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
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- 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
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- 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)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (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 (enExample) |
| EP (1) | EP3004352B1 (enExample) |
| JP (1) | JP2016520310A (enExample) |
| KR (1) | KR20160013110A (enExample) |
| CN (1) | CN105247052A (enExample) |
| BR (1) | BR112015029327A2 (enExample) |
| CA (1) | CA2907694A1 (enExample) |
| DK (1) | DK3004352T3 (enExample) |
| HK (1) | HK1213595A1 (enExample) |
| MX (1) | MX363846B (enExample) |
| RU (1) | RU2015155332A (enExample) |
| WO (1) | WO2014188001A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| CN112007045B (zh) | 2012-07-13 | 2026-01-06 | 波涛生命科学有限公司 | 手性控制 |
| DK2872485T3 (da) | 2012-07-13 | 2021-03-08 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 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 |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| KR102423317B1 (ko) | 2014-01-16 | 2022-07-22 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
| WO2017184082A1 (en) * | 2016-04-22 | 2017-10-26 | Nanyang Technological University | A lymphocyte permeating chimeric oligonucleotide, methods and uses thereof |
| WO2018119349A1 (en) * | 2016-12-22 | 2018-06-28 | 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 |
| AU2019382087B2 (en) | 2018-11-13 | 2026-02-19 | 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 |
| KR20260044219A (ko) | 2023-07-21 | 2026-04-01 | 매로우 테라퓨틱스, 인크. | 조혈 세포 표적화 접합체 및 관련 방법 |
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 |
| US9045518B2 (en) | 2002-11-18 | 2015-06-02 | 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 |
| ES2351674T3 (es) | 2006-01-27 | 2011-02-09 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos modificados en la posición 6. |
| DK2021472T3 (da) | 2006-05-05 | 2011-09-19 | Isis Pharmaceuticals Inc | Forbindelser og fremgangsmåder til modulering af genekspression |
| CA2651453C (en) | 2006-05-11 | 2014-10-14 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
| US9398493B2 (en) | 2006-10-30 | 2016-07-19 | Nokia Technologies Oy | Method, apparatus, and system providing operator controlled mobility for user equipment |
| DK2149605T3 (da) * | 2007-03-22 | 2013-09-30 | Santaris Pharma As | Korte RNA antagonist forbindelser til modulering af det ønskede mRNA |
| EP2170917B1 (en) | 2007-05-30 | 2012-06-27 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| EP2173760B2 (en) | 2007-06-08 | 2015-11-04 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| EP2176280B2 (en) | 2007-07-05 | 2015-06-24 | 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 |
| US8846639B2 (en) | 2008-04-04 | 2014-09-30 | Isis Pharmaceutical, Inc. | Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity |
| MX345116B (es) * | 2008-09-15 | 2017-01-17 | Children's Medical Center Corp | Modulacion de bcl11a para tratamiento de hemoglobinopatias. |
| US8501805B2 (en) | 2008-09-24 | 2013-08-06 | Isis Pharmaceuticals, Inc. | Substituted alpha-L-bicyclic nucleosides |
| 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 |
| WO2011115818A1 (en) | 2010-03-17 | 2011-09-22 | 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 KR KR1020157036075A patent/KR20160013110A/ko not_active Ceased
- 2014-05-26 WO PCT/EP2014/060813 patent/WO2014188001A1/en not_active Ceased
- 2014-05-26 EP EP14726003.8A patent/EP3004352B1/en active Active
- 2014-05-26 MX MX2015015564A patent/MX363846B/es active IP Right Grant
- 2014-05-26 DK DK14726003.8T patent/DK3004352T3/da active
- 2014-05-26 HK HK16101488.9A patent/HK1213595A1/zh unknown
- 2014-05-26 CN CN201480029327.XA patent/CN105247052A/zh active Pending
- 2014-05-26 JP JP2016514446A patent/JP2016520310A/ja active Pending
- 2014-05-26 US US14/892,497 patent/US20160130582A1/en not_active Abandoned
- 2014-05-26 RU RU2015155332A patent/RU2015155332A/ru not_active Application Discontinuation
- 2014-05-26 BR BR112015029327A patent/BR112015029327A2/pt not_active Application Discontinuation
- 2014-05-26 CA CA2907694A patent/CA2907694A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| WO2014188001A1 (en) | 2014-11-27 |
| DK3004352T3 (da) | 2017-11-27 |
| KR20160013110A (ko) | 2016-02-03 |
| HK1213595A1 (zh) | 2016-07-08 |
| EP3004352A1 (en) | 2016-04-13 |
| MX2015015564A (es) | 2016-02-05 |
| JP2016520310A (ja) | 2016-07-14 |
| MX363846B (es) | 2019-04-05 |
| EP3004352B1 (en) | 2017-09-27 |
| US20160130582A1 (en) | 2016-05-12 |
| BR112015029327A2 (pt) | 2017-09-19 |
| CA2907694A1 (en) | 2014-11-27 |
| CN105247052A (zh) | 2016-01-13 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA92 | Acknowledgement of application withdrawn (lack of supplementary materials submitted) |
Effective date: 20180511 |