UY37871A - Agentes de iarn y composiciones para inhibir la expresión de la apolipoproteína c-iii (apoc3) - Google Patents

Agentes de iarn y composiciones para inhibir la expresión de la apolipoproteína c-iii (apoc3)

Info

Publication number
UY37871A
UY37871A UY0001037871A UY37871A UY37871A UY 37871 A UY37871 A UY 37871A UY 0001037871 A UY0001037871 A UY 0001037871A UY 37871 A UY37871 A UY 37871A UY 37871 A UY37871 A UY 37871A
Authority
UY
Uruguay
Prior art keywords
iii
apoc3
agents
apolipoprotein
expression
Prior art date
Application number
UY0001037871A
Other languages
English (en)
Inventor
Steven Kanner
Tao Pei
Zhen Li
Rui Zhu
So Wong
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 UY37871A publication Critical patent/UY37871A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/04Anorexiants; Antiobesity agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H3/00Compounds containing only hydrogen atoms and saccharide radicals having only carbon, hydrogen, and oxygen atoms
    • C07H3/02Monosaccharides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Obesity (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

La presente descripción se refiere a agentes de iARN, por ejemplo, agentes de iARN de cadena doble, capaces de inhibir la expresión del gen de la Apolipoproteína C-III (también denominada APOC3, apoC-III, APOC-III, y APO C-III), y composiciones que incluyen agentes de iARN de APOC3. Los agentes de iARN de APOC3 descritos en la presente pueden estar conjugados con ligandos diana, que incluyen ligandos que incluyen N-acetil-galactosamina, para facilitar la administración a células, que incluyen a hepatocitos
UY0001037871A 2017-09-11 2018-09-11 Agentes de iarn y composiciones para inhibir la expresión de la apolipoproteína c-iii (apoc3) UY37871A (es)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
UY37871A true UY37871A (es) 2019-03-29

Family

ID=65630632

Family Applications (1)

Application Number Title Priority Date Filing Date
UY0001037871A UY37871A (es) 2017-09-11 2018-09-11 Agentes de iarn y composiciones para inhibir la expresión de la apolipoproteína c-iii (apoc3)

Country Status (24)

