TWI883468B - 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法 - Google Patents

甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法 Download PDF

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TWI883468B
TWI883468B TW112123223A TW112123223A TWI883468B TW I883468 B TWI883468 B TW I883468B TW 112123223 A TW112123223 A TW 112123223A TW 112123223 A TW112123223 A TW 112123223A TW I883468 B TWI883468 B TW I883468B
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ttr
double
rnai agent
nucleotides
sense strand
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TW112123223A
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TW202344688A (zh
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崔西 利莫爾曼
艾咪 陳
瓦聖特 基哈夫
馬丁 馬爾
卡拉司塔司爾Ｇ 拉傑
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美商阿尼拉製藥公司
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TW112123223A 2015-07-31 2016-07-29 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法 TWI883468B (zh)

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TW112123223A TWI883468B (zh) 2015-07-31 2016-07-29 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法
TW105124065A TWI727963B (zh) 2015-07-31 2016-07-29 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法
TW110113415A TWI807302B (zh) 2015-07-31 2016-07-29 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法
TW114112856A TW202532647A (zh) 2015-07-31 2016-07-29 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法

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TW110113415A TWI807302B (zh) 2015-07-31 2016-07-29 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法
TW114112856A TW202532647A (zh) 2015-07-31 2016-07-29 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法

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Families Citing this family (31)

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Publication number Priority date Publication date Assignee Title
PL2937418T3 (pl) 2008-10-20 2018-04-30 Alnylam Pharmaceuticals, Inc. Kompozycje i sposoby hamowania ekspresji transtyretyny
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
BR112012000828A8 (pt) 2009-07-06 2017-10-10 Ontorii Inc Novas pró-drogas de ácido nucleico e métodos de uso das mesmas
RS56011B1 (sr) 2010-04-29 2017-09-29 Ionis Pharmaceuticals Inc Modulacija ekspresije transtiretina
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
ES2626488T3 (es) 2011-07-19 2017-07-25 Wave Life Sciences Pte. Ltd. Procedimientos para la síntesis de ácidos nucleicos funcionalizados
AU2012340159B2 (en) 2011-11-18 2017-09-07 Alnylam Pharmaceuticals, Inc. RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
CN105861503A (zh) 2011-11-18 2016-08-17 阿尔尼拉姆医药品有限公司 修饰的RNAi试剂
ES2940887T3 (es) 2012-07-13 2023-05-12 Wave Life Sciences Ltd Método de preparación de oligonucleótidos quirales
ES2862073T3 (es) 2012-07-13 2021-10-06 Wave Life Sciences Ltd Grupo auxiliar asimétrico
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
WO2015108047A1 (ja) 2014-01-15 2015-07-23 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
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
RS61297B1 (sr) * 2015-07-31 2021-02-26 Alnylam Pharmaceuticals Inc Transtiretinske (ttr) kompozicije irnk i postupci za njihovu primenu u lečenju ili prevenciji bolesti povezanih sa ttr-om
JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
TWI865997B (zh) * 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
KR20250046363A (ko) 2017-09-19 2025-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
AU2018374219C1 (en) * 2017-12-01 2023-05-11 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof
CA3114396A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
EP4022062A1 (en) 2019-08-30 2022-07-06 Alnylam Pharmaceuticals, Inc. Neurofilament light chain (nfl) as a biomarker for transthyretin amyloidosis polyneuropathy
IL296106A (en) * 2020-03-06 2022-11-01 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of transthyretin (ttr)
EP4237005A4 (en) * 2020-10-28 2024-10-09 Novo Nordisk A/S ANTI-TRANSTHYRETIN ANTIBODIES AND METHODS OF USE THEREOF
WO2022204430A1 (en) * 2021-03-24 2022-09-29 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
JP2024523000A (ja) 2021-06-08 2024-06-25 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 シュタルガルト病及び／又は網膜結合タンパク質４（ｒｂｐ４）関連障害を治療又は予防するための組成物及び方法
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO
AR131389A1 (es) * 2022-12-16 2025-03-12 Amgen Inc CONSTRUCTOS DE ARNi PARA INHIBIR LA EXPRESIÓN DE TTR Y MÉTODOS DE USO DE LOS MISMOS
WO2025072748A2 (en) 2023-09-29 2025-04-03 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease
WO2025077916A1 (zh) * 2023-10-13 2025-04-17 北京安龙生物医药有限公司 靶向转甲状腺素蛋白的寡核苷酸及其用途
WO2025098117A1 (zh) * 2023-11-06 2025-05-15 成都倍特药业股份有限公司 一种抑制甲状腺素转运蛋白(TTR)表达的RNAi试剂、组合物及应用
TW202521133A (zh) * 2023-11-27 2025-06-01 大陸商上海舶望製藥有限公司 抑制甲狀腺素運載蛋白(ttr)表達的組合物和方法
US20250333742A1 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104080794A (zh) * 2011-11-18 2014-10-01 阿尔尼拉姆医药品有限公司 RNAi试剂、组合物及其用于治疗甲状腺素运载蛋白(TTR)相关疾病的使用方法
TWI727963B (zh) * 2015-07-31 2021-05-21 美商阿尼拉製藥公司 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法

