UY37881A - Agentes de iarn y composiciones para inhibir la expresión de la angiopoyetina tipo 3 (angptl3) y métodos de uso - Google Patents

Agentes de iarn y composiciones para inhibir la expresión de la angiopoyetina tipo 3 (angptl3) y métodos de uso

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Publication number
UY37881A
UY37881A UY0001037881A UY37881A UY37881A UY 37881 A UY37881 A UY 37881A UY 0001037881 A UY0001037881 A UY 0001037881A UY 37881 A UY37881 A UY 37881A UY 37881 A UY37881 A UY 37881A
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UY
Uruguay
Prior art keywords
angptl3
agents
compositions
expression
methods
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Application number
UY0001037881A
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English (en)
Inventor
Zhen Li
Rui Zhu
So Wong
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Arrowhead Pharmaceuticals Inc
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Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of UY37881A publication Critical patent/UY37881A/es

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    • C12N15/09Recombinant DNA-technology
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    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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Abstract

La presente divulgación se refiere a agentes de iARN, por ejemplo, agentes de iARN de cadena doble, capaces de inhibir la expresión del gen de la Angiopoyetina tipo 3 (también denominada ANGPTL3, ANGPL3, proteína angiopoyetina tipo 3), y composiciones que incluyen agentes de iARN de ANGPTL3. También se divulgan métodos para usar los agentes de iARN de ANGPTL3 y composiciones. Los agentes de iARN de ANGPTL3 divulgados en la presente pueden estar conjugados con ligandos de direccionamiento para facilitar la administración a células, inclusive a hepatocitos.
UY0001037881A 2017-09-14 2018-09-14 Agentes de iarn y composiciones para inhibir la expresión de la angiopoyetina tipo 3 (angptl3) y métodos de uso UY37881A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762558819P 2017-09-14 2017-09-14
US201762583919P 2017-11-09 2017-11-09
US201862651284P 2018-04-02 2018-04-02
US201862694976P 2018-07-07 2018-07-07

Publications (1)

Publication Number Publication Date
UY37881A true UY37881A (es) 2019-03-29

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UY0001037881A UY37881A (es) 2017-09-14 2018-09-14 Agentes de iarn y composiciones para inhibir la expresión de la angiopoyetina tipo 3 (angptl3) y métodos de uso

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US (2) US10995335B2 (es)
EP (1) EP3681513A4 (es)
JP (2) JP7281452B2 (es)
KR (1) KR20200044837A (es)
CN (2) CN111343994B (es)
AU (1) AU2018331383A1 (es)
BR (1) BR112020003126A2 (es)
CA (1) CA3074320A1 (es)
CL (1) CL2020000666A1 (es)
CO (1) CO2020001740A2 (es)
EC (1) ECSP20018305A (es)
IL (1) IL273173A (es)
JO (1) JOP20200050A1 (es)
MX (1) MX2020001912A (es)
PE (1) PE20201287A1 (es)
PH (1) PH12020500309A1 (es)
SG (1) SG11201912188RA (es)
TN (1) TN2020000038A1 (es)
TW (1) TWI823866B (es)
UY (1) UY37881A (es)
WO (1) WO2019055633A1 (es)
ZA (1) ZA202103669B (es)