Country Link
US (4) US10597657B2 (es)
EP (1) EP3681516A4 (es)
JP (2) JP7438103B2 (es)
KR (1) KR20200044013A (es)
CN (1) CN111107853B (es)
AU (1) AU2018329190A1 (es)
BR (1) BR112020002413A2 (es)
CA (1) CA3074303A1 (es)
CL (1) CL2020000593A1 (es)
CO (1) CO2020002586A2 (es)
CR (1) CR20200108A (es)
EC (1) ECSP20017905A (es)
IL (1) IL273172A (es)
JO (1) JOP20200054A1 (es)
MX (1) MX2020002648A (es)
MY (1) MY202025A (es)
PE (1) PE20201283A1 (es)
PH (1) PH12020500332A1 (es)
SG (1) SG11201912178VA (es)
TN (1) TN2020000039A1 (es)
TW (2) TWI811238B (es)
UY (1) UY37871A (es)
WO (1) WO2019051402A1 (es)
ZA (1) ZA202100806B (es)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015350120B2 (en) 2014-11-17 2021-05-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein C3 (APOC3) 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
JOP20200054A1 (ar) * 2017-09-11 2020-03-10 Arrowhead Pharmaceuticals Inc عوامل RNAi وتركيبات لتثبيط تعبير صَميمُ البروتينِ الشَّحْمِيّ C-III (APOC3)
KR20220044551A (ko) * 2019-08-05 2022-04-08 애로우헤드 파마슈티컬스 인코포레이티드 Apoc3-관련 질환 및 장애의 치료 방법
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
WO2022272108A2 (en) 2021-06-24 2022-12-29 Sirnaomics, Inc. Products and compositions
KR20240034787A (ko) * 2021-07-16 2024-03-14 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 함유하는 조성물 및 접합체, 제조 방법 및 용도
JP2024537251A (ja) * 2021-10-08 2024-10-10 イー-セラピューティクス・ピーエルシー 脱塩基ヌクレオシドを含有する核酸
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
WO2023138659A1 (zh) * 2022-01-20 2023-07-27 上海拓界生物医药科技有限公司 一种dsRNA、其应用及制备方法
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
CN117756866A (zh) 2022-01-30 2024-03-26 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
WO2024002093A1 (zh) * 2022-06-27 2024-01-04 大睿生物医药科技(上海)有限公司 抑制载脂蛋白C3表达的siRNA
WO2024015796A1 (en) * 2022-07-11 2024-01-18 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
WO2024023256A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
WO2024023254A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
WO2024023262A2 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
CN116814621A (zh) * 2022-08-05 2023-09-29 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
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 (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
AU776150B2 (en) 1999-01-28 2004-08-26 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
ATE408699T1 (de) 1999-03-10 2008-10-15 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
EP1272630A2 (en) 2000-03-16 2003-01-08 Genetica, Inc. Methods and compositions for rna interference
US6680068B2 (en) 2000-07-06 2004-01-20 The General Hospital Corporation Drug delivery formulations and targeting
WO2002044321A2 (en) 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna interference mediating small rna molecules
WO2002100435A1 (en) 2001-06-11 2002-12-19 Centre Hospitalier Universitaire De Montreal Compositions and methods for enhancing nucleic acid transfer into cells
CA2456977C (en) 2001-08-16 2011-06-07 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved dna lipofection and/or slow release prodrug and drug therapies
DK1519714T3 (da) 2002-06-28 2011-01-31 Protiva Biotherapeutics Inc Fremgangsmåde og apparat til fremstilling af liposomer
WO2004028471A2 (en) 2002-09-28 2004-04-08 Massachusetts Institute Of Technology Influenza therapeutic
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
CA2506714A1 (en) 2002-11-26 2004-06-10 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
EP2567693B1 (en) 2003-07-16 2015-10-21 Protiva Biotherapeutics Inc. Lipid encapsulated interfering RNA
WO2005021749A1 (en) 2003-08-28 2005-03-10 Novartis Ag Interfering rna duplex having blunt-ends and 3’-modifications
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
CA2644347C (en) 2006-03-23 2017-05-30 Santaris Pharma A/S Small internally segmented interfering rna
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
MX2009001207A (es) 2006-08-18 2009-02-11 Hoffmann La Roche Policonjugados para el suministro in vivo de polinucleotidos.
US8314227B2 (en) 2007-05-22 2012-11-20 Marina Biotech, Inc. Hydroxymethyl substituted RNA oligonucleotides and RNA complexes
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
ES2535419T3 (es) 2007-12-27 2015-05-11 Protiva Biotherapeutics Inc. Silenciamiento de expresión de quinasa tipo polo usando ARN interferente
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
BR112012015755B1 (pt) 2009-12-23 2021-06-22 Novartis Ag Lipídeo furtivo, e composição
PT2539451E (pt) 2010-02-24 2016-03-28 Arrowhead Res Corp Composições para entrega de arnsi dirigida ao alvo
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
KR20230084331A (ko) 2011-06-21 2023-06-12 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
US8932572B2 (en) 2011-08-26 2015-01-13 Arrowhead Madison Inc. Poly(vinyl ester) polymers for in vivo nucleic acid delivery
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
EP3011028B1 (en) * 2013-06-21 2019-06-12 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
US9988627B2 (en) 2013-10-04 2018-06-05 Novartis Ag Formats for organic compounds for use in RNA interference
WO2016011123A1 (en) 2014-07-16 2016-01-21 Arrowhead Research Corporation Organic compositions to treat apoc3-related diseases
AU2015350120B2 (en) * 2014-11-17 2021-05-27 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
EP3426261A4 (en) 2016-03-07 2020-03-25 Arrowhead Pharmaceuticals, Inc. TARGETED LIGANDS FOR THERAPEUTIC CONNECTIONS
CN116832169A (zh) * 2016-09-02 2023-10-03 箭头药业股份有限公司 靶向配体
US11603532B2 (en) * 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JOP20200054A1 (ar) * 2017-09-11 2020-03-10 Arrowhead Pharmaceuticals Inc عوامل RNAi وتركيبات لتثبيط تعبير صَميمُ البروتينِ الشَّحْمِيّ C-III (APOC3)

