AP3284A - Compositions for targeted delivery of SIRNA - Google Patents

Compositions for targeted delivery of SIRNA

Info

Publication number
AP3284A
AP3284A AP2012006453A AP2012006453A AP3284A AP 3284 A AP3284 A AP 3284A AP 2012006453 A AP2012006453 A AP 2012006453A AP 2012006453 A AP2012006453 A AP 2012006453A AP 3284 A AP3284 A AP 3284A
Authority
AP
ARIPO
Prior art keywords
sirna
compositions
targeted delivery
targeted
delivery
Prior art date
Application number
AP2012006453A
Other languages
English (en)
Other versions
AP2012006453A0 (en
Inventor
Peter Mohr
Eric A Kitas
David B Rozema
Original Assignee
Arrowhead Res Corp
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 Res Corp filed Critical Arrowhead Res Corp
Publication of AP2012006453A0 publication Critical patent/AP2012006453A0/xx
Application granted granted Critical
Publication of AP3284A publication Critical patent/AP3284A/xx

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
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/32—Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers, poly(meth)acrylates, or polyvinyl pyrrolidone
    • 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • A61K9/08—Solutions
    • 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
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • 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
    • A61P3/06—Antihyperlipidemics
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • 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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Inorganic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Communicable Diseases (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AP2012006453A 2010-02-24 2011-02-18 Compositions for targeted delivery of SIRNA AP3284A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US30749010P 2010-02-24 2010-02-24
PCT/EP2011/052393 WO2011104169A1 (en) 2010-02-24 2011-02-18 Compositions for targeted delivery of sirna

Publications (2)

Publication Number Publication Date
AP2012006453A0 AP2012006453A0 (en) 2012-08-31
AP3284A true AP3284A (en) 2015-05-31

Family

ID=43901424

Family Applications (1)

Application Number Title Priority Date Filing Date
AP2012006453A AP3284A (en) 2010-02-24 2011-02-18 Compositions for targeted delivery of SIRNA

Country Status (26)

Country Link
US (4) US8313772B2 (enExample)
EP (2) EP3023495B1 (enExample)
JP (2) JP6137834B2 (enExample)
KR (1) KR101956623B1 (enExample)
CN (1) CN102869774B (enExample)
AP (1) AP3284A (enExample)
AU (1) AU2011219941C1 (enExample)
BR (1) BR112012021256A2 (enExample)
CA (1) CA2788600C (enExample)
DK (1) DK2539451T3 (enExample)
EA (1) EA024534B1 (enExample)
ES (1) ES2562817T3 (enExample)
HR (1) HRP20160093T1 (enExample)
HU (1) HUE026811T2 (enExample)
IL (1) IL221396A (enExample)
MX (1) MX2012009178A (enExample)
NZ (1) NZ601737A (enExample)
PH (1) PH12012501596A1 (enExample)
PL (1) PL2539451T3 (enExample)
PT (1) PT2539451E (enExample)
RS (1) RS54578B1 (enExample)
SG (1) SG183374A1 (enExample)
SI (1) SI2539451T1 (enExample)
SM (1) SMT201600079B (enExample)
WO (1) WO2011104169A1 (enExample)
ZA (1) ZA201205685B (enExample)

