MX2020002648A - Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3). - Google Patents

Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3).

Info

Publication number
MX2020002648A
MX2020002648A MX2020002648A MX2020002648A MX2020002648A MX 2020002648 A MX2020002648 A MX 2020002648A MX 2020002648 A MX2020002648 A MX 2020002648A MX 2020002648 A MX2020002648 A MX 2020002648A MX 2020002648 A MX2020002648 A MX 2020002648A
Authority
MX
Mexico
Prior art keywords
apoc3
rnai agents
iii
expression
apolipoprotein
Prior art date
Application number
MX2020002648A
Other languages
English (en)
Spanish (es)
Inventor
So Wong
Steven Kanner
Tao Pei
Zhen Li
Rui Zhu
Original Assignee
Arrowhead Pharmaceuticals Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of MX2020002648A publication Critical patent/MX2020002648A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H3/00—Compounds containing only hydrogen atoms and saccharide radicals having only carbon, hydrogen, and oxygen atoms
    • C07H3/02—Monosaccharides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Biophysics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
MX2020002648A 2017-09-11 2018-09-10 Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3). MX2020002648A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762556818P 2017-09-11 2017-09-11
US201862643927P 2018-03-16 2018-03-16
US201862720434P 2018-08-21 2018-08-21
PCT/US2018/050248 WO2019051402A1 (en) 2017-09-11 2018-09-10 RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)

Publications (1)

Publication Number Publication Date
MX2020002648A true MX2020002648A (es) 2020-07-22

Family

ID=65630632

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020002648A MX2020002648A (es) 2017-09-11 2018-09-10 Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3).

Country Status (24)

Country Link
US (4) US10597657B2 (OSRAM)
EP (1) EP3681516A4 (OSRAM)
JP (2) JP7438103B2 (OSRAM)
KR (2) KR102723833B1 (OSRAM)
CN (2) CN118879696A (OSRAM)
AU (1) AU2018329190B2 (OSRAM)
BR (1) BR112020002413A2 (OSRAM)
CA (1) CA3074303A1 (OSRAM)
CL (2) CL2020000593A1 (OSRAM)
CO (1) CO2020002586A2 (OSRAM)
CR (1) CR20200108A (OSRAM)
EC (1) ECSP20017905A (OSRAM)
IL (2) IL273172B1 (OSRAM)
JO (1) JOP20200054A1 (OSRAM)
MX (1) MX2020002648A (OSRAM)
MY (1) MY202025A (OSRAM)
PE (1) PE20201283A1 (OSRAM)
PH (1) PH12020500332A1 (OSRAM)
SG (1) SG11201912178VA (OSRAM)
TN (1) TN2020000039A1 (OSRAM)
TW (2) TW202415389A (OSRAM)
UY (1) UY37871A (OSRAM)
WO (1) WO2019051402A1 (OSRAM)
ZA (1) ZA202100806B (OSRAM)

