JP2024028749A5 - - Google Patents

Download PDF

Info

Publication number
JP2024028749A5
JP2024028749A5 JP2023199064A JP2023199064A JP2024028749A5 JP 2024028749 A5 JP2024028749 A5 JP 2024028749A5 JP 2023199064 A JP2023199064 A JP 2023199064A JP 2023199064 A JP2023199064 A JP 2023199064A JP 2024028749 A5 JP2024028749 A5 JP 2024028749A5
Authority
JP
Japan
Prior art keywords
salt
double
pharmaceutical composition
stranded
rnai agent
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2023199064A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024028749A (ja
JP7711150B2 (ja
Filing date
Publication date
Priority claimed from JP2018504819A external-priority patent/JP6896703B2/ja
Application filed filed Critical
Publication of JP2024028749A publication Critical patent/JP2024028749A/ja
Publication of JP2024028749A5 publication Critical patent/JP2024028749A5/ja
Priority to JP2025115513A priority Critical patent/JP2025165939A/ja
Application granted granted Critical
Publication of JP7711150B2 publication Critical patent/JP7711150B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2023199064A 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 Active JP7711150B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
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 (6)

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
JP2018504819A 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 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

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025115513A Division 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

Publications (3)

Publication Number Publication Date
JP2024028749A JP2024028749A (ja) 2024-03-05
JP2024028749A5 true JP2024028749A5 (enExample) 2024-07-03
JP7711150B2 JP7711150B2 (ja) 2025-07-22

Family

ID=56682264

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

Family Applications Before (2)

