JP2018523655A5 - - Google Patents

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Publication number
JP2018523655A5
JP2018523655A5 JP2018504819A JP2018504819A JP2018523655A5 JP 2018523655 A5 JP2018523655 A5 JP 2018523655A5 JP 2018504819 A JP2018504819 A JP 2018504819A JP 2018504819 A JP2018504819 A JP 2018504819A JP 2018523655 A5 JP2018523655 A5 JP 2018523655A5
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double
rnai agent
nucleotide
stranded rnai
seq
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JP2018504819A
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English (en)
Japanese (ja)
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JP2018523655A (ja
JP6896703B2 (ja
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Priority claimed from PCT/US2016/044359 external-priority patent/WO2017023660A1/en
Publication of JP2018523655A publication Critical patent/JP2018523655A/ja
Publication of JP2018523655A5 publication Critical patent/JP2018523655A5/ja
Priority to JP2021096344A priority Critical patent/JP7417559B2/ja
Application granted granted Critical
Publication of JP6896703B2 publication Critical patent/JP6896703B2/ja
Priority to JP2023199064A priority patent/JP7711150B2/ja
Priority to JP2025115513A priority patent/JP2025165939A/ja
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JP2018504819A 2015-07-31 2016-07-28 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法 Active JP6896703B2 (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2021096344A JP7417559B2 (ja) 2015-07-31 2021-06-09 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
JP2023199064A JP7711150B2 (ja) 2015-07-31 2023-11-24 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
JP2025115513A JP2025165939A (ja) 2015-07-31 2025-07-09 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562199563P 2015-07-31 2015-07-31
US62/199,563 2015-07-31
US201662287518P 2016-01-27 2016-01-27
US62/287,518 2016-01-27
PCT/US2016/044359 WO2017023660A1 (en) 2015-07-31 2016-07-28 TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES

Related Child Applications (1)

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JP2021096344A Division JP7417559B2 (ja) 2015-07-31 2021-06-09 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法

Publications (3)

Publication Number Publication Date
JP2018523655A JP2018523655A (ja) 2018-08-23
JP2018523655A5 true JP2018523655A5 (OSRAM) 2019-08-29
JP6896703B2 JP6896703B2 (ja) 2021-06-30

Family

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Family Applications (4)

Application Number Title Priority Date Filing Date
JP2018504819A Active JP6896703B2 (ja) 2015-07-31 2016-07-28 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
JP2021096344A Active JP7417559B2 (ja) 2015-07-31 2021-06-09 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
JP2023199064A Active JP7711150B2 (ja) 2015-07-31 2023-11-24 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
JP2025115513A Pending JP2025165939A (ja) 2015-07-31 2025-07-09 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法

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JP2021096344A Active JP7417559B2 (ja) 2015-07-31 2021-06-09 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
JP2023199064A Active JP7711150B2 (ja) 2015-07-31 2023-11-24 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法
JP2025115513A Pending JP2025165939A (ja) 2015-07-31 2025-07-09 TTR関連疾患を治療または予防するためのトランスサイレチン(TTR)iRNA組成物およびその使用方法

Country Status (35)

