JP2011528910A - センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進 - Google Patents

センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進 Download PDF

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JP2011528910A
JP2011528910A JP2011520219A JP2011520219A JP2011528910A JP 2011528910 A JP2011528910 A JP 2011528910A JP 2011520219 A JP2011520219 A JP 2011520219A JP 2011520219 A JP2011520219 A JP 2011520219A JP 2011528910 A JP2011528910 A JP 2011528910A
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sirna
irna agent
sense strand
gene
expression
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JP2011528910A5 (enExample
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マイアー，マーティン
アッデパリ，ハリプリヤ
マノハラン，ムシアー
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Alnylam Pharmaceuticals Inc
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JP2011520219A 2008-07-25 2009-07-24 センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進 Pending JP2011528910A (ja)

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US8376308P 2008-07-25 2008-07-25
US61/083,763 2008-07-25
PCT/US2009/051648 WO2010011895A1 (en) 2008-07-25 2009-07-24 Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand

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JP2016189465A Active JP6476155B2 (ja) 2008-07-25 2016-09-28 センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進

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Cited By (5)

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Publication number Priority date Publication date Assignee Title
JP2015518704A (ja) * 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 膜タンパク質の産生のための修飾ポリヌクレオチド
WO2018143475A1 (ja) * 2017-02-06 2018-08-09 日産化学工業株式会社 一本鎖オリゴヌクレオチド
JP2022538404A (ja) * 2019-06-21 2022-09-02 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 構造的に明確なｓｉＲＮＡ－二重可変ドメイン免疫グロブリンコンジュゲート
US11530409B2 (en) 2016-01-26 2022-12-20 Nissan Chemical Corporation Single-stranded oligonucleotide
JP2024515734A (ja) * 2021-04-23 2024-04-10 南▲開▼大学 コンジュゲート、その製造方法、及び使用

