HK1213595A1 - B-细胞cll/淋巴瘤11a(bcl11a)的寡核苷酸调节剂及其用途 - Google Patents

B-细胞cll/淋巴瘤11a(bcl11a)的寡核苷酸调节剂及其用途 Download PDF

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HK1213595A1
HK1213595A1 HK16101488.9A HK16101488A HK1213595A1 HK 1213595 A1 HK1213595 A1 HK 1213595A1 HK 16101488 A HK16101488 A HK 16101488A HK 1213595 A1 HK1213595 A1 HK 1213595A1
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seq
antisense oligonucleotide
lna
bcl11a
group
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HK16101488.9A
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English (en)
Chinese (zh)
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馬伊．黑特耶恩
马伊．黑特耶恩
尼爾斯．菲斯克爾．尼爾森
尼尔斯．菲斯克尔．尼尔森
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罗氏创新中心哥本哈根有限公司
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Publication of HK1213595A1 publication Critical patent/HK1213595A1/zh

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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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 ===---===
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • 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)
HK16101488.9A 2013-05-24 2014-05-26 B-细胞cll/淋巴瘤11a(bcl11a)的寡核苷酸调节剂及其用途 HK1213595A1 (zh)

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
HK1213595A1 true HK1213595A1 (zh) 2016-07-08

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HK16101488.9A HK1213595A1 (zh) 2013-05-24 2014-05-26 B-细胞cll/淋巴瘤11a(bcl11a)的寡核苷酸调节剂及其用途

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)

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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
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AU2024299328A1 (en) 2023-07-21 2026-01-22 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods

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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
RU2015155332A (ru) 2017-06-27
MX2015015564A (es) 2016-02-05
WO2014188001A1 (en) 2014-11-27
EP3004352B1 (en) 2017-09-27
CA2907694A1 (en) 2014-11-27
EP3004352A1 (en) 2016-04-13
JP2016520310A (ja) 2016-07-14
KR20160013110A (ko) 2016-02-03

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