JP7424728B2 - Rna干渉のための長鎖の二本鎖rna - Google Patents

Rna干渉のための長鎖の二本鎖rna Download PDF

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JP7424728B2
JP7424728B2 JP2019565074A JP2019565074A JP7424728B2 JP 7424728 B2 JP7424728 B2 JP 7424728B2 JP 2019565074 A JP2019565074 A JP 2019565074A JP 2019565074 A JP2019565074 A JP 2019565074A JP 7424728 B2 JP7424728 B2 JP 7424728B2
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ldrna
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mrna
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JP2020508078A5 (enExample
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キ イ、ドン
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オリック パルマセゥティカルズ インコーポレイテッド
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JP2019565074A 2017-02-10 2018-02-12 Rna干渉のための長鎖の二本鎖rna Active JP7424728B2 (ja)

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US201762457282P 2017-02-10 2017-02-10
US62/457,282 2017-02-10
PCT/IB2018/000330 WO2018146557A2 (en) 2017-02-10 2018-02-12 Long double-stranded rna for rna interference

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JP2020508078A JP2020508078A (ja) 2020-03-19
JP2020508078A5 JP2020508078A5 (enExample) 2021-03-25
JP7424728B2 true JP7424728B2 (ja) 2024-01-30

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US (1) US11591600B2 (enExample)
EP (1) EP3580339A4 (enExample)
JP (1) JP7424728B2 (enExample)
KR (1) KR20190108167A (enExample)
CN (1) CN110770343A (enExample)
WO (1) WO2018146557A2 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103328633B (zh) 2010-10-22 2018-07-10 成均馆大学校产学协力团 诱导rna干扰的核酸分子及其用途
CN108148838B (zh) 2012-05-22 2026-07-31 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
JP7027311B2 (ja) 2015-11-16 2022-03-01 オリックス ファーマシューティカルズ,インコーポレーテッド MyD88又はTLR3を標的とするRNA複合体を使用した加齢黄斑変性の治療
KR102825945B1 (ko) 2016-02-02 2025-06-30 올릭스 주식회사 Angpt2 및 pdgfb를 표적화하는 rna 복합체를 사용하는 혈관신생 관련 질환의 치료
WO2017134525A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. TREATMENT OF ATOPIC DERMATITIS AND ASTHMA USING RNA COMPLEXES THAT TARGET lL4Rα, TRPA1, OR F2RL1
JP7049262B2 (ja) 2016-04-11 2022-04-06 オリックス ファーマシューティカルズ,インコーポレーテッド 結合組織成長因子を標的とするrna複合体を用いた特発性肺胞線維症の治療
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
WO2018146557A2 (en) 2017-02-10 2018-08-16 Dong Ki Lee Long double-stranded rna for rna interference
KR102421750B1 (ko) 2020-05-26 2022-07-19 올릭스 주식회사 MyD88을 표적으로 하는 RNAi 제제 및 이의 용도
WO2023043239A1 (ko) * 2021-09-15 2023-03-23 올릭스 주식회사 선천성 면역 반응 유도 효과를 갖는 이중가닥 rna 및 이의 용도
WO2024186166A1 (ko) * 2023-03-08 2024-09-12 올릭스 주식회사 선천성 면역 반응 유도 효과를 갖는 이중가닥 rna 및 이의 용도
WO2026054553A1 (ko) * 2024-09-06 2026-03-12 올릭스 주식회사 선천성 면역 반응 기반 항암 요법을 위한 이중 가닥 rna 및 이의 용도

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140249304A1 (en) 2011-05-30 2014-09-04 Research & Business Foundation Sungkyunkwan University Long interfering dsrna simultaneously inducing an immune reaction and the inhibition of the expression of target genes
US20140288148A1 (en) 2002-02-20 2014-09-25 Sirna Therapeutics Inc RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)