Family Cites Families (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030027216A1 (en) 2001-07-02 2003-02-06 Kiernan Urban A. Analysis of proteins from biological fluids using mass spectrometric immunoassay
WO2001057064A2 (en) 2000-02-07 2001-08-09 Roche Diagnostics Corporation Cationic amphiphiles for use in nucleic acid transfection
US20030072794A1 (en) 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
CZ308053B6 (cs) 2000-12-01 2019-11-27 Max Planck Gesellschaft Izolovaná molekula dvouřetězcové RNA, způsob její výroby a její použití
US20020160394A1 (en) 2001-01-24 2002-10-31 Bayer Corporation Regulation of transthyretin to treat obesity
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
US20030170891A1 (en) 2001-06-06 2003-09-11 Mcswiggen James A. RNA interference mediated inhibition of epidermal growth factor receptor gene expression using short interfering nucleic acid (siNA)
AU2002318396A1 (en) 2001-06-21 2003-01-08 The Cleveland Clinic Foundation Homocysteinylated transthyretin
US20030191056A1 (en) 2002-04-04 2003-10-09 Kenneth Walker Use of transthyretin peptide/protein fusions to increase the serum half-life of pharmacologically active peptides/proteins
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20040119010A1 (en) 2002-11-01 2004-06-24 The Regents Of The University Of Colorado Quantitative analysis of protein isoforms using matrix-assisted laser desorption/ionization time of flight mass spectrometry
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US8090542B2 (en) 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
ATE479752T1 (de) 2003-03-07 2010-09-15 Alnylam Pharmaceuticals Inc Therapeutische zusammensetzungen
JP2006522158A (ja) 2003-04-03 2006-09-28 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 ｉＲＮＡ複合体
JP4951338B2 (ja) 2003-07-16 2012-06-13 プロチバ バイオセラピューティクス インコーポレイティッド 脂質に封入された干渉ｒｎａ
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
JP2008501694A (ja) 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク． 遺伝子調節の使用のために個別に修飾された鎖を有する二本鎖組成物
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
WO2005120583A2 (en) 2004-06-14 2005-12-22 Wisconsin Alumni Research Foundation Method for preventing or treating cardiac hypertrophy
KR100890410B1 (ko) 2004-12-08 2009-03-26 시리온 테라퓨틱스, 인크. 레티놀-관련 질환의 치료 방법, 검정법 및 치료용 조성물
WO2007086881A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
JP2008537551A (ja) 2005-03-31 2008-09-18 カランド ファーマシューティカルズ， インコーポレイテッド リボヌクレオチドレダクターゼサブユニット２の阻害剤およびその使用
US7915230B2 (en) 2005-05-17 2011-03-29 Molecular Transfer, Inc. Reagents for transfection of eukaryotic cells
CN101267805A (zh) 2005-07-27 2008-09-17 普洛体维生物治疗公司 制造脂质体的系统和方法
CA2628300C (en) 2005-11-02 2018-04-17 Protiva Biotherapeutics, Inc. Modified sirna molecules and uses thereof
EP2008274B1 (en) 2006-03-31 2015-05-27 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of eg5 gene
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
GB0704764D0 (en) 2007-03-12 2007-04-18 Electrophoretics Ltd Isobarically labelled reagents and methods of their use
US20110046206A1 (en) 2007-06-22 2011-02-24 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
EP2238251B1 (en) 2007-12-27 2015-02-11 Protiva Biotherapeutics Inc. Silencing of polo-like kinase expression using interfering rna
JP5749494B2 (ja) 2008-01-02 2015-07-15 テクミラ ファーマシューティカルズ コーポレイション 核酸の送達のための改善された組成物および方法
AU2009241591A1 (en) 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of DSRNA targeting the PCSK9 gene
WO2009100390A2 (en) 2008-02-08 2009-08-13 Mayo Foundation For Medical Education And Research Classifying amyloidosis
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
TWI455944B (zh) 2008-07-01 2014-10-11 Daiichi Sankyo Co Ltd 雙股多核苷酸
EP2323667A4 (en) 2008-08-07 2012-07-25 Isis Pharmaceuticals Inc MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES
JP5777519B2 (ja) 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