Country Link
US (5) US10208307B2 (OSRAM)
EP (1) EP3329002B1 (OSRAM)
JP (4) JP6896703B2 (OSRAM)
KR (2) KR20240074895A (OSRAM)
CN (2) CN108138182B (OSRAM)
BR (1) BR112018000542B1 (OSRAM)
CA (1) CA2994285A1 (OSRAM)
CL (1) CL2018000198A1 (OSRAM)
CY (1) CY1124545T1 (OSRAM)
DK (1) DK3329002T3 (OSRAM)
EA (1) EA201890394A1 (OSRAM)
ES (1) ES2842300T3 (OSRAM)
FI (1) FIC20230006I1 (OSRAM)
FR (1) FR23C1008I2 (OSRAM)
HK (1) HK1256683A1 (OSRAM)
HR (1) HRP20202082T1 (OSRAM)
HU (2) HUE051998T2 (OSRAM)
IL (3) IL313082A (OSRAM)
LT (2) LT3329002T (OSRAM)
LU (1) LUC00297I2 (OSRAM)
MA (1) MA43335B1 (OSRAM)
MX (2) MX2018000981A (OSRAM)
MY (1) MY195720A (OSRAM)
NL (1) NL301216I2 (OSRAM)
NO (1) NO2023007I1 (OSRAM)
PL (1) PL3329002T3 (OSRAM)
PT (1) PT3329002T (OSRAM)
RS (1) RS61297B1 (OSRAM)
SG (1) SG10201912341SA (OSRAM)
SI (1) SI3329002T1 (OSRAM)
SM (1) SMT202100020T1 (OSRAM)
TW (4) TW202532647A (OSRAM)
UA (1) UA126276C2 (OSRAM)
WO (1) WO2017023660A1 (OSRAM)
ZA (2) ZA201800571B (OSRAM)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2937418T (pt) 2008-10-20 2018-01-23 Alnylam Pharmaceuticals Inc Composições e métodos de inibição da expressão de transtirretina
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
IN2012DN00720A (OSRAM) 2009-07-06 2015-06-19 Ontorii Inc
MX343559B (es) 2010-04-29 2016-11-10 Ionis Pharmaceuticals Inc Modulacion de la expresion de transtiretina.
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.
PE20142362A1 (es) 2011-11-18 2015-01-30 Alnylam Pharmaceuticals Inc Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr)
DK3366775T4 (da) * 2011-11-18 2025-10-27 Alnylam Pharmaceuticals Inc Modificerede rnai-midler
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
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
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
CN106068325B (zh) 2014-01-16 2021-07-09 波涛生命科学有限公司 手性设计
ES2842300T3 (es) 2015-07-31 2021-07-13 Alnylam Pharmaceuticals Inc Composiciones de ARNi de transtiretina (TTR) y métodos para su uso para el tratamiento o la prevención de enfermedades asociadas con TTR
JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
EP3555292A1 (en) * 2016-12-16 2019-10-23 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
US11806360B2 (en) 2017-09-19 2023-11-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
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
AU2021232029A1 (en) * 2020-03-06 2022-10-06 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
US20240182892A1 (en) * 2021-03-24 2024-06-06 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO
EP4634385A2 (en) * 2022-12-16 2025-10-22 Amgen Inc. Rnai constructs for inhibiting ttr expression and methods of use thereof
TW202530408A (zh) 2023-09-29 2025-08-01 美商英西特羅公司 用於治療非酒精性脂肪肝病的組合物與方法
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
WO2025113470A1 (en) * 2023-11-27 2025-06-05 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of transthyretin (ttr)
US20250333742A1 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease

Family Cites Families (80)