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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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 (enExample)
EP (1) EP3329002B1 (enExample)
JP (4) JP6896703B2 (enExample)
KR (2) KR20240074895A (enExample)
CN (2) CN108138182B (enExample)
BR (1) BR112018000542B1 (enExample)
CA (1) CA2994285A1 (enExample)
CL (1) CL2018000198A1 (enExample)
CY (1) CY1124545T1 (enExample)
DK (1) DK3329002T3 (enExample)
EA (1) EA201890394A1 (enExample)
ES (1) ES2842300T3 (enExample)
FI (1) FIC20230006I1 (enExample)
FR (1) FR23C1008I2 (enExample)
HK (1) HK1256683A1 (enExample)
HR (1) HRP20202082T1 (enExample)
HU (2) HUE051998T2 (enExample)
IL (3) IL313082A (enExample)
LT (2) LT3329002T (enExample)
LU (1) LUC00297I2 (enExample)
MA (1) MA43335B1 (enExample)
MX (2) MX2018000981A (enExample)
MY (1) MY195720A (enExample)
NL (1) NL301216I2 (enExample)
NO (1) NO2023007I1 (enExample)
PL (1) PL3329002T3 (enExample)
PT (1) PT3329002T (enExample)
RS (1) RS61297B1 (enExample)
SG (1) SG10201912341SA (enExample)
SI (1) SI3329002T1 (enExample)
SM (1) SMT202100020T1 (enExample)
TW (4) TWI727963B (enExample)
UA (1) UA126276C2 (enExample)
WO (1) WO2017023660A1 (enExample)
ZA (2) ZA201800571B (enExample)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20150796T1 (hr) 2008-10-20 2015-09-11 Alnylam Pharmaceuticals, Inc. Pripravci i postupci za inhibiciju ekspresije transtiretina
RU2015151857A (ru) 2008-12-02 2019-01-15 Уэйв Лайф Сайенсес Джапан, Инк. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
NZ603339A (en) 2010-04-29 2015-01-30 Isis Pharmaceuticals Inc Modulation of transthyretin expression
US10428019B2 (en) 2010-09-24 2019-10-01 Wave Life Sciences Ltd. Chiral auxiliaries
EP3248982A1 (en) 2011-07-19 2017-11-29 Wave Life Sciences Ltd. Thiosulfonate reagents for the synthesis of functionalized nucleic acids
KR20250133484A (ko) * 2011-11-18 2025-09-05 알닐람 파마슈티칼스 인코포레이티드 변형된 RNAi 제제
MY167390A (en) 2011-11-18 2018-08-16 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
CA2879023C (en) 2012-07-13 2017-03-28 Wave Life Sciences Japan 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
MX2016009290A (es) 2014-01-16 2017-02-28 Wave Life Sciences Ltd Diseño quiral.
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
KR20190098181A (ko) * 2016-12-16 2019-08-21 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물을 사용하여 TTR-관련 질병을 치료하거나 예방하는 방법
MA50267A (fr) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals Inc Compositions et méthodes de traitement de l'amylose médiée par la transthyrétine (ttr)
WO2019105418A1 (zh) * 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
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
JP2023516748A (ja) * 2020-03-06 2023-04-20 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)の発現を阻害するための組成物および方法
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
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
MX2024001194A (es) 2021-08-03 2024-02-27 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de transtiretina (ttr) y sus metodos de uso.
IL321194A (en) * 2022-12-16 2025-07-01 Amgen Inc RNA interference constructs for inhibiting TTR expression and methods for using them
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
EP1261620A2 (en) 2000-02-07 2002-12-04 Roche Diagnostics Corporation Cationic amphiphiles for use in nucleic acid transfection
US20070026394A1 (en) 2000-02-11 2007-02-01 Lawrence Blatt 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
RU2322500C2 (ru) 2000-12-01 2008-04-20 Макс-Планк-Гезелльшафт Цур Фердерунг Дер Виссеншафтен Е.Ф. Малые молекулы рнк, опосредующие интерференцию рнк
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)
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
ES2280826T5 (es) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Nuevas formas adicionales de moléculas de ARN de interferencia
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)
JP2006504971A (ja) 2002-11-01 2006-02-09 ザ・リージェンツ・オブ・ザ・ユニバーシティ・オブ・コロラド,ア・ボディー・コーポレイト マトリックス支援レーザー脱離イオン化−飛行時間型質量分析によるタンパク質アイソフォームの定量的解析
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
EP3450559A1 (en) 2003-03-07 2019-03-06 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
EP1608735A4 (en) 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
CA2568735A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
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
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
US7838561B2 (en) 2004-06-14 2010-11-23 Wisconsin Alumni Research Foundation Method for preventing or treating cardiac hypertrophy
SG144145A1 (en) 2004-12-08 2008-07-29 Sirion Therapeutics Inc Methods, assays and compositions for treating retinol-related diseases
WO2007086883A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
US7427605B2 (en) 2005-03-31 2008-09-23 Calando Pharmaceuticals, Inc. Inhibitors of ribonucleotide reductase subunit 2 and uses thereof
US7915230B2 (en) 2005-05-17 2011-03-29 Molecular Transfer, Inc. Reagents for transfection of eukaryotic cells
JP5639338B2 (ja) 2005-07-27 2014-12-10 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 リポソームの製造システムおよび製造方法
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2007115168A2 (en) 2006-03-31 2007-10-11 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
WO2009002944A1 (en) 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
EP3705125B1 (en) 2007-12-04 2023-07-05 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2710713C (en) 2007-12-27 2017-09-19 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
US20110117125A1 (en) 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids
EP2245039A4 (en) 2008-01-31 2012-06-06 Alnylam Pharmaceuticals Inc OPTIMIZED METHODS FOR EXPORING DSRNA TO TARGET THE PCSK9 GEN
US20100323381A1 (en) 2008-02-08 2010-12-23 Bergen Iii Harold R Classifying amyloidosis
US8058069B2 (en) 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
TWI455944B (zh) 2008-07-01 2014-10-11 Daiichi Sankyo Co Ltd 雙股多核苷酸
WO2010017509A1 (en) 2008-08-07 2010-02-11 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
WO2010042877A1 (en) 2008-10-09 2010-04-15 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
HRP20150796T1 (hr) 2008-10-20 2015-09-11 Alnylam Pharmaceuticals, Inc. Pripravci i postupci za inhibiciju ekspresije transtiretina
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
CN105152939A (zh) 2008-11-10 2015-12-16 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
KR20210031549A (ko) 2009-05-05 2021-03-19 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
KR20230098713A (ko) 2009-06-10 2023-07-04 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
EP2442792A4 (en) 2009-06-15 2015-12-23 Alnylam Pharmaceuticals Inc TESTING GENE PCSK9 LIPID-FORMULATED DSRNA
JP5723378B2 (ja) 2009-11-03 2015-05-27 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. トランスサイレチン(ttr)を阻害する脂質製剤化組成物及び方法
JP5860029B2 (ja) 2010-03-29 2016-02-16 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. トランスチレチン(TTR)関連眼アミロイドーシスのためのsiRNA療法
US20230340473A1 (en) 2010-03-29 2023-10-26 Alnylam Pharmaceuticals, Inc. Sirna therapy for transthyretin (ttr) related ocular amyloidosis
NZ603339A (en) 2010-04-29 2015-01-30 Isis Pharmaceuticals Inc Modulation of transthyretin expression
EP2616543A1 (en) 2010-09-15 2013-07-24 Alnylam Pharmaceuticals, Inc. MODIFIED iRNA AGENTS
US9260471B2 (en) 2010-10-29 2016-02-16 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
CN103370054A (zh) 2010-11-09 2013-10-23 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
CA2839896A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
MY167390A (en) * 2011-11-18 2018-08-16 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
KR20250133484A (ko) * 2011-11-18 2025-09-05 알닐람 파마슈티칼스 인코포레이티드 변형된 RNAi 제제
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
AR097738A1 (es) * 2013-09-23 2016-04-13 Alnylam Pharmaceuticals Inc Métodos para tratar o prevenir enfermedades asociadas con la transtiretina (ttr)
WO2015085158A1 (en) 2013-12-06 2015-06-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of transthyretin (ttr) by double-stranded rna
KR102344559B1 (ko) * 2013-12-12 2021-12-31 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
DK3185957T3 (da) 2014-08-29 2022-08-29 Alnylam Pharmaceuticals Inc Patisiran til anvendelse ved behandlingen af transthyretin-medieret amyloidosis
EP3221299A1 (en) 2014-11-21 2017-09-27 BSIM2 Biomolecular Simulations Lda. 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
KR20190098181A (ko) 2016-12-16 2019-08-21 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물을 사용하여 TTR-관련 질병을 치료하거나 예방하는 방법
MA50267A (fr) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals Inc Compositions et méthodes de traitement de l'amylose médiée par la transthyrétine (ttr)
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
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
JP2023516748A (ja) 2020-03-06 2023-04-20 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)の発現を阻害するための組成物および方法
WO2022235537A1 (en) 2021-05-03 2022-11-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
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
MX2024001194A (es) 2021-08-03 2024-02-27 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de transtiretina (ttr) y sus metodos de uso.