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3221451A1 (en) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
IL300869A (en) * 2016-09-02 2023-04-01 Arrowhead Pharmaceuticals Inc Ligands containing a targeting factor, preparations containing them and their uses
EP4219713A3 (en) 2017-04-05 2023-08-16 Silence Therapeutics GmbH Products and compositions
EP3681516A4 (en) * 2017-09-11 2021-09-29 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)
JP7365052B2 (ja) 2017-12-01 2023-10-19 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
EP3903830A4 (en) * 2018-12-28 2023-04-26 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH NUCLEIC ACID, PROCESS FOR THEIR PREPARATION AND ITS USE
AU2020280438B2 (en) 2019-05-22 2025-03-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
MX2022001460A (es) * 2019-08-05 2022-02-22 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de enfermedades y trastornos relacionados con apoc3.
CA3171654A1 (en) * 2020-02-18 2021-08-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
CN114480382A (zh) * 2020-11-12 2022-05-13 北京大学 用于抑制SURF4基因表达的RNAi试剂和组合物
EP4176061A1 (en) * 2021-01-30 2023-05-10 E-Therapeutics plc Nucleic acids containing abasic nucleotides
JP2024522882A (ja) * 2021-06-24 2024-06-21 サーナオミクス インコーポレイテッド 製造物および組成物
KR20240034787A (ko) * 2021-07-16 2024-03-14 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 함유하는 조성물 및 접합체, 제조 방법 및 용도
EP4413130A1 (en) * 2021-10-08 2024-08-14 E-Therapeutics plc Nucleic acids containing abasic nucleosides
CN118355120A (zh) 2021-12-01 2024-07-16 迪克纳制药公司 用于调节apoc3表达的组合物和方法
CA3243093A1 (en) * 2022-01-20 2025-04-08 Tuojie Biotech (Shanghai) Co., Ltd. DOUBLE-STRANDED RNA, ITS USES AND PREPARATION PROCESS
TW202345865A (zh) * 2022-01-24 2023-12-01 大陸商上海舶望製藥有限公司 抑制LPA(Apo(a))蛋白表達的組合物和方法
CN116917477B (zh) 2022-01-30 2024-04-09 大睿生物医药科技(上海)有限公司 含有n-乙酰半乳糖胺的靶向配体
CN119487191A (zh) 2022-06-14 2025-02-18 大睿生物医药科技(上海)有限公司 调控PCSK9基因活性的siRNA分子
EP4545641A1 (en) 2022-06-27 2025-04-30 Rona Bioscience, Limited Sirna for inhibiting apolipoprotein c3 expression
IL317824A (en) * 2022-07-11 2025-02-01 Sanegene Bio Usa Inc Ribose derivatives modified at the '2' position and methods of use
AU2023313318A1 (en) * 2022-07-27 2025-01-09 E-Therapeutics Plc Nucleic acid compounds
AU2023313030A1 (en) * 2022-07-27 2025-01-09 E-Therapeutics Plc Nucleic acid compounds
WO2024023262A2 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
CN116814621B (zh) * 2022-08-05 2025-09-09 厦门甘宝利生物医药有限公司 一种抑制apoc3基因表达的rna抑制剂及其应用
WO2024032680A1 (zh) * 2022-08-11 2024-02-15 益杰立科(上海)生物科技有限公司 一种表观编辑靶点的方法及用途
WO2024165571A2 (en) * 2023-02-06 2024-08-15 E-Therapeutics Plc Inhibitors of expression and/or function
CN121219413A (zh) * 2023-02-06 2025-12-26 e-生物有限公司 表达和/或功能的抑制剂
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
WO2025059466A1 (en) 2023-09-14 2025-03-20 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing apociii expression
CN120344664A (zh) * 2023-10-27 2025-07-18 北京安龙生物医药有限公司 靶向脂蛋白a的寡核苷酸及其用途
WO2025125576A2 (en) * 2023-12-15 2025-06-19 E-Therapeutics Plc Inhibitors of expression and/or function
WO2025133348A1 (en) * 2023-12-22 2025-06-26 E-Therapeutics Plc Inhibitors of expression and/or function
WO2025184301A1 (en) * 2024-02-29 2025-09-04 Arrowhead Pharmaceuticals, Inc. Rnai agents for dual inhibition of expression of apolipoprotein c-iii (apoc3) and proprotein convertase subtilisin kexin 9 (pcsk9), compositions thereof, and methods of use

Family Cites Families (49)