Families Citing this family (88)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
US8796436B2 (en) * 2003-04-17 2014-08-05 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
WO2004094595A2 (en) 2003-04-17 2004-11-04 Alnylam Pharmaceuticals Inc. MODIFIED iRNA AGENTS
CA2744093A1 (en) 2008-12-03 2010-06-10 Marina Biotech, Inc. Una oligomer structures for therapeutic agents
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
WO2011109427A2 (en) 2010-03-01 2011-09-09 Alnylam Pharmaceuticals, Inc. Improving the biological activity of sirna through modulation of its thermodynamic profile
WO2012007477A1 (en) 2010-07-12 2012-01-19 Santaris Pharma A/S Anti hcv oligomers
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9750819B2 (en) * 2012-05-23 2017-09-05 Ohio State Innovation Foundation Lipid nanoparticle compositions and methods of making and methods of using the same
WO2013179292A1 (en) * 2012-05-31 2013-12-05 Qbi Enterprises Ltd. Therapeutic oligonucleotides comprising pyrazolotriazine nucleotide analogues
EA201690863A1 (ru) * 2012-10-24 2016-09-30 ОБЩЕСТВО С ОГРАНИЧЕННОЙ ОТВЕТСТВЕННОСТЬЮ "НоваМедика" Нуклеиново-кислотная регуляция специфического белка блокировки роста 6 (gas6)
KR20160021076A (ko) 2013-03-15 2016-02-24 테출론 인코포레이티드 스타필로코커스 아우레우스 감염의 치료를 위한 안티센스 분자
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3234141A4 (en) 2014-12-18 2018-06-20 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
US10264976B2 (en) 2014-12-26 2019-04-23 The University Of Akron Biocompatible flavonoid compounds for organelle and cell imaging
US11045488B2 (en) 2014-12-26 2021-06-29 Nitto Denko Corporation RNA interference agents for GST-π gene modulation
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
EP3919619A1 (en) 2015-07-17 2021-12-08 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
EP3334499A4 (en) 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
HK1258758A1 (zh) 2016-01-31 2019-11-22 University Of Massachusetts 分支的寡核苷酸
US11208653B2 (en) 2016-04-27 2021-12-28 Whitehead Institute For Biomedical Research Increasing RNAi efficiency through single nucleotide mismatches
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
CN110799647A (zh) 2017-06-23 2020-02-14 马萨诸塞大学 双尾自递送sirna及相关的方法
US12397013B2 (en) 2018-05-07 2025-08-26 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
JP7470107B2 (ja) 2018-09-28 2024-04-17 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 トランスサイレチン（ＴＴＲ）ｉＲＮＡ組成物及びＴＴＲ関連眼疾患を治療又は予防するためのその使用方法
EP3911747A4 (en) 2019-01-18 2023-05-24 University Of Massachusetts DYNAMIC PHARMACOKINETICS-MODIFYING ANCHORS
CA3138915A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
MX2022004388A (es) 2019-11-01 2022-05-06 Alnylam Pharmaceuticals Inc Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas.
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
KR20220110749A (ko) 2019-11-06 2022-08-09 알닐람 파마슈티칼스 인코포레이티드 간외 전달
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
MX2022006433A (es) 2019-12-13 2022-06-23 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) del marco de lectura abierto 72 del cromosoma 9 humano (c9orf72) y metodos de uso de los mismos.
AU2021220765A1 (en) 2020-02-10 2022-09-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing VEGF-A expression
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
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US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
WO2021207167A1 (en) 2020-04-06 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
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WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CA3179678A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
WO2021222065A1 (en) 2020-04-27 2021-11-04 Alnylam Pharmaceuticals, Inc. Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
US20230183707A1 (en) 2020-05-21 2023-06-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
KR102361479B1 (ko) * 2020-06-18 2022-02-11 고려대학교 산학협력단 ｓｉＲＮＡ의 생성 및 기능 증진 활성을 갖는 ＣＭＴＲ１의 신규용도
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
US20240343746A1 (en) 2020-12-31 2024-10-17 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4271695A2 (en) 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
CA3210763A1 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
CN114958839A (zh) * 2021-02-25 2022-08-30 上海市东方医院（同济大学附属东方医院） 抑制肌肉瘤细胞中FOXO1基因表达的siRNA序列及应用
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
KR20230162024A (ko) 2021-03-29 2023-11-28 알닐람 파마슈티칼스 인코포레이티드 헌팅틴(HTT) iRNA 제제 조성물 및 이의 사용 방법
EP4341401A1 (en) 2021-05-18 2024-03-27 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
BR112023025224A2 (pt) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos
AU2022299169A1 (en) 2021-06-23 2024-02-08 Beth Israel Deaconess Medical Center, Inc. Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
EP4367237A2 (en) 2021-07-09 2024-05-15 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
JP2024528697A (ja) 2021-07-20 2024-07-30 エイジーエス・セラピューティクス・ソシエテ・パール・アクシオン・サンプリフィエ 微細藻類由来の細胞外小胞、その調製および使用
KR20240036041A (ko) 2021-07-21 2024-03-19 알닐람 파마슈티칼스 인코포레이티드 대사 장애-연관 표적 유전자 iRNA 조성물 및 이의 사용 방법
MX2024002440A (es) 2021-08-31 2024-03-08 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) del efector b similar al factor de fragmentacion de adn subunidad alfa (dffa) que induce la muerte celular (cideb) y metodos de uso de estas.
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
CA3234887A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
EP4423272A2 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
TW202400787A (zh) 2022-03-16 2024-01-01 美商安彼瑞可股份有限公司 改良siRNA生物可利用性之GalNAc組合物
WO2023192830A2 (en) 2022-03-28 2023-10-05 Empirico Inc. Modified oligonucleotides
JP2025516680A (ja) 2022-05-13 2025-05-30 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 一本鎖ループオリゴヌクレオチド
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
JP2025532985A (ja) 2022-09-30 2025-10-03 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 修飾二本鎖ｒｎａ剤
EP4608424A1 (en) 2022-10-24 2025-09-03 AGS Therapeutics SAS Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof
WO2024197280A1 (en) * 2023-03-23 2024-09-26 Atalanta Therapeutics, Inc. Compositions and methods for improved gene silencing
WO2024216155A1 (en) 2023-04-12 2024-10-17 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
WO2025176847A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Ocular delivery of active agents via microalgae extracellular vesicles
WO2025259747A2 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070054279A1 (en) * 2004-08-04 2007-03-08 Alnylam Pharmaceuticals Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
WO2007053696A2 (en) * 2005-11-01 2007-05-10 Alnylam Pharmaceuticals, Inc. Rnai inhibition of influenza virus replication