Similar Documents

Publication Publication Date Title
JP2024028749A5 (enExample)
JP2018523655A5 (enExample)
US11884920B2 (en) Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
HRP20202082T1 (hr) Transtiretin (ttr) irna pripravci i postupci njihove upotrebe za liječenje ili sprječavanje bolesti povezanih s ttr
JP2020502142A5 (enExample)
JP2023103244A5 (enExample)
ES2988612T3 (es) Agentes de ARNi para la infección por el virus de la hepatitis B
IL308752B1 (en) Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
JP2016506240A5 (enExample)
CA2858576A1 (en) Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
KR20250046303A (ko) 안지오텐시노겐 (agt)을 표적화하는 이중 가닥 rna 및 그의 사용 방법
WO2023220561A1 (en) Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
JPWO2021188795A5 (enExample)
JPWO2021167841A5 (enExample)
CN112055597A (zh) 用于治疗胆管缺乏相关病况的方法和组合物
JPWO2022218941A5 (enExample)
JPWO2022187435A5 (enExample)
JPWO2023014677A5 (enExample)
JPWO2021178778A5 (enExample)
US20240309383A1 (en) Small interfering rna targeting hbv and uses thereof
JPWO2023278607A5 (enExample)
US20240294916A1 (en) Small interfering rna targeting cfb and uses thereof
RU2024104666A (ru) КОМПОЗИЦИИ НА ОСНОВЕ иРНК ДЛЯ ТРАНСТИРЕТИНА (TTR) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
CN115141848A (zh) 一种用于治疗亨廷顿病的rna递送系统
JPWO2022260939A5 (enExample)