* 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
US5030453A (en) 1983-03-24 1991-07-09 The Liposome Company, Inc. Stable plurilamellar vesicles
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
US6977244B2 (en) 1996-10-04 2005-12-20 Board Of Regents, The University Of Texas Systems Inhibition of Bcl-2 protein expression by liposomal antisense oligodeoxynucleotides
US5891467A (en) 1997-01-31 1999-04-06 Depotech Corporation Method for utilizing neutral lipids to modify in vivo release from multivesicular liposomes
CA2361201A1 (en) 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
WO2000053722A2 (en) 1999-03-10 2000-09-14 Phogen Limited Delivery of nucleic acids and proteins to cells
AU2001245793A1 (en) 2000-03-16 2001-09-24 Cold Spring Harbor Laboratory Methods and compositions for rna interference
US6680068B2 (en) 2000-07-06 2004-01-20 The General Hospital Corporation Drug delivery formulations and targeting
EP1873259B1 (en) 2000-12-01 2012-01-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. RNA interference mediated by 21 and 22nt RNAs
WO2002100435A1 (en) 2001-06-11 2002-12-19 Centre Hospitalier Universitaire De Montreal Compositions and methods for enhancing nucleic acid transfer into cells
AU2002324723B2 (en) 2001-08-16 2007-10-25 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved DNA lipofection and/or slow release pro-drug and drug therapies
EP2338478B1 (en) 2002-06-28 2014-07-23 Protiva Biotherapeutics Inc. Method for producing liposomes
WO2004028471A2 (en) 2002-09-28 2004-04-08 Massachusetts Institute Of Technology Influenza therapeutic
EP2314691A3 (en) 2002-11-14 2012-01-18 Dharmacon, Inc. Fuctional and hyperfunctional siRNA
EP1585756B1 (en) 2002-11-26 2010-04-21 University of Massachusetts Delivery of sirnas
US20040208921A1 (en) 2003-01-14 2004-10-21 Ho Rodney J. Y. Lipid-drug formulations and methods for targeted delivery of lipid-drug complexes to lymphoid tissues
US7598227B2 (en) * 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
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
AU2004269150C1 (en) 2003-08-28 2010-11-18 Novartis Ag Interfering RNA duplex having blunt-ends and 3'-modifications
CN101052717A (zh) 2004-05-11 2007-10-10 α基因株式会社 诱发rna干扰的多核苷酸以及使用该多核苷酸的基因表达抑制方法
EP1948674A4 (en) 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF
EP2002004B1 (en) 2006-03-23 2015-10-14 Roche Innovation Center Copenhagen A/S Small internally segmented interfering rna
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
KR101133799B1 (ko) 2006-08-18 2012-04-24 에프. 호프만-라 로슈 아게 폴리뉴클레오타이드의 생체내 전달을 위한 다가접합체
KR101629017B1 (ko) 2007-05-22 2016-06-10 아크투루스 쎄라퓨틱스, 인크. 히드록시메틸 치환된 ｒｎａ 올리고뉴클레오티드 및 ｒｎａ 복합체
US7615374B2 (en) 2007-09-25 2009-11-10 Wisconsin Alumni Research Foundation Generation of clonal mesenchymal progenitors and mesenchymal stem cell lines under serum-free conditions
AU2008342535B2 (en) 2007-12-27 2015-02-05 Arbutus Biopharma Corporation Silencing of polo-like kinase expression using interfering RNA
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
US9023820B2 (en) * 2009-01-26 2015-05-05 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein C-III expression
WO2011076807A2 (en) 2009-12-23 2011-06-30 Novartis Ag Lipids, lipid compositions, and methods of using them
SI2539451T1 (sl) 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
EP3693464A3 (en) 2011-06-21 2020-12-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
HK1199736A1 (en) 2011-08-26 2015-07-17 箭头研究公司 Poly(vinyl ester) polymers for in vivo nucleic acid delivery
US8933047B2 (en) 2012-04-18 2015-01-13 Arrowhead Madison Inc. Poly(acrylate) polymers for in vivo nucleic acid delivery
WO2013165816A2 (en) * 2012-05-02 2013-11-07 Merck Sharp & Dohme Corp. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
JP6694382B2 (ja) * 2013-06-21 2020-05-13 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 標的核酸を調節するための組成物および方法
US9988627B2 (en) 2013-10-04 2018-06-05 Novartis Ag Formats for organic compounds for use in RNA interference
EP3169784B1 (en) * 2014-07-16 2020-06-10 Arrowhead Pharmaceuticals, Inc. Rnai compositions to treat apoc3-related diseases
EP3221451A1 (en) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
WO2016149020A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
JOP20170057B1 (ar) 2016-03-07 2022-03-14 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة كمركبات علاجية
IL300869A (en) * 2016-09-02 2023-04-01 Arrowhead Pharmaceuticals Inc Ligands containing a targeting factor, preparations containing them and their uses
CN120884604A (zh) * 2017-06-02 2025-11-04 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
EP3681516A4 (en) * 2017-09-11 2021-09-29 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)

