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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3004352B1 (OSRAM) |
| JP (1) | JP2016520310A (OSRAM) |
| KR (1) | KR20160013110A (OSRAM) |
| CN (1) | CN105247052A (OSRAM) |
| BR (1) | BR112015029327A2 (OSRAM) |
| CA (1) | CA2907694A1 (OSRAM) |
| DK (1) | DK3004352T3 (OSRAM) |
| HK (1) | HK1213595A1 (OSRAM) |
| MX (1) | MX363846B (OSRAM) |
| RU (1) | RU2015155332A (OSRAM) |
| WO (1) | WO2014188001A1 (OSRAM) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2744987C (en) | 2008-12-02 | 2018-01-16 | Chiralgen, Ltd. | Method for the synthesis of phosphorus atom modified nucleic acids |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| WO2013012758A1 (en) | 2011-07-19 | 2013-01-24 | Ontorii, Inc. | Methods for the synthesis of functionalized nucleic acids |
| JP6268157B2 (ja) | 2012-07-13 | 2018-01-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| KR102712879B1 (ko) | 2012-07-13 | 2024-10-04 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| 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 |
| 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 |
| EP4137572A1 (en) | 2014-01-16 | 2023-02-22 | Wave Life Sciences Ltd. | Chiral design |
| 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 |
| WO2020099478A2 (en) * | 2018-11-13 | 2020-05-22 | 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 |
| WO2025024334A1 (en) | 2023-07-21 | 2025-01-30 | 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 |
| EP1569661B1 (en) | 2002-11-18 | 2009-09-09 | Santaris Pharma A/S | Antisense design |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| US7399845B2 (en) | 2006-01-27 | 2008-07-15 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| JP5825754B2 (ja) | 2006-05-05 | 2015-12-02 | 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 | Apobの発現を調節するための化合物および方法 |
| CN101490074B (zh) | 2006-05-11 | 2013-06-26 | Isis制药公司 | 5’-修饰的双环核酸类似物 |
| US9398493B2 (en) | 2006-10-30 | 2016-07-19 | Nokia Technologies Oy | Method, apparatus, and system providing operator controlled mobility for user equipment |
| US8580756B2 (en) * | 2007-03-22 | 2013-11-12 | Santaris Pharma A/S | Short oligomer antagonist compounds for the modulation of target mRNA |
| DK2170917T3 (da) | 2007-05-30 | 2012-10-08 | Isis Pharmaceuticals Inc | N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro |
| ES2386492T3 (es) | 2007-06-08 | 2012-08-21 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos carbocíclicos |
| ATE538127T1 (de) | 2007-07-05 | 2012-01-15 | Isis Pharmaceuticals Inc | 6-disubstituierte bicyclische nukleinsäureanaloga |
| WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
| US20100323967A1 (en) * | 2007-12-07 | 2010-12-23 | 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 |
| US8530640B2 (en) | 2008-02-07 | 2013-09-10 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexitol nucleic acid analogs |
| US8846639B2 (en) | 2008-04-04 | 2014-09-30 | Isis Pharmaceutical, Inc. | Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity |
| DK2334794T3 (en) * | 2008-09-15 | 2017-02-20 | Children's Medical Center Corp | 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 |
| US8779118B2 (en) | 2010-01-11 | 2014-07-15 | 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 MX MX2015015564A patent/MX363846B/es active IP Right Grant
- 2014-05-26 BR BR112015029327A patent/BR112015029327A2/pt not_active Application Discontinuation
- 2014-05-26 CN CN201480029327.XA patent/CN105247052A/zh active Pending
- 2014-05-26 JP JP2016514446A patent/JP2016520310A/ja active Pending
- 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 CA CA2907694A patent/CA2907694A1/en not_active Abandoned
- 2014-05-26 KR KR1020157036075A patent/KR20160013110A/ko not_active Ceased
- 2014-05-26 DK DK14726003.8T patent/DK3004352T3/da active
- 2014-05-26 HK HK16101488.9A patent/HK1213595A1/zh unknown
- 2014-05-26 US US14/892,497 patent/US20160130582A1/en not_active Abandoned
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