RU2015155332A - Олигонуклеотидные модуляторы в-клеточной cll/лимфомы 11а (bcl11а) и их применение - Google Patents
Олигонуклеотидные модуляторы в-клеточной cll/лимфомы 11а (bcl11а) и их применение Download PDFInfo
- Publication number
- RU2015155332A RU2015155332A RU2015155332A RU2015155332A RU2015155332A RU 2015155332 A RU2015155332 A RU 2015155332A RU 2015155332 A RU2015155332 A RU 2015155332A RU 2015155332 A RU2015155332 A RU 2015155332A RU 2015155332 A RU2015155332 A RU 2015155332A
- Authority
- RU
- Russia
- Prior art keywords
- seq
- antisense oligonucleotide
- units
- oligonucleotide according
- zna
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title claims 30
- 102100022976 B-cell lymphoma/leukemia 11A Human genes 0.000 title claims 4
- 101710145992 B-cell lymphoma/leukemia 11A Proteins 0.000 title 2
- 239000000074 antisense oligonucleotide Substances 0.000 claims 23
- 238000012230 antisense oligonucleotides Methods 0.000 claims 23
- 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
- 238000000034 method Methods 0.000 claims 4
- 108020004414 DNA Proteins 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 208000007056 sickle cell anemia Diseases 0.000 claims 3
- 208000018020 Sickle cell-beta-thalassemia disease syndrome Diseases 0.000 claims 2
- 206010043391 Thalassaemia beta Diseases 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 108010029485 Protein Isoforms Proteins 0.000 claims 1
- 102000001708 Protein Isoforms Human genes 0.000 claims 1
- 239000002253 acid Substances 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
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical group 0.000 claims 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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 (1)
| Publication Number | Publication Date |
|---|---|
| RU2015155332A true RU2015155332A (ru) | 2017-06-27 |
Family
ID=50780484
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2015155332A RU2015155332A (ru) | 2013-05-24 | 2014-05-26 | Олигонуклеотидные модуляторы в-клеточной cll/лимфомы 11а (bcl11а) и их применение |
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
Also Published As
| Publication number | Publication date |
|---|---|
| DK3004352T3 (da) | 2017-11-27 |
| CN105247052A (zh) | 2016-01-13 |
| MX363846B (es) | 2019-04-05 |
| US20160130582A1 (en) | 2016-05-12 |
| BR112015029327A2 (pt) | 2017-09-19 |
| MX2015015564A (es) | 2016-02-05 |
| WO2014188001A1 (en) | 2014-11-27 |
| EP3004352B1 (en) | 2017-09-27 |
| CA2907694A1 (en) | 2014-11-27 |
| HK1213595A1 (zh) | 2016-07-08 |
| EP3004352A1 (en) | 2016-04-13 |
| JP2016520310A (ja) | 2016-07-14 |
| KR20160013110A (ko) | 2016-02-03 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA92 | Acknowledgement of application withdrawn (lack of supplementary materials submitted) |
Effective date: 20180511 |