Family Cites Families (89)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5837258A (en) 1991-08-30 1998-11-17 University Of South Florida Induction of tissue, bone or cartilage formation using connective tissue growth factor
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20080188430A1 (en) 2001-05-18 2008-08-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA)
BR0211111A (pt) * 2001-07-12 2004-06-22 Univ Massachusetts Molécula de ácido nucleico isolada, vetor, célula hospedeira, transgene, precursor de rna engenheirado, animal transgênico não humano, e, método de induzir a interferência de ácido ribonucleico de um gene alvo em uma célula
US20050119202A1 (en) 2001-10-26 2005-06-02 Roland Kreutzer Medicament to treat a fibrotic disease
US20040138163A1 (en) 2002-05-29 2004-07-15 Mcswiggen James RNA interference mediated inhibition of vascular edothelial growth factor and vascular edothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
US20040198640A1 (en) 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
US20070167384A1 (en) 2003-04-02 2007-07-19 Dharmacon, Inc. Modified polynucleotides for use in rna interference
US20050136437A1 (en) 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
US8680063B2 (en) * 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
US20100247540A1 (en) * 2003-10-30 2010-09-30 Chemocentryx, Inc. Methods and Compositions For Modulating Angiogenesis
US20100145038A1 (en) * 2003-11-24 2010-06-10 Merck & Co., Inc. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
US20060069050A1 (en) 2004-02-17 2006-03-30 University Of Massachusetts Methods and compositions for mediating gene silencing
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
EP1765847A4 (en) * 2004-05-27 2010-10-20 Alnylam Pharmaceuticals Inc NUCLEASERESISTENT DOUBLE-STRANDED RIBONUCLEIC ACID
WO2006031901A2 (en) 2004-09-10 2006-03-23 Somagenics, Inc. SMALL INTERFERING RNAs THAT EFFICIENTLY INHIBIT VIRAL GENE EXPRESSION AND METHODS OF USE THEREOF
CA2580707C (en) 2004-09-24 2014-07-08 Alnylam Pharmaceuticals, Inc. Rnai modulation of apob and uses thereof
US20060142228A1 (en) 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
TWI386225B (zh) 2004-12-23 2013-02-21 Alcon Inc 用於治療眼睛病症的結締組織生長因子(CTGF)RNA干擾(RNAi)抑制技術
CA2603842A1 (en) 2005-04-08 2006-10-19 Nastech Pharmaceutical Company Inc. Rnai therapeutic for respiratory virus infection
US8067572B2 (en) 2005-05-25 2011-11-29 The University Of York Hybrid interfering RNA
EP1896069B1 (en) 2005-06-24 2013-03-13 Intervet International BV Inactivated chimeric vaccines and related methods of use
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
FR2890859B1 (fr) 2005-09-21 2012-12-21 Oreal Oligonucleotide d'arn double brin inhibant l'expression de la tyrosinase
US7825099B2 (en) 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
AU2007212700A1 (en) 2006-01-26 2007-08-16 University Of Massachusetts RNA interference agents for therapeutic use
EP2526968A3 (en) 2006-01-27 2013-05-22 Biogen Idec MA Inc. Nogo receptor antagonists
US20070218495A1 (en) 2006-03-16 2007-09-20 Dharmacon, Inc. Methods, libraries and computer program products for gene silencing with reduced off-target effects
US7700541B2 (en) 2006-04-06 2010-04-20 Nitto Denko Corporation Biodegradable cationic polymers
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
US7915399B2 (en) * 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
JP2010519907A (ja) 2007-03-02 2010-06-10 エムディーアールエヌエー,インコーポレイテッド Vegfファミリー遺伝子の発現を抑制するための核酸化合物およびその使用
US20100105134A1 (en) 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
DK2164967T3 (en) 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
US20090004668A1 (en) 2007-06-22 2009-01-01 The Board Of Trustees Of The Leland Stanford Junior University Pre-miRNA loop-modulated target regulation
CA2728571A1 (en) * 2007-06-29 2009-01-08 Boston Biomedical, Inc. Bacteria-mediated gene modulation via microrna machinery
WO2009008645A2 (en) * 2007-07-06 2009-01-15 Mogam Biotechnology Research Institute Linear double-stranded rna molecule interfering with different target genes
WO2009020344A2 (en) 2007-08-06 2009-02-12 Postech Acad Ind Found Small interfering rnas (sirnas) controlling multiple target genes and method for preparing the same
EP3121281B1 (en) 2007-08-27 2021-03-31 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
JP5723154B2 (ja) 2007-09-19 2015-05-27 アプライド バイオシステムズ リミテッド ライアビリティー カンパニー RNAiにおけるオフターゲット表現型の影響を減少させるためのSiRNA配列非依存性修飾フォーマットおよびその安定化型
CA2701845A1 (en) 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures
US8614311B2 (en) 2007-12-12 2013-12-24 Quark Pharmaceuticals, Inc. RTP801L siRNA compounds and methods of use thereof
KR100949791B1 (ko) 2007-12-18 2010-03-30 이동기 오프-타겟 효과를 최소화하고 RNAi 기구를 포화시키지않는 신규한 siRNA 구조 및 그 용도
AU2009215795A1 (en) 2008-02-21 2009-08-27 University Of Kentucky Research Foundation Ultra-small RNAs as toll-like receptor-3 antagonists