Also Published As

Publication number Publication date
CN111107853B (zh) 2024-07-30
US12365899B2 (en) 2025-07-22
KR20240161202A (ko) 2024-11-12
SG11201912178VA (en) 2020-01-30
US10597657B2 (en) 2020-03-24
KR102723833B1 (ko) 2024-11-01
CN118879696A (zh) 2024-11-01
AU2018329190B2 (en) 2025-08-14
EP3681516A4 (en) 2021-09-29
PH12020500332A1 (en) 2020-09-28
PE20201283A1 (es) 2020-11-24
IL273172A (en) 2020-04-30
KR20200044013A (ko) 2020-04-28
TN2020000039A1 (en) 2021-10-04
US20190078088A1 (en) 2019-03-14
IL324223A (en) 2025-12-01
CR20200108A (es) 2020-06-28
JOP20200054A1 (ar) 2020-03-10
US20230323350A1 (en) 2023-10-12
US20200299690A1 (en) 2020-09-24
UY37871A (es) 2019-03-29
ZA202100806B (en) 2023-03-29
ECSP20017905A (es) 2020-06-30
CN111107853A (zh) 2020-05-05
US20220064646A1 (en) 2022-03-03
CA3074303A1 (en) 2019-03-14
CL2024003470A1 (es) 2025-04-11
JP7438103B2 (ja) 2024-02-26
AU2018329190A1 (en) 2020-04-23
CO2020002586A2 (es) 2020-04-01
TW201920227A (zh) 2019-06-01
IL273172B1 (en) 2025-12-01
EP3681516A1 (en) 2020-07-22
WO2019051402A1 (en) 2019-03-14
JP2023073358A (ja) 2023-05-25
TW202415389A (zh) 2024-04-16
CL2020000593A1 (es) 2020-09-25
BR112020002413A2 (pt) 2020-07-28
JP2020533334A (ja) 2020-11-19
TWI811238B (zh) 2023-08-11
US11214801B2 (en) 2022-01-04
MY202025A (en) 2024-03-29

Similar Documents

Publication Publication Date Title
ECSP20017905A (es) AGENTES DE iARN Y COMPOSICIONES PARA INHIBIR LA EXPRESIÓN DE LA APOLIPOPROTEÍNA C-III (APOC3)
CO2020001740A2 (es) Agentes de iarn y composiciones para inhibir la expresión de la angiopoyetina tipo 3 (angptl3) y métodos de uso
MX2022013010A (es) Composiciones y metodos para inhibir la expresion genica de lpa.
ECSP21027262A (es) Agentes de ARNi para inhibir la expresión de 17beta-HSD tipo 13 (HSD17B13), composiciones de dichos agentes, y métodos de uso
MX2017011422A (es) Composiciones y metodos para inhibir la expresion del gen del factor xii.
MX2019008252A (es) Agentes de interferencia de acido ribonucleico (iarn) de alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y metodos de uso.
MX2020002184A (es) Composiciones y metodos para modular la expresion de la apolipoproteina (a).
MX375836B (es) Composiciones y métodos para inhibir la expresión del gen de alfa-1 antitripsina.
EA201792103A1 (ru) Композиции и способы для ингибирования экспрессии генов hif2альфа
MX2022001460A (es) Metodos para el tratamiento de enfermedades y trastornos relacionados con apoc3.
AR113014A1 (es) AGENTES DE iARN Y COMPOSICIONES PARA INHIBIR LA EXPRESIÓN DE LA APOLIPOPROTEÍNA C-III (APOC3)
EA202090687A1 (ru) РНКи-АГЕНТЫ И КОМПОЗИЦИИ ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (APOC3)
AR113254A1 (es) AGENTES DE iARN Y COMPOSICIONES PARA INHIBIR LA EXPRESIÓN DE LA ANGIOPOYETINA TIPO 3 (ANGPTL3) Y MÉTODOS DE USO
AR106227A1 (es) COMPOSICIONES Y MÉTODOS PARA INHIBIR LA EXPRESIÓN GÉNICA DEL GEN DE LA APOLIPOPROTEÍNA APO(a) (LPA)
AR133962A1 (es) Composiciones y métodos para tratar enfermedad de hígado graso no alcohólico
EA202090717A1 (ru) СРЕДСТВА И КОМПОЗИЦИИ РНКи ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ АНГИОПОЭТИН-ПОДОБНОГО БЕЛКА 3 (ANGPTL3) И СПОСОБЫ ПРИМЕНЕНИЯ
TW202600825A (zh) 抑制lpa之基因表現之組合物及方法
CR20200117A (es) Agentes de iarn y composiciones para inhibir la expresión de la angiopoyetina tipo 3 (angptl3) y métodos de uso
AR110742A1 (es) AGENTES DE iARN a-1 ANTITRIPSINA (AAT), COMPOSICIONES QUE INCLUYEN AGENTES DE iARN AAT Y MÉTODOS DE USO