Families Citing this family (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10201913683WA (en) 2011-06-21 2020-03-30 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
CN116832169A (zh) * 2016-09-02 2023-10-03 箭头药业股份有限公司 靶向配体
PL3607069T3 (pl) 2017-04-05 2023-03-06 Silence Therapeutics Gmbh Produkty i kompozycje
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CN114728018B (zh) 2019-11-01 2024-07-19 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
CA3161703A1 (en) 2019-11-13 2021-05-20 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
WO2021119226A1 (en) 2019-12-13 2021-06-17 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
WO2021126734A1 (en) 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
CA3171654A1 (en) 2020-02-18 2021-08-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
AU2021232014A1 (en) 2020-03-06 2022-10-06 Alnylam Pharmaceuticals, Inc. Ketohexokinase (KHK) IRNA compositions and methods of use thereof
CN116096889A (zh) * 2020-03-18 2023-05-09 迪克纳制药公司 用于抑制angptl3表达的组合物和方法
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
KR20220158011A (ko) * 2020-03-26 2022-11-29 애로우헤드 파마슈티컬스 인코포레이티드 PNPLA3의 발현을 억제하기 위한 RNAi 작용제, 이의 약제학적 조성물, 및 사용 방법
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
JP2023523993A (ja) 2020-04-27 2023-06-08 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 アポリポタンパク質E(ApoE)iRNA剤組成物およびその使用方法
AU2021265813A1 (en) 2020-04-30 2022-11-10 Alnylam Pharmaceuticals, Inc. Complement factor B (CFB) iRNA compositions and methods of use thereof
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
MX2022015149A (es) 2020-06-18 2023-01-11 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de xantina dehidrogenasa (xdh) y metodos de uso de las mismas.
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
JP2023536685A (ja) * 2020-09-30 2023-08-29 納▲タイ▼得(青島)生物医薬有限公司 アンギオポエチン様3(ANGPTL3)のsiRNA及びその用途
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
JP2023546103A (ja) * 2020-10-16 2023-11-01 サノフイ Angptl3を阻害するための新規のrna組成物および方法
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
WO2022103999A1 (en) 2020-11-13 2022-05-19 Alnylam Pharmaceuticals, Inc. COAGULATION FACTOR V (F5) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
IL304880A (en) 2021-02-12 2023-10-01 Alnylam Pharmaceuticals Inc Superoxide dismutase 1 (SOD1) IRNA compositions and methods of using them to treat or prevent superoxide dismutase 1- (SOD1-) associated neurodegenerative diseases
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
KR20230150844A (ko) 2021-02-26 2023-10-31 알닐람 파마슈티칼스 인코포레이티드 케토헥소키나아제(KHK) iRNA 조성물 및 이의 사용 방법
IL305414A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
MX2023011466A (es) 2021-03-29 2024-02-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de ácido ribonucleico de interferencia (arni) de huntingtina (htt) y métodos de uso de estas.
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
CA3216106A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
JP2024519293A (ja) 2021-04-29 2024-05-10 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 シグナル伝達兼転写活性化因子6(STAT6)iRNA組成物およびその使用方法
JP2024522068A (ja) 2021-05-18 2024-06-11 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 ナトリウム-グルコース共輸送体2(sglt2)irna組成物およびその使用方法
TW202317762A (zh) 2021-06-02 2023-05-01 美商艾拉倫製藥股份有限公司 含有類PATATIN磷脂酶結構域3(PNPLA3)的iRNA組成物及其使用方法
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
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
JP2024527304A (ja) 2021-06-30 2024-07-24 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 アンジオテンシノーゲン(agt)関連障害を治療するための方法および組成物
TW202333748A (zh) 2021-07-19 2023-09-01 美商艾拉倫製藥股份有限公司 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物
MX2024000981A (es) 2021-07-21 2024-02-12 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de gen diana asociado con trastorno metabolico y sus metodos de uso.
IL309905A (en) 2021-07-23 2024-03-01 Alnylam Pharmaceuticals Inc IRNA compositions in β-catenin (CTNNB1) and methods of using them
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
KR20240042004A (ko) 2021-08-03 2024-04-01 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물 및 이의 사용 방법
WO2023014765A1 (en) 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
AU2022328347A1 (en) 2021-08-13 2024-02-08 Alnylam Pharmaceuticals, Inc. Factor xii (f12) irna compositions and methods of use thereof
JP2024535850A (ja) 2021-09-17 2024-10-02 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 補体成分(C3)をサイレンシングするためのiRNA組成物および方法
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
KR20240053627A (ko) * 2021-09-23 2024-04-24 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. 안지오포이에틴-유사 3(angptl3) 단백질의 발현을 억제하기 위한 조성물 및 방법
WO2023064530A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
WO2023076451A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
US20240271141A1 (en) * 2021-11-29 2024-08-15 Shanghai Argo Biopharmaceutical Co., Ltd. Composition and method for inhibiting expression of hepatitis b virus (hbv)protein
WO2023122656A1 (en) 2021-12-22 2023-06-29 Regeneron Pharmaceuticals, Inc. Treatment of kidney diseases with angiopoietin like 3 (angptl3) inhibitors
WO2023134705A1 (zh) * 2022-01-11 2023-07-20 上海金中锘美生物医药科技有限公司 抑制angptl3表达的rna干扰剂及其用途
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023196941A1 (en) * 2022-04-08 2023-10-12 Arrowhead Pharmaceuticals, Inc. Treatment of a non-alcoholic fatty liver disease
CN116987696A (zh) * 2022-08-04 2023-11-03 北京福元医药股份有限公司 用于抑制angptl3基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2024032608A1 (zh) * 2022-08-08 2024-02-15 大睿生物医药科技(上海)有限公司 调控ANGPTL3基因活性的siRNA分子
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024044542A2 (en) * 2022-08-22 2024-02-29 Arrowhead Pharmaceuticals, Inc. Methods for the treatment of angptl3-related diseases and disorders
US20240158795A1 (en) 2022-11-10 2024-05-16 Regeneron Pharmaceuticals, Inc. Treatment Of Kidney Diseases With A Combination of Angiopoietin Like 3 (ANGPTL3) Inhibitors And Solute Carrier Family 5 Member 2 (SLC5A2) Inhibitors
WO2024164008A1 (en) * 2023-02-03 2024-08-08 Sirnaomics, Inc. Products and compositions
WO2024188173A1 (zh) * 2023-03-10 2024-09-19 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及其用途