CA2731779A1 (en) 2008-07-24 2010-01-28 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
US8580562B2 (en) 2008-09-08 2013-11-12 Fukuoka University Inhibitory RNA for modulating the molecular function of ZFAT gene
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
US20100227920A1 (en) 2008-09-29 2010-09-09 The Regents Of The University Of California Aldehyde dehydrogenase inhibitors as novel depigmenting agents
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010094491A1 (en) 2009-02-18 2010-08-26 Silence Therapeutics Ag Means for inhibiting the expression of ang2
JP2012520683A (ja) 2009-03-19 2012-09-10 メルク・シャープ・エンド・ドーム・コーポレイション 低分子干渉核酸(siNA)を用いた結合組織増殖因子(CTGF)遺伝子発現のRNA干渉媒介性阻害
JP6010458B2 (ja) 2009-04-03 2016-10-19 ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. 非対称二本鎖rnaによる特異的阻害のための、方法および組成物
WO2011019887A2 (en) 2009-08-14 2011-02-17 Massachusetts Institute Of Technology Waveguide coupler having continuous three-dimensional tapering
KR101207561B1 (ko) 2009-12-15 2012-12-04 주식회사 코리아나화장품 티로시나제의 발현을 저해하는 siRNA 올리고뉴클레오타이드 및 이를 함유하는 화장료 조성물
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
US9095504B2 (en) 2010-03-24 2015-08-04 Rxi Pharmaceuticals Corporation RNA interference in ocular indications
EP2569445B1 (en) 2010-05-12 2017-01-04 Val-Chum, Limited Partnership Screening assays based on abhd6 for selecting insulin secretion promoting agents
US9290760B2 (en) 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
CN103328633B (zh) 2010-10-22 2018-07-10 成均馆大学校产学协力团 诱导rna干扰的核酸分子及其用途
CA2818024C (en) 2010-12-06 2019-09-24 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
AU2012223366B2 (en) 2011-03-03 2017-02-23 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
CN102719432B (zh) 2011-03-29 2013-10-23 南京大学 特异性抑制肿瘤凋亡抑制基因Bcl2的双链不对称小核酸干扰分子asiRNA及其应用
CA2831613A1 (en) * 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
JP6313203B2 (ja) 2011-07-18 2018-04-18 ユニバーシティー オブ ケンタッキー リサーチ ファウンデイションUniversity Of Kentucky Research Foundation Alu−rna誘導変性からの細胞の保護及び細胞保護用阻害剤
US9707235B1 (en) 2012-01-13 2017-07-18 University Of Kentucky Research Foundation Protection of cells from degeneration and treatment of geographic atrophy
CN108148838B (zh) 2012-05-22 2026-07-31 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
WO2014043291A1 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded nucleic acid compounds
RU2656154C2 (ru) 2013-07-05 2018-05-31 Байонир Корпорейшн СВЯЗАННАЯ С РЕСПИРАТОРНЫМ ЗАБОЛЕВАНИЕМ ГЕН-СПЕЦИФИЧЕСКАЯ миРНК, ДВУСПИРАЛЬНАЯ КОНСТРУКЦИЯ ОЛИГО-РНК, СОДЕРЖАЩАЯ миРНК, И СОДЕРЖАЩАЯ ЕЕ КОМПОЗИЦИЯ ДЛЯ ПРЕДУПРЕЖДЕНИЯ ИЛИ ЛЕЧЕНИЯ РЕСПИРАТОРНОГО ЗАБОЛЕВАНИЯ
EP3027223A1 (en) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
US9790506B2 (en) 2013-10-02 2017-10-17 The Regents Of The University Of California Diagnostic and screening methods for atopic dermatitis
US8980273B1 (en) 2014-07-15 2015-03-17 Kymab Limited Method of treating atopic dermatitis or asthma using antibody to IL4RA
US20170226507A1 (en) 2014-05-05 2017-08-10 The Brigham And Women's Hospital, Inc. Coordinate control of pathogenic signaling by the mir-130/301 family in pulmonary hypertension and fibroproliferative diseases
US9139648B1 (en) 2014-07-15 2015-09-22 Kymab Limited Precision medicine by targeting human NAV1.9 variants for treatment of pain
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
CN108138181B (zh) 2015-07-27 2022-05-24 奥利克斯医药有限公司 抑制黑色素生成的rna复合物
JP7027311B2 (ja) 2015-11-16 2022-03-01 オリックス ファーマシューティカルズ,インコーポレーテッド MyD88又はTLR3を標的とするRNA複合体を使用した加齢黄斑変性の治療
KR102825945B1 (ko) 2016-02-02 2025-06-30 올릭스 주식회사 Angpt2 및 pdgfb를 표적화하는 rna 복합체를 사용하는 혈관신생 관련 질환의 치료
WO2017134525A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. TREATMENT OF ATOPIC DERMATITIS AND ASTHMA USING RNA COMPLEXES THAT TARGET lL4Rα, TRPA1, OR F2RL1
JP7049262B2 (ja) 2016-04-11 2022-04-06 オリックス ファーマシューティカルズ,インコーポレーテッド 結合組織成長因子を標的とするrna複合体を用いた特発性肺胞線維症の治療
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
WO2018146557A2 (en) 2017-02-10 2018-08-16 Dong Ki Lee Long double-stranded rna for rna interference

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140288148A1 (en) 2002-02-20 2014-09-25 Sirna Therapeutics Inc RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
US20140249304A1 (en) 2011-05-30 2014-09-04 Research & Business Foundation Sungkyunkwan University Long interfering dsrna simultaneously inducing an immune reaction and the inhibition of the expression of target genes

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
FEBS Letters,2014年,Vol.588,P.1037-1043
nature biotechnology,2006年,Vol.24, No.5,P.559-565
Ui-Tei K et al.,"RNAi induced in mammalian and Drosophila cells via transfection of dimers and trimers of small interfering RNA.",Journal of RNAi and gene silencing,2005年10月14日,Vol. 1, No. 2,p.79-87

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