JP2009524419A - Ｌｎａ修飾したホスホロチオール化オリゴヌクレオチド - Google Patents

Ｌｎａ修飾したホスホロチオール化オリゴヌクレオチド Download PDF

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Publication number
JP2009524419A
JP2009524419A JP2008551732A JP2008551732A JP2009524419A JP 2009524419 A JP2009524419 A JP 2009524419A JP 2008551732 A JP2008551732 A JP 2008551732A JP 2008551732 A JP2008551732 A JP 2008551732A JP 2009524419 A JP2009524419 A JP 2009524419A
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Japan
Prior art keywords
lna
oligonucleotide
mixed sequence
rna
sequence oligonucleotide
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Pending
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JP2008551732A
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Japanese (ja)
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JP2009524419A5 (https=
Inventor
ヨアヒム・エルメン
ヘンリク・フリゼンルン・ハンセン
ヘンリク・エルム
トレルス・コッホ
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Roche Innovation Center Copenhagen AS
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Santaris Pharma AS
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2008551732A 2006-01-27 2007-01-29 Ｌｎａ修飾したホスホロチオール化オリゴヌクレオチド Pending JP2009524419A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US76292006P 2006-01-27 2006-01-27
PCT/EP2007/000741 WO2007085485A2 (en) 2006-01-27 2007-01-29 Lna modified phosphorothiolated oligonucleotides

Publications (2)

Publication Number Publication Date
JP2009524419A true JP2009524419A (ja) 2009-07-02
JP2009524419A5 JP2009524419A5 (https=) 2010-03-11

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JP2008551732A Pending JP2009524419A (ja) 2006-01-27 2007-01-29 Ｌｎａ修飾したホスホロチオール化オリゴヌクレオチド

Country Status (7)

Country Link
US (1) US20090176977A1 (https=)
EP (1) EP1984382B1 (https=)
JP (1) JP2009524419A (https=)
AU (1) AU2007209481B2 (https=)
CA (1) CA2638837A1 (https=)
DK (1) DK1984382T3 (https=)
WO (1) WO2007085485A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018504380A (ja) * 2014-12-18 2018-02-15 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｒｅｖｅｒｓｉｒ（商標）化合物

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE443765T1 (de) 2003-03-21 2009-10-15 Santaris Pharma As Analoga kurzer interferierender rna (sirna)
WO2005073378A1 (en) * 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna
EP2195428B1 (en) 2007-09-19 2013-12-11 Applied Biosystems, LLC SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
WO2009043759A2 (en) * 2007-10-04 2009-04-09 Santaris Pharma A/S Short rna antagonist compounds for the modulation of hif1alpha
ES2609655T3 (es) * 2009-05-06 2017-04-21 Curna, Inc. Tratamiento de enfermedades relacionadas con tristetraprolina (TTP) mediante inhibición de transcrito antisentido natural para TTP
US20150025122A1 (en) 2009-10-12 2015-01-22 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
EP2542678B1 (en) 2010-03-04 2017-04-12 InteRNA Technologies B.V. A MiRNA MOLECULE DEFINED BY ITS SOURCE AND ITS THERAPEUTIC USES IN CANCER ASSOCIATED WITH EMT
EP2591106A1 (en) 2010-07-06 2013-05-15 InteRNA Technologies B.V. Mirna and its diagnostic and therapeutic uses in diseases or conditions associated with melanoma, or in diseases or conditions associated with activated braf pathway
EP2474617A1 (en) 2011-01-11 2012-07-11 InteRNA Technologies BV Mir for treating neo-angiogenesis
CA2855362A1 (en) * 2011-11-11 2013-05-16 Santaris Pharma A/S Compounds for the modulation of beta-catenin expression and uses thereof
EP3369818B1 (en) 2011-12-22 2021-06-09 InteRNA Technologies B.V. Mirna for treating head and neck cancer
EP2917348A1 (en) 2012-11-06 2015-09-16 InteRNA Technologies B.V. Combination for use in treating diseases or conditions associated with melanoma, or treating diseases or conditions associated with activated b-raf pathway
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
WO2014140856A2 (en) 2013-03-15 2014-09-18 Graham Lord Mir-142 and antagonists thereof for treating disease
HRP20200250T1 (hr) * 2013-08-08 2020-05-29 The Scripps Research Institute Metoda site-specifično enzimsko obilježavanje nukleinskih kiselina in vitro ugradnjom neprirodnih nukleotida
EP3181698A1 (en) 2015-12-16 2017-06-21 European Molecular Biology Laboratory (EMBL) Microrna mir-142 as stem cell marker
JP7325341B2 (ja) 2017-07-11 2023-08-14 シンソークス，インク． 非天然ヌクレオチドの組み込み及びその方法
KR20240141853A (ko) 2017-08-03 2024-09-27 신톡스, 인크. 자가면역 질환의 치료를 위한 사이토카인 접합체
CN111566212A (zh) 2017-11-03 2020-08-21 因特尔纳技术有限公司 miRNA分子，等同物，安塔够妙或其来源用于治疗和/或诊断与神经元缺陷相关的病症和/或疾病或用于神经元生成和/或再生
JP2021513508A (ja) 2018-02-12 2021-05-27 インテアールエヌエー テクノロジーズ ビー．ヴイ．ＩｎｔｅＲＮＡ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． 抗がんマイクロｒｎａ及びその脂質製剤
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
CN112534055A (zh) 2018-07-13 2021-03-19 豪夫迈·罗氏有限公司 用于调节rtel1表达的寡核苷酸
EP4122943B1 (en) * 2018-07-31 2024-05-29 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorotrithioate internucleoside linkage
EP3923974A4 (en) 2019-02-06 2023-02-08 Synthorx, Inc. IL-2 CONJUGATES AND METHODS OF USE THEREOF
EP3947680A2 (en) 2019-04-03 2022-02-09 Bristol-Myers Squibb Company Angptl2 antisense oligonucleotides and uses thereof
WO2021122735A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of sept9 inhibitors for treating hepatitis b virus infection
WO2021122921A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of cops3 inhibitors for treating hepatitis b virus infection
WO2021122869A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of scamp3 inhibitors for treating hepatitis b virus infection
JP2023506954A (ja) 2019-12-19 2023-02-20 エフ． ホフマン－ラ ロシュ エージー． Ｂ型肝炎ウイルス感染を処置するためのｓａｒａｆ阻害剤の使用
EP4077669A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of sbds inhibitors for treating hepatitis b virus infection
EP4081217A1 (en) 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
MX2022007908A (es) 2019-12-24 2022-07-21 Hoffmann La Roche Combinacion farmaceutica de un oligonucleotido terapeutico que actua sobre hbv y un agonista de tlr7 para el tratamiento de hbv.
JP2023525770A (ja) 2020-05-11 2023-06-19 ジェネンテック， インコーポレイテッド 神経学的疾患を処置するための補体ｃ４阻害剤および関連する組成物、ならびにそれを使用するシステムおよび方法
EP4149486A1 (en) 2020-05-11 2023-03-22 Genentech, Inc. Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
WO2021231210A1 (en) 2020-05-11 2021-11-18 Genentech, Inc. Complement component c1r inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
CN116157522A (zh) 2020-08-21 2023-05-23 豪夫迈·罗氏有限公司 A1cf抑制剂用于治疗乙型肝炎病毒感染的用途
WO2022122613A1 (en) * 2020-12-08 2022-06-16 F. Hoffmann-La Roche Ag Novel synthesis of phosphorodithioate oligonucleotides
KR20240101580A9 (ko) 2021-11-11 2025-12-10 에프. 호프만-라 로슈 아게 Hbv 치료를 위한 약학 조합물
WO2023111210A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Combination of oligonucleotides for modulating rtel1 and fubp1
US20250340934A1 (en) * 2024-05-06 2025-11-06 Gt Molecular, Inc. Selective blocking to detect and amplify low abundant template

