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

Info

Publication number
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
Authority
HK
Hong Kong
Prior art keywords
seq
antisense oligonucleotide
lna
bcl11a
group
Prior art date
Application number
HK16101488.9A
Other languages
English (en)
Chinese (zh)
Inventor
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
Original Assignee
罗氏创新中心哥本哈根有限公司
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 罗氏创新中心哥本哈根有限公司 filed Critical 罗氏创新中心哥本哈根有限公司
Publication of HK1213595A1 publication Critical patent/HK1213595A1/zh

Links

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)
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

Family

ID=50780484

Family Applications (1)

Application Number Title Priority Date Filing Date
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 (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)

* Cited by examiner, † Cited by third party
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
IN2012DN00720A (enExample) 2009-07-06 2015-06-19 Ontorii Inc
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
MX347361B (es) 2011-07-19 2017-04-12 Wave Life Sciences Ltd Metodos para la sintesis de acidos nucleicos funcionalizados.
EP2872147B1 (en) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Method for making chiral oligonucleotides
EP2872485B1 (en) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Asymmetric auxiliary group
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
CN106068325B (zh) 2014-01-16 2021-07-09 波涛生命科学有限公司 手性设计
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
MX2021005507A (es) * 2018-11-13 2021-06-18 Lipigon Pharmaceuticals Ab Oligonucleotidos de angptl4 que influyen en la regulacion del metabolismo de los acidos grasos.
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)

* Cited by examiner, † Cited by third party
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
CA2506576C (en) 2002-11-18 2018-03-06 Santaris Pharma A/S Antisense gapmer oligonucleotides
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
JP5342881B2 (ja) 2006-01-27 2013-11-13 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 ６−修飾された二環式核酸類似体
EP2505646A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of CRP
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
EP2090127A2 (en) 2006-10-30 2009-08-19 Nokia Corporation 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
CA2692579C (en) 2007-07-05 2016-05-03 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
WO2009071680A2 (en) * 2007-12-07 2009-06-11 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
EP2265627A2 (en) 2008-02-07 2010-12-29 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
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2462153B1 (en) 2009-08-06 2015-07-29 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
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
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
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
WO2013055985A1 (en) * 2011-10-12 2013-04-18 Children's Medical Center Corporation Combinatorial compositions and methods of treating hemoglobinopathies

Also Published As

Publication number Publication date
CA2907694A1 (en) 2014-11-27
MX2015015564A (es) 2016-02-05
DK3004352T3 (da) 2017-11-27
EP3004352B1 (en) 2017-09-27
JP2016520310A (ja) 2016-07-14
KR20160013110A (ko) 2016-02-03
RU2015155332A (ru) 2017-06-27
MX363846B (es) 2019-04-05
CN105247052A (zh) 2016-01-13
BR112015029327A2 (pt) 2017-09-19
WO2014188001A1 (en) 2014-11-27
EP3004352A1 (en) 2016-04-13
US20160130582A1 (en) 2016-05-12

Similar Documents

Publication Publication Date Title
EP3004352B1 (en) Oligonucleotide modulators of b-cell cll/lymphoma 11a (bcl11a) and uses thereof
US8404659B2 (en) Pharmaceutical compositions for treatment of MicroRNA related diseases
EP2563920B1 (en) Modulation of transthyretin expression
JP2021513861A (ja) Ｃａｍｋ２ｄアンチセンスオリゴヌクレオチドおよびその使用
US20180153919A1 (en) Organic compositions to treat kras-related diseases
CN105814204A (zh) 促血管生成素样3表达的调节
KR20100024399A (ko) 베타-카테닌의 조절용 ｒｎａ 길항제 화합물
WO2015023937A1 (en) Heterochromatin forming non-coding rnas
JP2014533944A (ja) Ｓｍｎ２スプライシングの調節のための化合物
KR20100105648A (ko) Ｐｉｋ３ｃａ 발현 조절용 ｒｎａ 길항제 화합물
US20110124709A1 (en) Rna antagonists targeting gli2
JPWO2020023737A5 (enExample)
WO2009043759A2 (en) Short rna antagonist compounds for the modulation of hif1alpha
US20230287035A1 (en) Pharmaceutical compositions for treatment of microrna related diseases
CN115698290A (zh) 用于治疗神经系统疾病的补体组分4抑制剂以及使用它们的相关组合物、系统和方法
CN115551519A (zh) 用于治疗神经系统疾病的补体组分c1s抑制剂以及相关的组合物、系统和使用它们的方法
EP3841220A1 (en) Microrna-134 biomarker
RU2793459C2 (ru) Композиции и способы модуляции smn2 сплайсинга у субъекта
WO2023217890A9 (en) Antisense oligonucleotides targeting cfp-elk1 intergene region
WO2025137143A2 (en) Compositions for modulating cd33 expression
HK40084486A (en) Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
EA050113B1 (ru) Антисмысловые олигонуклеотиды angptl2 и их применения
EA046329B1 (ru) Антисмысловые олигонуклеотиды camk2d и их применение
EP2205738A2 (en) Short rna antagonist compounds for the modulation of hif1alpha