CA3120580A1 - Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use - Google Patents

Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use Download PDF

Info

Publication number
CA3120580A1
CA3120580A1 CA3120580A CA3120580A CA3120580A1 CA 3120580 A1 CA3120580 A1 CA 3120580A1 CA 3120580 A CA3120580 A CA 3120580A CA 3120580 A CA3120580 A CA 3120580A CA 3120580 A1 CA3120580 A1 CA 3120580A1
Authority
CA
Canada
Prior art keywords
invab
rnai agent
sense strand
nucleotides
hif
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.)
Pending
Application number
CA3120580A
Other languages
English (en)
French (fr)
Inventor
Zhen Li
Dongxu Shu
Anthony Nicholas
Rui ZHU
Jeffrey Carlson
So Wong
Xiaokai Li
Erich ALTENHOFER
Matthew FOWLER-WATTERS
Bo Chen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arrowhead Pharmaceuticals Inc
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 CA3120580A1 publication Critical patent/CA3120580A1/en
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/545—Heterocyclic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/02—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D207/30—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members
    • C07D207/34—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D207/36—Oxygen or sulfur atoms
    • C07D207/40—2,5-Pyrrolidine-diones
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • 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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/12—Animals modified by administration of exogenous cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0331—Animal model for proliferative diseases
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CA3120580A 2019-01-09 2020-01-08 Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use Pending CA3120580A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962790360P 2019-01-09 2019-01-09
US62/790,360 2019-01-09
US201962827564P 2019-04-01 2019-04-01
US62/827,564 2019-04-01
US201962839381P 2019-04-26 2019-04-26
US62/839,381 2019-04-26
PCT/US2020/012775 WO2020146521A2 (en) 2019-01-09 2020-01-08 Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use

Publications (1)

Publication Number Publication Date
CA3120580A1 true CA3120580A1 (en) 2020-07-16

Family

ID=71520182

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3120580A Pending CA3120580A1 (en) 2019-01-09 2020-01-08 Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use

Country Status (14)

Country Link
US (1) US12221610B2 (enExample)
EP (1) EP3908288A4 (enExample)
JP (1) JP7808963B2 (enExample)
KR (1) KR20210113273A (enExample)
CN (1) CN113613661B (enExample)
AU (1) AU2020207050A1 (enExample)
BR (1) BR112021013362A2 (enExample)
CA (1) CA3120580A1 (enExample)
IL (1) IL284696A (enExample)
MX (1) MX2021008261A (enExample)
SG (1) SG11202105577TA (enExample)
TW (1) TW202043470A (enExample)
UY (1) UY38539A (enExample)
WO (1) WO2020146521A2 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114053426B (zh) * 2020-07-30 2024-06-18 成都科岭源医药技术有限公司 一种双药链接组装单元及双药靶向接头-药物偶联物
WO2022056273A1 (en) * 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Lipid conjugates for the delivery of therapeutic agents
WO2022056269A1 (en) * 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Skeletal muscle delivery platforms and methods of use
WO2022086882A1 (en) 2020-10-21 2022-04-28 Nikang Therapeutics, Inc. Diagnostic and therapeutic methods for treatment of cancer with a hif-2(alpha) inhibitor
CN115105607B (zh) * 2021-03-22 2024-12-20 成都科岭源医药技术有限公司 一种用于adc的双药-接头的制备方法及其用途
CN113304274B (zh) * 2021-04-23 2025-03-04 天津全和诚生物技术有限公司 缀合物及其制备方法和用途
WO2023038027A1 (ja) * 2021-09-07 2023-03-16 国立大学法人京都大学 セノリシス薬のスクリーニング方法及びセノリシス薬

Family Cites Families (55)