Also Published As

Publication number Publication date
KR20200044013A (ko) 2020-04-28
TWI811238B (zh) 2023-08-11
ZA202100806B (en) 2023-03-29
JOP20200054A1 (ar) 2020-03-10
BR112020002413A2 (pt) 2020-07-28
US20220064646A1 (en) 2022-03-03
CO2020002586A2 (es) 2020-04-01
CN111107853B (zh) 2024-07-30
EP3681516A1 (en) 2020-07-22
MY202025A (en) 2024-03-29
US20200299690A1 (en) 2020-09-24
CL2020000593A1 (es) 2020-09-25
CN111107853A (zh) 2020-05-05
MX2020002648A (es) 2020-07-22
US11214801B2 (en) 2022-01-04
CR20200108A (es) 2020-06-28
AU2018329190A1 (en) 2020-04-23
JP7438103B2 (ja) 2024-02-26
TW202415389A (zh) 2024-04-16
SG11201912178VA (en) 2020-01-30
US20190078088A1 (en) 2019-03-14
PH12020500332A1 (en) 2020-09-28
TW201920227A (zh) 2019-06-01
TN2020000039A1 (en) 2021-10-04
JP2020533334A (ja) 2020-11-19
PE20201283A1 (es) 2020-11-24
CA3074303A1 (en) 2019-03-14
IL273172A (en) 2020-04-30
ECSP20017905A (es) 2020-06-30
US10597657B2 (en) 2020-03-24
US20230323350A1 (en) 2023-10-12
JP2023073358A (ja) 2023-05-25
WO2019051402A1 (en) 2019-03-14
EP3681516A4 (en) 2021-09-29

Similar Documents

Publication Publication Date Title
UY37871A (es) Agentes de iarn y composiciones para inhibir la expresión de la apolipoproteína c-iii (apoc3)
UY37881A (es) Agentes de iarn y composiciones para inhibir la expresión de la angiopoyetina tipo 3 (angptl3) y métodos de uso
CO2018003678A2 (es) Composiciones y métodos para inhibir la expresión génica de lpa
CL2021002998A1 (es) Composiciones de arni contra tmprss6 y métodos para su uso (divisional de solicitud n° 03440-2015)
CL2019001711A1 (es) Composiciones y métodos para inhibir la acción de la arginasa.
CO2017010583A2 (es) Compuestos de pirazol y método para elaborar y usar los compuestos
CL2019000819A1 (es) Compuestos y composiciones para inhibir la actividad arginasa. (divisional solicitud 201801134)
UY37145A (es) Ligandos de direccionamiento
CL2016003041A1 (es) Ácidos ribonucleicos bicatenarios aislados, composiciones farmacéuticas que los comprenden y método para inhibir la expresión del gen alas1 basado en la administración de dicha composición. divisional de solicitud 2725-2014.
CR20140232A (es) Agentes de iarn, composiciones y métodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr)
UY36671A (es) COMPOSICIONES DE ARNi CONTRA Factor XII (FACTOR DE Hageman) (F12),CALICREÍNA B, PLASMÁTICA (FACTOR DE FLETCHER) 1 (KLKB1), Y QUININÓGENO 1 (KNG1) Y MÉTODOS DE USO DE LOS MISMO
DOP2015000288A (es) Composiciones de arni de serpina1 y sus métodos de uso.
DOP2019000132A (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO DE LAS MISMAS.
BR112015027319A8 (pt) Métodos e composições para modular a expressão de apolipoproteína (a)
EA201692318A1 (ru) Композиции и способы для ингибирования экспрессии гена альфа-1-антитрипсина
DOP2014000098A (es) 2-tiopirimidinonas
CO2018002198A2 (es) Composiciones farmacéuticas que comprenden un compuesto oligomérico para modular la expresión de apolipoproteína (a)
UY37800A (es) AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO
AR099381A1 (es) Composiciones de pulpa en grado de disolución, método para tratar un material celulósico y producto celulósico preparado mediante el método
CU20170134A7 (es) 2-tiopirimidinonas
CL2019003571A1 (es) Lipido que comprende ácido docosapentanoico. (divisional solicitud 3351-2016)
AR113014A1 (es) AGENTES DE iARN Y COMPOSICIONES PARA INHIBIR LA EXPRESIÓN DE LA APOLIPOPROTEÍNA C-III (APOC3)
BR112017003597A2 (pt) disposição para proteção contra corrosão
AR101881A1 (es) Composición blanqueadora
AR106227A1 (es) COMPOSICIONES Y MÉTODOS PARA INHIBIR LA EXPRESIÓN GÉNICA DEL GEN DE LA APOLIPOPROTEÍNA APO(a) (LPA)