* 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
WO2005121370A2 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Oligomeric compounds that facilitate risc loading
AU2001247951A1 (en) 2000-02-07 2001-08-14 Roche Diagnostics Corporation Novel cationic amphiphiles
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
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
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
WO2002059621A2 (en) 2001-01-24 2002-08-01 Bayer Corporation Regulation of transthyretin to treat obesity
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)
WO2003001182A2 (en) 2001-06-21 2003-01-03 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
DK1527176T4 (en) 2002-08-05 2017-07-03 Silence Therapeutics Gmbh ADDITIONAL NEW FORMS OF INTERFERRING 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)
AU2003301882A1 (en) 2002-11-01 2004-06-07 The Regents Of The University Of Colorado, A Body Corporate 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
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
EP2216407B1 (en) 2003-03-07 2016-01-13 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
CA2488224A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals, Inc. Irna conjugates
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
EP1781593B1 (en) 2004-06-07 2011-12-14 Protiva Biotherapeutics Inc. Cationic lipids and methods of use
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
US7838561B2 (en) 2004-06-14 2010-11-23 Wisconsin Alumni Research Foundation Method for preventing or treating cardiac hypertrophy
CN101129344A (zh) 2004-12-08 2008-02-27 西来昂诊疗公司 治疗与视黄醇有关的疾病的方法、分析和组合物
WO2007086883A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
ATE541928T1 (de) 2005-03-31 2012-02-15 Calando Pharmaceuticals Inc Inhibitoren der untereinheit 2 der ribonukleotidreduktase und ihre verwendungen
US7915230B2 (en) 2005-05-17 2011-03-29 Molecular Transfer, Inc. Reagents for transfection of eukaryotic cells
EP1937213B1 (en) 2005-07-27 2017-10-25 Protiva Biotherapeutics Inc. Systems and methods for manufacturing liposomes
CA2628300C (en) 2005-11-02 2018-04-17 Protiva Biotherapeutics, Inc. Modified sirna molecules and uses thereof
CN101448849B (zh) 2006-03-31 2013-08-21 阿尔尼拉姆医药品有限公司 抑制Eg5基因表达的组合物和方法
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
MX2009003548A (es) 2006-10-03 2009-04-15 Alnylam Pharmaceuticals Inc Formulaciones que contienen lipidos.
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
EP4074344A1 (en) 2007-12-04 2022-10-19 Arbutus Biopharma Corporation Targeting lipids
ES2535419T3 (es) 2007-12-27 2015-05-11 Protiva Biotherapeutics Inc. Silenciamiento de expresión de quinasa tipo polo usando ARN interferente
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
JP2011511004A (ja) 2008-01-31 2011-04-07 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 PCSK9遺伝子を標的とするdsRNAを送達するための最適化された方法
US20100323381A1 (en) 2008-02-08 2010-12-23 Bergen Iii Harold R Classifying amyloidosis
ES2638448T3 (es) 2008-04-15 2017-10-20 Protiva Biotherapeutics Inc. Novedosas formulaciones de lípidos para la administración de ácidos nucleicos
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
EP2743265B1 (en) 2008-10-09 2017-03-15 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
PT2937418T (pt) 2008-10-20 2018-01-23 Alnylam Pharmaceuticals Inc Composições e métodos de inibição da expressão de transtirretina
US8969353B2 (en) 2008-11-07 2015-03-03 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
EA037404B1 (ru) 2008-11-10 2021-03-24 Арбутус Биофарма Корпорэйшн Липиды и композиции для доставки лекарственных средств
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
EP2391343B1 (en) 2009-01-29 2017-03-01 Arbutus Biopharma Corporation Improved lipid formulation for the delivery of nucleic acids
CA3042927C (en) 2009-05-05 2022-05-17 Arbutus Biopharma Corporation Lipid compositions for the delivery of therapeutic agents
HRP20211619T1 (hr) 2009-06-10 2022-02-04 Arbutus Biopharma Corporation Poboljšana formulacija lipida
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
EP2496238A4 (en) 2009-11-03 2013-10-02 Alnylam Pharmaceuticals Inc FORMULATED LIPID COMPOSITIONS AND METHODS FOR INHIBITING TRANSTHYRETIN EXPRESSION (TTR)
ES2893199T3 (es) 2010-03-29 2022-02-08 Alnylam Pharmaceuticals Inc Terapia de ARNbc para amiloidosis ocular relacionada con transtiretina (TTR)
US20230340473A1 (en) 2010-03-29 2023-10-26 Alnylam Pharmaceuticals, Inc. Sirna therapy for transthyretin (ttr) related ocular amyloidosis
MX343559B (es) 2010-04-29 2016-11-10 Ionis Pharmaceuticals Inc Modulacion de la expresion de transtiretina.
JP2013541334A (ja) 2010-09-15 2013-11-14 アルニラム ファーマスーティカルズ インコーポレイテッド 修飾されたiRNA剤
EP3327125B1 (en) 2010-10-29 2020-08-05 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
WO2012064824A1 (en) 2010-11-09 2012-05-18 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
AU2012272908A1 (en) 2011-06-21 2013-12-19 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
PE20142362A1 (es) 2011-11-18 2015-01-30 Alnylam Pharmaceuticals Inc Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr)
DK3366775T4 (da) * 2011-11-18 2025-10-27 Alnylam Pharmaceuticals Inc Modificerede rnai-midler
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
WO2014022739A2 (en) 2012-08-03 2014-02-06 Alnylam Pharmaceuticals, Inc. Modified rnai agents
TW201610151A (zh) * 2013-09-23 2016-03-16 阿尼拉製藥公司 治療或預防與甲狀腺素運載蛋白相關疾病之方法
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
BR112016013148B1 (pt) * 2013-12-12 2024-02-27 Alnylam Pharmaceuticals, Inc ÁCIDO RIBONUCLEICO DE FITA DUPLA (dsRNA) PARA INIBIÇÃO DA EXPRESSÃO DO FATOR B DO COMPLEMENTO (CFB) EM UMA CÉLULA, COMPOSIÇÃO FARMACÊUTICA PARA INIBIÇÃO DA EXPRESSÃO DE UM GENE DO FATOR B DO COMPLEMENTO, SEU USO, E MÉTODO IN VITRO DE INIBIÇÃO DA EXPRESSÃO DO FATOR B DO COMPLEMENTO (CFB) EM UMA CÉLULA
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
DK3185957T3 (da) 2014-08-29 2022-08-29 Alnylam Pharmaceuticals Inc Patisiran til anvendelse ved behandlingen af transthyretin-medieret amyloidosis
US11117877B2 (en) 2014-11-21 2021-09-14 Bsim Therapeutics, S.A. 2-thioxothiazolidin-4-one derivatives active as transthyretin ligands and uses thereof
ES2842300T3 (es) 2015-07-31 2021-07-13 Alnylam Pharmaceuticals Inc Composiciones de ARNi de transtiretina (TTR) y métodos para su uso para el tratamiento o la prevención de enfermedades asociadas con TTR
EP3555292A1 (en) 2016-12-16 2019-10-23 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
US11806360B2 (en) 2017-09-19 2023-11-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
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
AU2021232029A1 (en) 2020-03-06 2022-10-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin (TTR)
EP4334448A1 (en) 2021-05-03 2024-03-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO

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