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008518608A (ja) * 2004-11-09 2008-06-05 サンタリス ファーマ アー／エス ＨＩＦ−１ａ発現阻害のための強力なＬＮＡオリゴヌクレオチド

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
DE19956568A1 (de) * 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
IL149694A0 (en) * 1999-12-23 2002-11-10 Exiqon As Therapeutic uses of lna-modified oligonucleotides
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
ES2336887T5 (es) * 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
CA2429814C (en) * 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
EP1386004A4 (en) * 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
ATE556714T1 (de) * 2002-02-01 2012-05-15 Life Technologies Corp Doppelsträngige oligonukleotide
DE60310944T3 (de) * 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Weitere neue formen von interferierende rns moleküle
ATE443765T1 (de) * 2003-03-21 2009-10-15 Santaris Pharma As Analoga kurzer interferierender rna (sirna)
WO2005073378A1 (en) * 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008518608A (ja) * 2004-11-09 2008-06-05 サンタリス ファーマ アー／エス ＨＩＦ−１ａ発現阻害のための強力なＬＮＡオリゴヌクレオチド

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
JPN6012025542; Ｂｉｏｃｈｅｍｉｓｔｒｙ Ｖｏｌ．４２, 2003, ｐ．７９６７-７９７５ *
JPN6012025544; Ｂｉｏｏｒｇ．　Ｍｅｄ．　Ｃｈｅｍ．　Ｌｅｔｔ． Ｖｏｌ．８, 1998, ｐ．２２１９-２２２２ *
JPN6012025546; Ｎｕｃｌｅｉｃ　Ａｃｉｄｓ　Ｒｅｓｅａｒｃｈ Ｖｏｌ．３１，　Ｎｏ．２１, 2003, ｐ．６３６５-６３７２ *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018504380A (ja) * 2014-12-18 2018-02-15 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｒｅｖｅｒｓｉｒ（商標）化合物
JP2023018065A (ja) * 2014-12-18 2023-02-07 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｒｅｖｅｒｓｉｒ（商標）化合物
JP2024037841A (ja) * 2014-12-18 2024-03-19 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｒｅｖｅｒｓｉｒ（商標）化合物
US12241064B2 (en) 2014-12-18 2025-03-04 Alnylam Pharmaceuticals, Inc. REVERSIR™ compounds

Also Published As

Publication number Publication date
US20090176977A1 (en) 2009-07-09
AU2007209481B2 (en) 2012-01-12
CA2638837A1 (en) 2007-08-02
DK1984382T3 (da) 2012-09-03
WO2007085485A2 (en) 2007-08-02
AU2007209481A1 (en) 2007-08-02
EP1984382B1 (en) 2012-08-15
WO2007085485A3 (en) 2007-09-13
EP1984382A2 (en) 2008-10-29

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