* 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
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
US6017926A (en) 1997-12-17 2000-01-25 Merck & Co., Inc. Integrin receptor antagonists
SK9192000A3 (en) 1997-12-17 2001-03-12 Merck & Co Inc Integrin receptor antagonists
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
WO2000053722A2 (en) 1999-03-10 2000-09-14 Phogen Limited Delivery of nucleic acids and proteins to cells
US6309401B1 (en) 1999-04-30 2001-10-30 Vladimir Redko Apparatus and method for percutaneous implant of a paddle style lead
JP2003526367A (ja) 2000-03-16 2003-09-09 ジェネティカ インコーポレイテッド Ｒｎａ干渉の方法とｒｎａ干渉組成物
US6680068B2 (en) 2000-07-06 2004-01-20 The General Hospital Corporation Drug delivery formulations and targeting
US20080188430A1 (en) 2001-05-18 2008-08-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA)
WO2002100435A1 (en) 2001-06-11 2002-12-19 Centre Hospitalier Universitaire De Montreal Compositions and methods for enhancing nucleic acid transfer into cells
WO2003015757A1 (en) 2001-08-16 2003-02-27 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved dna lipofection and/or slow release prodrug and drug therapies
DE10230997A1 (de) 2001-10-26 2003-07-17 Ribopharma Ag Medikament zur Erhöhung der Wirksamkeit eines Rezeptor-vermittelt Apoptose in Tumorzellen auslösenden Arzneimittels
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
EP1658304A4 (en) 2002-09-28 2009-01-14 Massachusetts Inst Technology THERAPY ANTIGRIPPALE
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2004048545A2 (en) 2002-11-26 2004-06-10 University Of Massachusetts DELIVERY OF siRNAs______________________________________________
AU2004257373B2 (en) 2003-07-16 2011-03-24 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
CN1845993B (zh) 2003-08-28 2010-06-23 诺瓦提斯公司 具有平端和3＇修饰的干扰rna双链体
CA2559955C (en) 2004-03-15 2016-02-16 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna
CA2566286A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
EP2990410A1 (en) 2004-08-10 2016-03-02 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
EP1948674A4 (en) 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF
ES2556128T3 (es) 2006-03-23 2016-01-13 Roche Innovation Center Copenhagen A/S ARN interfiriente pequeño internamente segmentado
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
NZ580712A (en) 2007-05-22 2011-12-22 Marina Biotech Inc Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers
CN101868251B (zh) 2007-08-06 2012-10-10 千寿制药株式会社 含有HIF-1α和HIF-2α表达抑制物质的药物
US9006191B2 (en) 2007-12-27 2015-04-14 Protiva Biotherapeutics, Inc. 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
US8067178B2 (en) 2008-03-14 2011-11-29 Genomic Health, Inc. Gene expression markers for prediction of patient response to chemotherapy
EP3673922A1 (en) * 2008-03-14 2020-07-01 VisEn Medical, Inc. Integrin targeting agents and methods of using same
WO2009117531A1 (en) 2008-03-18 2009-09-24 Seattle Genetics, Inc. Auristatin drug linker conjugates
JP2011516065A (ja) * 2008-04-04 2011-05-26 カランド ファーマシューティカルズ， インコーポレイテッド Ｅｐａｓ１阻害剤の組成物および使用
KR20110110776A (ko) 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
WO2011076807A2 (en) 2009-12-23 2011-06-30 Novartis Ag Lipids, lipid compositions, and methods of using them
PH12012501596A1 (en) 2010-02-24 2017-02-03 Arrowhead Res Corporation Compositions for targeted delivery of sirna
KR101223660B1 (ko) 2010-05-20 2013-01-17 광주과학기술원 ＨＩＦ－２α 억제제를 유효성분으로 포함하는 관절염 예방 또는 치료용 약제학적 조성물
RU2013117288A (ru) 2010-12-17 2015-01-27 Эрроухэд Рисерч Корпорейшн СОДЕРЖАЩАЯ ГАЛАКТОЗНЫЙ КЛАСТЕР НАЦЕЛИВАЮЩАЯ ГРУППА ДЛЯ миРНК, МОДУЛИРУЮЩАЯ ФОРМАКОКИНЕТИЧЕСКИЕ СВОЙСТВА
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
RU2582235C2 (ru) 2010-12-29 2016-04-20 Ф.Хоффманн-Ля Рош Аг Низкомолекулярные конъюгаты для внутриклеточной доставки нуклеиновых кислот
WO2012174224A2 (en) 2011-06-17 2012-12-20 Calando Pharmaceuticals, Inc. Methods for administering nucleic acid-based therapeutics
CA2842039A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
MX2014001965A (es) 2012-04-18 2014-03-31 Arrowhead Res Corp Polimeros de poli(acrilato) para suministro de acido nucleico in vivo.
HK1214301A1 (zh) 2013-02-28 2016-07-22 箭头研究公司 治療epas1相關疾病的有機組合物
US9504747B2 (en) 2013-03-08 2016-11-29 Novartis Ag Lipids and lipid compositions for the delivery of active agents
WO2015021092A1 (en) 2013-08-07 2015-02-12 Arrowhead Research Corporation Polyconjugates for delivery of rnai triggers to tumor cells in vivo
US20160272970A1 (en) 2015-03-17 2016-09-22 Arrowhead Madison Inc. RNA Interference Agents
TWI761305B (zh) * 2015-05-29 2022-04-21 美商愛羅海德製藥公司 抑制Ｈｉｆ２α基因表現之組合物及方法
CN120247964A (zh) 2018-02-17 2025-07-04 箭头药业股份有限公司 三炔连接剂和使用方法
IL309955A (en) 2018-04-27 2024-03-01 Arrowhead Pharmaceuticals Inc Integrin-targeting ligands and their uses

