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DK3004352T3 - Oligonukleotidmodulatorer af b-celle-cll/lymfom 11a (bcl11a) og anvendelser deraf - Google Patents

Oligonukleotidmodulatorer af b-celle-cll/lymfom 11a (bcl11a) og anvendelser deraf

Info

Publication number
DK3004352T3
DK3004352T3 DK14726003.8T DK14726003T DK3004352T3 DK 3004352 T3 DK3004352 T3 DK 3004352T3 DK 14726003 T DK14726003 T DK 14726003T DK 3004352 T3 DK3004352 T3 DK 3004352T3
Authority
DK
Denmark
Prior art keywords
bcl11a
lymphoma
cell
oligonucleotide modulators
modulators
Prior art date
Application number
DK14726003.8T
Other languages
English (en)
Inventor
Maj Hedtjärn
Niels Fisker Nielsen
Original Assignee
Roche Innovation Ct Copenhagen As
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Roche Innovation Ct Copenhagen As filed Critical Roche Innovation Ct Copenhagen As
Application granted granted Critical
Publication of DK3004352T3 publication Critical patent/DK3004352T3/da

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special 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)
DK14726003.8T 2013-05-24 2014-05-26 Oligonukleotidmodulatorer af b-celle-cll/lymfom 11a (bcl11a) og anvendelser deraf DK3004352T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361827484P 2013-05-24 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
DK3004352T3 true DK3004352T3 (da) 2017-11-27

Family

ID=50780484

Family Applications (1)

Application Number Title Priority Date Filing Date
DK14726003.8T DK3004352T3 (da) 2013-05-24 2014-05-26 Oligonukleotidmodulatorer af b-celle-cll/lymfom 11a (bcl11a) og anvendelser deraf

Country Status (12)

Country Link
US (1) US20160130582A1 (da)
EP (1) EP3004352B1 (da)
JP (1) JP2016520310A (da)
KR (1) KR20160013110A (da)
CN (1) CN105247052A (da)
BR (1) BR112015029327A2 (da)
CA (1) CA2907694A1 (da)
DK (1) DK3004352T3 (da)
HK (1) HK1213595A1 (da)
MX (1) MX363846B (da)
RU (1) RU2015155332A (da)
WO (1) WO2014188001A1 (da)

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RU2015151857A (ru) 2008-12-02 2019-01-15 Уэйв Лайф Сайенсес Джапан, Инк. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
US10428019B2 (en) 2010-09-24 2019-10-01 Wave Life Sciences Ltd. Chiral auxiliaries
EP3248982A1 (en) 2011-07-19 2017-11-29 Wave Life Sciences Ltd. Thiosulfonate reagents for the synthesis of functionalized nucleic acids
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
CA2879023C (en) 2012-07-13 2017-03-28 Wave Life Sciences Japan Asymmetric auxiliary group
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
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
MX2016009290A (es) 2014-01-16 2017-02-28 Wave Life Sciences Ltd Diseño quiral.
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
US12024708B2 (en) * 2018-11-13 2024-07-02 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

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US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
DK2752488T3 (da) 2002-11-18 2020-04-20 Roche Innovation Ct Copenhagen As Antisense-design
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
ES2516815T3 (es) 2006-01-27 2014-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en la posición 6
WO2007136989A2 (en) 2006-05-05 2007-11-29 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of dgat2
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WO2009071680A2 (en) * 2007-12-07 2009-06-11 Santaris Pharma A/S Rna antagonist compounds for the modulation of mcl-1
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WO2010030963A2 (en) * 2008-09-15 2010-03-18 Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
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Also Published As

Publication number Publication date
MX363846B (es) 2019-04-05
BR112015029327A2 (pt) 2017-09-19
US20160130582A1 (en) 2016-05-12
JP2016520310A (ja) 2016-07-14
WO2014188001A1 (en) 2014-11-27
CN105247052A (zh) 2016-01-13
EP3004352B1 (en) 2017-09-27
CA2907694A1 (en) 2014-11-27
EP3004352A1 (en) 2016-04-13
MX2015015564A (es) 2016-02-05
RU2015155332A (ru) 2017-06-27
HK1213595A1 (zh) 2016-07-08
KR20160013110A (ko) 2016-02-03

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