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
FR2645866B1 (fr) * 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5283185A (en) * 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5691316A (en) * 1994-06-01 1997-11-25 Hybridon, Inc. Cyclodextrin cellular delivery system for oligonucleotides
US6509323B1 (en) * 1998-07-01 2003-01-21 California Institute Of Technology Linear cyclodextrin copolymers
US6066500A (en) * 1999-06-25 2000-05-23 Isis Pharmaceuticals Inc. Antisense modulation of Beta catenin expression
WO2002081628A2 (en) * 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
US6627425B1 (en) * 2000-06-02 2003-09-30 Millennium Pharmaceuticals, Inc. Human glucose-6-phosphatase molecules and uses thereof
TWI321054B (en) * 2000-12-19 2010-03-01 California Inst Of Techn Compositions containing inclusion complexes
US7517864B2 (en) * 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7888324B2 (en) * 2001-08-01 2011-02-15 Genzyme Corporation Antisense modulation of apolipoprotein B expression
AU2002343792A1 (en) * 2001-11-28 2003-06-10 Center For Advanced Science And Technology Incubation, Ltd. siRNA EXPRESSION SYSTEM AND PROCESS FOR PRODUCING FUNCTIONAL GENE-KNOCKDOWN CELLS AND THE LIKE USING THE SAME
US20050233342A1 (en) * 2003-03-07 2005-10-20 Muthiah Manoharan Methods of preventing off-target gene silencing
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
ES2357116T5 (es) * 2003-06-02 2021-09-03 Univ Massachusetts Métodos y composiciones para mejorar la eficacia y especificad de FNAi
EP1666593A4 (en) * 2003-08-18 2009-07-15 Japan Health Science Found IMPROVED SIRNA MOLECULE AND METHOD FOR INHIBITING GENE EXPRESSION USED THEREOF
WO2005073378A1 (en) * 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
CA2574088C (en) * 2004-07-21 2013-09-17 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a modified or non-natural nucleobase
US20080213891A1 (en) * 2004-07-21 2008-09-04 Alnylam Pharmaceuticals, Inc. RNAi Agents Comprising Universal Nucleobases
US20060089324A1 (en) * 2004-10-22 2006-04-27 Sailen Barik RNAi modulation of RSV, PIV and other respiratory viruses and uses thereof
WO2009142822A2 (en) * 2008-03-26 2009-11-26 Alnylam Pharmaceuticals, Inc. 2-f modified rna interference agents
JP2012147677A (ja) * 2009-05-14 2012-08-09 Nippon Shinyaku Co Ltd 二本鎖修飾ｒｎａ

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070054279A1 (en) * 2004-08-04 2007-03-08 Alnylam Pharmaceuticals Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
WO2007053696A2 (en) * 2005-11-01 2007-05-10 Alnylam Pharmaceuticals, Inc. Rnai inhibition of influenza virus replication

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
JPN6014004590; ＡＣＳ　ＣＨＥＭＩＣＡＬ　ＢＩＯＬＯＧＹ ｖｏｌ．１　ｎｏ．３, 2006, ｐｐ．１７６-１８３ *
JPN6014004598; ＭＯＬＥＣＵＬＡＲ　ＴＨＥＲＡＰＹ ｖｏｌ．１３　ｎｏ．３, 2006, ｐｐ．４９４-５０５ *
JPN6014004601; ＣＨＥＭＩＳＴＲＹ　＆　ＢＩＯＤＩＶＥＲＳＩＴＹ ｖｏｌ．４, 2007, ｐｐ．８５８-８７３ *
JPN6014004604; Ｊ．　Ｍｅｄ．　Ｃｈｅｍ． ｖｏｌ．４８, 2005, ｐｐ．４２４７-４２５３ *
JPN6014004609; Ｎｕｃｌｅｉｃ　Ａｃｉｄｓ　Ｒｅｓｅａｒｃｈ ｖｏｌ．３５　ｎｏ．１９, 2007, ｐｐ．６４２４-６４３８ *

Cited By (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015518704A (ja) * 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 膜タンパク質の産生のための修飾ポリヌクレオチド
US11530409B2 (en) 2016-01-26 2022-12-20 Nissan Chemical Corporation Single-stranded oligonucleotide
WO2018143475A1 (ja) * 2017-02-06 2018-08-09 日産化学工業株式会社 一本鎖オリゴヌクレオチド
JPWO2018143475A1 (ja) * 2017-02-06 2019-12-12 日産化学株式会社 一本鎖オリゴヌクレオチド
JP7188087B2 (ja) 2017-02-06 2022-12-13 日産化学株式会社 一本鎖オリゴヌクレオチド
US11572558B2 (en) 2017-02-06 2023-02-07 Nissan Chemical Corporation Single-stranded oligonucleotide
JP2023025156A (ja) * 2017-02-06 2023-02-21 日産化学株式会社 一本鎖オリゴヌクレオチド
JP7601080B2 (ja) 2017-02-06 2024-12-17 日産化学株式会社 一本鎖オリゴヌクレオチド
JP2022538404A (ja) * 2019-06-21 2022-09-02 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 構造的に明確なｓｉＲＮＡ－二重可変ドメイン免疫グロブリンコンジュゲート
JP7785543B2 (ja) 2019-06-21 2025-12-15 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 構造的に明確なｓｉＲＮＡ－二重可変ドメイン免疫グロブリンコンジュゲート
JP2024515734A (ja) * 2021-04-23 2024-04-10 南▲開▼大学 コンジュゲート、その製造方法、及び使用
JP7668378B2 (ja) 2021-04-23 2025-04-24 コーズィケム・バイオテクノロジー・（テンシン）・カンパニー・リミテッド コンジュゲート、その製造方法、及び使用

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