Also Published As

Publication number Publication date
JP7808963B2 (ja) 2026-01-30
MX2021008261A (es) 2021-08-16
WO2020146521A3 (en) 2020-08-20
WO2020146521A2 (en) 2020-07-16
SG11202105577TA (en) 2021-06-29
IL284696A (en) 2021-08-31
JP2022518384A (ja) 2022-03-15
BR112021013362A2 (pt) 2021-12-14
CN113613661A (zh) 2021-11-05
EP3908288A4 (en) 2023-12-20
EP3908288A2 (en) 2021-11-17
US20220204976A1 (en) 2022-06-30
KR20210113273A (ko) 2021-09-15
TW202043470A (zh) 2020-12-01
CN113613661B (zh) 2025-11-14
AU2020207050A1 (en) 2021-06-17
UY38539A (es) 2020-07-31
US12221610B2 (en) 2025-02-11

Similar Documents

Publication Publication Date Title
US20250360089A1 (en) Nanoparticle formulations for delivery of nucleic acid complexes
JP7808963B2 (ja) ＨＩＦ－２アルファ（ＥＰＡＳ１）の発現を阻害するためのＲＮＡｉ剤、その組成物及び使用方法
JP2021104037A (ja) アンチセンス核酸
KR20230064620A (ko) DUX4의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
AU2019260738B2 (en) Integrin targeting ligands and uses thereof
TW201540724A (zh) 反義核酸
CA3011946A1 (en) Targeting ligands for therapeutic compounds
AU2022349576A1 (en) Multivalent ligand clusters with diamine scaffold for targeted delivery of therapeutic agents
CN119730859A (zh) 用于抑制超氧化物歧化酶1(SOD1)表达的RNAi剂、其组合物及使用方法
WO2019210308A1 (en) Integrin targeting ligands and uses thereof
WO2026030215A1 (en) Lipid conjugates for the delivery of therapeutic agents
CA3003267C (en) Nanoparticle formulations for delivery of nucleic acid complexes
CN116801885A (zh) 用于抑制DUX4表达的RNAi试剂、其组合物和使用方法
HK40047192A (en) Integrin targeting ligands and uses thereof
HK40047192B (en) Integrin targeting ligands and uses thereof