708 Protein Isoforms Human genes 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 208000005980 beta thalassemia Diseases 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 239000005547 deoxyribonucleotide Substances 0.000 claims 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 102000052096 human BCL11A Human genes 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361827484P | 2013-05-24 | 2013-05-24 | |
| US61/827,484 | 2013-05-24 | ||
| PCT/EP2014/060813 WO2014188001A1 (en) | 2013-05-24 | 2014-05-26 | Oligonucleotide modulators of b-cell cll/lymphoma 11a (bcl11a) and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016520310A JP2016520310A (ja) | 2016-07-14 |
| JP2016520310A5 true JP2016520310A5 (https=) | 2017-06-01 |
Family
ID=50780484
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016514446A Pending JP2016520310A (ja) | 2013-05-24 | 2014-05-26 | Ｂ細胞ｃｌｌ／リンパ腫１１ａ（ｂｃｌ１１ａ）のオリゴヌクレオチドモデュレーター及びその使用 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20160130582A1 (https=) |
| EP (1) | EP3004352B1 (https=) |
| JP (1) | JP2016520310A (https=) |
| KR (1) | KR20160013110A (https=) |
| CN (1) | CN105247052A (https=) |
| BR (1) | BR112015029327A2 (https=) |
| CA (1) | CA2907694A1 (https=) |
| DK (1) | DK3004352T3 (https=) |
| HK (1) | HK1213595A1 (https=) |
| MX (1) | MX363846B (https=) |
| RU (1) | RU2015155332A (https=) |
| WO (1) | WO2014188001A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| WO2017184082A1 (en) * | 2016-04-22 | 2017-10-26 | Nanyang Technological University | A lymphocyte permeating chimeric oligonucleotide, methods and uses thereof |
| US11957718B2 (en) | 2016-12-22 | 2024-04-16 | University Of Maryland, Baltimore | Methods for generating multipotent stem cells from tonsillar biopsies |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| EP3880819A2 (en) | 2018-11-13 | 2021-09-22 | Lipigon Pharmaceuticals AB | Angptl4 oligonucleotides influencing the regulation of the fatty acid metabolism |
| CN114903992B (zh) * | 2022-04-18 | 2024-01-16 | 南通大学 | 转录因子bcl11a在制备施万细胞调控药物中的应用 |
| AU2024299328A1 (en) | 2023-07-21 | 2026-01-22 | Marrow Therapeutics, Inc. | Hematopoietic cell targeting conjugates and related methods |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6617442B1 (en) | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
| WO2004046160A2 (en) | 2002-11-18 | 2004-06-03 | Santaris Pharma A/S | Amino-lna, thio-lna and alpha-l-oxy-ln |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| WO2007090071A2 (en) | 2006-01-27 | 2007-08-09 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| JP2009536222A (ja) | 2006-05-05 | 2009-10-08 | 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 | Ｐｃｓｋ９の発現を調節するための化合物および方法 |
| AU2007249349B2 (en) | 2006-05-11 | 2012-03-08 | Isis Pharmaceuticals, Inc. | 5'-Modified bicyclic nucleic acid analogs |
| CN101554074B (zh) | 2006-10-30 | 2012-08-22 | 诺基亚公司 | 为用户设备提供运营商控制的移动性的方法、设备和系统 |
| DK2149605T3 (da) * | 2007-03-22 | 2013-09-30 | Santaris Pharma As | Korte RNA antagonist forbindelser til modulering af det ønskede mRNA |
| CA2688321A1 (en) | 2007-05-30 | 2008-12-11 | 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 |
| AU2008272918B2 (en) | 2007-07-05 | 2012-09-13 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
| EP2238249A2 (en) * | 2007-12-07 | 2010-10-13 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of mcl-1 |
| WO2009090182A1 (en) | 2008-01-14 | 2009-07-23 | Santaris Pharma A/S | C4'-substituted - dna nucleotide gapmer oligonucleotides |
| WO2009100320A2 (en) | 2008-02-07 | 2009-08-13 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexitol nucleic acid analogs |
| EP2274423A2 (en) | 2008-04-04 | 2011-01-19 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity |
| US8383604B2 (en) * | 2008-09-15 | 2013-02-26 | Children's Medical Center Corporation | Modulation of BCL11A for treatment of hemoglobinopathies |
| DK2356129T3 (da) | 2008-09-24 | 2013-05-13 | Isis Pharmaceuticals Inc | Substituerede alpha-L-bicykliske nukleosider |
| US9012421B2 (en) | 2009-08-06 | 2015-04-21 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| WO2011085102A1 (en) | 2010-01-11 | 2011-07-14 | Isis Pharmaceuticals, Inc. | Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom |
| US9193752B2 (en) | 2010-03-17 | 2015-11-24 | Isis Pharmaceuticals, Inc. | 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom |
| WO2012073047A2 (en) * | 2010-12-03 | 2012-06-07 | Genome Research Limited | Compositions and methods |
| EP2648763A4 (en) | 2010-12-10 | 2014-05-14 | Alnylam Pharmaceuticals Inc | COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A |
| CN102352356B (zh) * | 2011-09-28 | 2012-12-05 | 暨南大学 | 抑制BCL11A表达和肿瘤性B细胞增殖的Bcl11a siRNA-2292及其应用 |
| CN102329794B (zh) * | 2011-09-28 | 2012-11-14 | 暨南大学 | 抑制BCL11A表达和肿瘤性B细胞增殖的Bcl11a siRNA-585及其应用 |
| US20140271657A1 (en) * | 2011-10-12 | 2014-09-18 | Children's Medical Center Corporation | Combinatorial compositions and methods of treating hemoglobinopathies |
-
2014
- 2014-05-26 CN CN201480029327.XA patent/CN105247052A/zh active Pending
- 2014-05-26 MX MX2015015564A patent/MX363846B/es active IP Right Grant
- 2014-05-26 KR KR1020157036075A patent/KR20160013110A/ko not_active Ceased
- 2014-05-26 CA CA2907694A patent/CA2907694A1/en not_active Abandoned
- 2014-05-26 EP EP14726003.8A patent/EP3004352B1/en active Active
- 2014-05-26 RU RU2015155332A patent/RU2015155332A/ru not_active Application Discontinuation
- 2014-05-26 WO PCT/EP2014/060813 patent/WO2014188001A1/en not_active Ceased
- 2014-05-26 US US14/892,497 patent/US20160130582A1/en not_active Abandoned
- 2014-05-26 DK DK14726003.8T patent/DK3004352T3/da active
- 2014-05-26 BR BR112015029327A patent/BR112015029327A2/pt not_active Application Discontinuation
- 2014-05-26 JP JP2016514446A patent/JP2016520310A/ja active Pending
- 2014-05-26 HK HK16101488.9A patent/HK1213595A1/zh unknown
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