PL2937418T3 (pl) 2008-10-20 2018-04-30 Alnylam Pharmaceuticals, Inc. Kompozycje i sposoby hamowania ekspresji transtyretyny
CA3006395C (en) 2008-11-07 2022-05-31 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
PL2355851T3 (pl) 2008-11-10 2018-08-31 Arbutus Biopharma Corporation Nowe lipidy i kompozycje do dostarczania środków terapeutycznych
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
US8883202B2 (en) 2009-05-05 2014-11-11 Tekmira Pharmaceuticals Corporation Lipid compositions
LT2440183T (lt) 2009-06-10 2018-08-10 Arbutus Biopharma Corporation Patobulinta lipido kompozicija
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
MX2011013421A (es) 2009-06-15 2012-03-16 Alnylam Pharmaceuticals Inc Arnds formulado con lipido de direccionamiento del gen pcsk9.
WO2011056883A1 (en) 2009-11-03 2011-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (ttr)
US20230340473A1 (en) 2010-03-29 2023-10-26 Alnylam Pharmaceuticals, Inc. Sirna therapy for transthyretin (ttr) related ocular amyloidosis
WO2011123468A1 (en) 2010-03-29 2011-10-06 Alnylam Pharmaceuticals, Inc. Sirna therapy for transthyretin (ttr) related ocular amyloidosis
RS56011B1 (sr) 2010-04-29 2017-09-29 Ionis Pharmaceuticals Inc Modulacija ekspresije transtiretina
WO2012037254A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. MODIFIED iRNA AGENTS
EP2632472B1 (en) 2010-10-29 2017-12-13 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
CN110123830A (zh) 2010-11-09 2019-08-16 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因的表达的脂质配制的组合物和方法
US20140275211A1 (en) 2011-06-21 2014-09-18 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
CN105861503A (zh) 2011-11-18 2016-08-17 阿尔尼拉姆医药品有限公司 修饰的RNAi试剂
WO2013166041A1 (en) 2012-05-01 2013-11-07 The Trustees Of Columbia University In The City Of New York Transthyretin ligands capable of inhibiting retinol-dependent rbp4-ttr interaction for treatment of age-related macular
EP2880162B1 (en) 2012-08-03 2017-07-05 Alnylam Pharmaceuticals, Inc. Modified rnai agents
WO2015042564A1 (en) * 2013-09-23 2015-03-26 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing transthyretin (ttr) associated diseases
CA2932904A1 (en) 2013-12-06 2015-06-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of transthyretin (ttr) by double-stranded rna
KR20240170595A (ko) * 2013-12-12 2024-12-03 알닐람 파마슈티칼스 인코포레이티드 보체 성분 ｉＲＮＡ 조성물 및 이의 이용 방법
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
KR102410072B1 (ko) 2014-08-29 2022-06-20 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증의 치료 방법
EP4029859A1 (en) 2014-11-21 2022-07-20 Bsim Therapeutics, S.A. 2-oxothiazolidin-4-one derivatives active as transthyretin ligands and uses thereof
TWI865997B (zh) 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
KR20250046363A (ko) 2017-09-19 2025-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
CA3114396A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
EP4022062A1 (en) 2019-08-30 2022-07-06 Alnylam Pharmaceuticals, Inc. Neurofilament light chain (nfl) as a biomarker for transthyretin amyloidosis polyneuropathy
WO2021092145A1 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna composition and methods of use thereof for treating or preventing ttr-associated ocular diseases
IL296106A (en) 2020-03-06 2022-11-01 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of transthyretin (ttr)
JP2024518374A (ja) 2021-05-03 2024-05-01 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）媒介性アミロイドーシスを治療するための組成物および方法
JP2024523000A (ja) 2021-06-08 2024-06-25 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 シュタルガルト病及び／又は網膜結合タンパク質４（ｒｂｐ４）関連障害を治療又は予防するための組成物及び方法
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104080794A (zh) * 2011-11-18 2014-10-01 阿尔尼拉姆医药品有限公司 RNAi试剂、组合物及其用于治疗甲状腺素运载蛋白(TTR)相关疾病的使用方法
TWI727963B (zh) * 2015-07-31 2021-05-21 美商阿尼拉製藥公司 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關疾病之使用方法

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