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
ATE408699T1 (de) 1999-03-10 2008-10-15 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
CZ20021853A3 (cs) 1999-12-09 2002-11-13 Sankyo Company, Limited Způsob testování účinku látky, polynukleotid, DNA, protilátka, souprava pro testování, pouľití polynukleotidu, pouľití polypeptidu, pouľití protilátky, RNA a terapeutické činidlo
AU2002348286B2 (en) 2001-11-16 2008-04-03 Genentech, Inc. Composition comprising and method of using angiopoietin-like protein 3 Angptl3
AU2003282877B9 (en) * 2002-09-25 2011-05-12 University Of Massachusetts In Vivo gene silencing by chemically modified and stable siRNA
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
US20040198640A1 (en) 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
US20040224405A1 (en) 2003-05-06 2004-11-11 Dharmacon Inc. siRNA induced systemic gene silencing in mammalian systems
US20070276134A1 (en) * 2006-05-24 2007-11-29 Nastech Pharmaceutical Company Inc. Compositions and methods for complexes of nucleic acids and organic cations
MX2009001207A (es) 2006-08-18 2009-02-11 Hoffmann La Roche Policonjugados para el suministro in vivo de polinucleotidos.
CA3043911A1 (en) 2007-12-04 2009-07-02 Arbutus Biopharma Corporation Targeting lipids
JP2013511990A (ja) * 2009-11-26 2013-04-11 クォーク ファーマシューティカルズ インコーポレーティッド 末端置換を含むsiRNA化合物
AU2011203986C1 (en) * 2010-01-08 2015-03-05 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
PT2539451E (pt) 2010-02-24 2016-03-28 Arrowhead Res Corp Composições para entrega de arnsi dirigida ao alvo
ES2663009T3 (es) 2010-10-29 2018-04-10 Sirna Therapeutics, Inc. Inhibición de la expresión génica mediada por interferencia por ARN utilizando ácidos nucleicos de interferencia cortos (ANic)
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
SG10201913683WA (en) * 2011-06-21 2020-03-30 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
US8932572B2 (en) 2011-08-26 2015-01-13 Arrowhead Madison Inc. Poly(vinyl ester) polymers for in vivo nucleic acid delivery
EA201490993A1 (ru) * 2011-11-18 2014-09-30 Элнилэм Фармасьютикалз, Инк. МОДИФИЦИРОВАННЫЕ СРЕДСТВА РНКи
KR20150001710A (ko) 2012-04-18 2015-01-06 애로우헤드 리서치 코오포레이션 In Vivo 핵산 전달용 폴리(아크릴레이트) 고분자
EP3919620A1 (en) * 2012-05-02 2021-12-08 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
CA2931510A1 (en) 2013-12-24 2015-07-02 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
EP3137605B1 (en) * 2014-05-01 2020-10-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
SG10201903290YA (en) 2014-08-20 2019-05-30 Alnylam Pharmaceuticals Inc Modified double-stranded rna agents
US9803205B2 (en) * 2015-03-17 2017-10-31 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of factor XII
US20180245077A1 (en) 2015-03-20 2018-08-30 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
CN115927335A (zh) 2015-04-13 2023-04-07 阿尔尼拉姆医药品有限公司 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
EP3426261A4 (en) 2016-03-07 2020-03-25 Arrowhead Pharmaceuticals, Inc. TARGETED LIGANDS FOR THERAPEUTIC CONNECTIONS
MA45328A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45340A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques du récepteur des androgènes et leurs utilisations
CN116832169A (zh) 2016-09-02 2023-10-03 箭头药业股份有限公司 靶向配体

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