Families Citing this family (215)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080281041A1 (en) * 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
JP2012520085A (ja) * 2009-03-13 2012-09-06 エーゲン、インコーポレイテッド 生物活性ｒｎａの送達のための組成物及び方法
SI2539451T1 (sl) * 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna
US10913767B2 (en) * 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2625189B1 (en) 2010-10-01 2018-06-27 ModernaTX, Inc. Engineered nucleic acids and methods of use thereof
AU2011343664B2 (en) 2010-12-17 2015-10-08 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for siRNA
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
CA3131967A1 (en) 2010-12-29 2012-07-05 F. Hoffman-La Roche Ag Small molecule conjugates for intracellular delivery of nucleic acids
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US9481661B2 (en) 2011-06-17 2016-11-01 Arrowhead Madison, Inc. Disubstituted maleic anhydrides with altered kinetics of ring closure
WO2013032829A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
EP2807216A4 (en) * 2011-08-26 2015-10-28 Arrowhead Res Corp POLY (VINYLESTER) POLYMERS FOR IN VIVO NUCLEIC ACID RELIEF
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
AU2012358181A1 (en) * 2011-12-23 2014-07-10 Egen, Inc. Compositions and methods for the delivery of biologically active RNAs
WO2013102878A2 (en) 2012-01-05 2013-07-11 Department Of Biotechnology (Dbt) Fat1 gene in cancer and inflammation
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP2838544A4 (en) * 2012-04-18 2015-10-14 Arrowhead Res Corp POLY (ACRYLATE) POLYMERS FOR IN VIVO NUCLEIC ACID ADMINISTRATION
CA2842041A1 (en) 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
TW201808342A (zh) * 2012-05-02 2018-03-16 喜納製藥公司 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法
AR090905A1 (es) * 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
US10323279B2 (en) * 2012-08-14 2019-06-18 10X Genomics, Inc. Methods and systems for processing polynucleotides
AU2013302756C1 (en) 2012-08-14 2018-05-17 10X Genomics, Inc. Microcapsule compositions and methods
US9801953B2 (en) 2012-10-15 2017-10-31 Emory University Nanoparticles carrying nucleic acid cassettes for expressing RNA
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
CA2900238A1 (en) * 2013-02-22 2014-08-28 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) molecules containing a 2' internucleoside linkage
CN105247051A (zh) 2013-02-28 2016-01-13 箭头研究公司 治疗epas1相关疾病的有机组合物
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
US9506030B2 (en) 2013-05-01 2016-11-29 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
SG10201803157XA (en) 2013-05-01 2018-05-30 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-122
WO2015021092A1 (en) 2013-08-07 2015-02-12 Arrowhead Research Corporation Polyconjugates for delivery of rnai triggers to tumor cells in vivo
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
LT3052628T (lt) 2013-10-04 2020-09-10 Alnylam Pharmaceuticals, Inc. Kompozicijos ir būdai alas1 geno raiškai nuslopinti
US9994846B2 (en) 2013-10-25 2018-06-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
MX2016005855A (es) * 2013-11-14 2016-07-13 Roche Innovation Ct Copenhagen As Compuestos de conjugados antisentido de apolipoproteina b (apob).
WO2015168172A1 (en) 2014-04-28 2015-11-05 Isis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
BR112016022742B1 (pt) 2014-05-01 2022-06-14 Ionis Pharmaceuticals, Inc Composto químico, composição compreendendo o composto e uso dos mesmos
HUE052709T2 (hu) 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
US9382540B2 (en) 2014-05-01 2016-07-05 Isis Pharmaceuticals, Inc Compositions and methods for modulating angiopoietin-like 3 expression
EA036496B1 (ru) 2014-05-01 2020-11-17 Ионис Фармасьютикалз, Инк. Конъюгированные олигонуклеотиды для модулирования экспрессии фактора комплемента b
KR102589295B1 (ko) 2014-05-14 2023-10-13 타르그이뮨 테라퓨틱스 아게 개선된 폴리에틸렌이민 폴리에틸렌글리콜 벡터
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3151839A4 (en) * 2014-06-06 2018-02-28 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
KR20170033439A (ko) 2014-08-07 2017-03-24 레굴루스 테라퓨틱스 인크 대사 장애에 대한 마이크로rna의 표적화
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
TW202503057A (zh) * 2014-10-10 2025-01-16 美商艾爾妮蘭製藥公司 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法
AU2015330670B2 (en) 2014-10-10 2022-01-06 Novo Nordisk Health Care Ag Therapeutic inhibition of lactate dehydrogenase and agents therefor
CN106795200B (zh) 2014-10-10 2020-06-19 豪夫迈·罗氏有限公司 Galnac亚磷酰胺、其核酸缀合物及其用途
WO2016061487A1 (en) * 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3221445B1 (en) 2014-11-20 2021-07-14 The Regents of The University of California Compositions and methods related to hematologic recovery
WO2016138132A1 (en) * 2015-02-25 2016-09-01 The Trustees Of Columbia University In The City Of New York Dipeptidyl peptidase-iv(dpp4) inhibitors, methods and compositions for suppressing adipose tissue inflammation
CA2979703A1 (en) * 2015-03-17 2016-09-22 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of factor xii
AR104754A1 (es) 2015-05-29 2017-08-09 Arrowhead Pharmaceuticals Inc Agentes de unión de tetrapéptido escindibles biológicamente
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
MX2018000412A (es) 2015-07-10 2018-03-14 Ionis Pharmaceuticals Inc Moduladores de diaciglicerol aciltransferasa 2 (dgat2).
KR102579520B1 (ko) 2015-08-06 2023-09-15 에프. 호프만-라 로슈 아게 Galnac 산 유도체의 제조 방법
AU2016306275A1 (en) 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
CN108513588A (zh) 2015-09-24 2018-09-07 Ionis制药公司 Kras表达的调节剂
JOP20160211B1 (ar) 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
KR20250078597A (ko) 2015-11-06 2025-06-02 아이오니스 파마수티컬즈, 인코포레이티드 아포리포프로테인 (a) 발현 조정
PL4119569T3 (pl) 2015-11-06 2024-11-18 Ionis Pharmaceuticals, Inc. Sprzężone związki antysensowne do zastosowania w terapii
BR112018003910B1 (pt) 2015-11-16 2022-03-22 F. Hoffmann-La Roche Ag Derivado de fosforamidita galnac, processo para a preparação de um derivado de fosforamidita galnac de fórmula i e uso dos derivados de fosforamidita galnac de fórmula i
JP7027311B2 (ja) 2015-11-16 2022-03-01 オリックス ファーマシューティカルズ，インコーポレーテッド ＭｙＤ８８又はＴＬＲ３を標的とするＲＮＡ複合体を使用した加齢黄斑変性の治療
KR20180095843A (ko) 2015-12-07 2018-08-28 젠자임 코포레이션 Serpinc1-연관 장애의 치료를 위한 방법 및 조성물
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
JP6944942B2 (ja) 2016-02-02 2021-10-06 オリックス ファーマシューティカルズ，インコーポレーテッド ＩＬ４Ｒα、ＴＲＰＡ１、またはＦ２ＲＬ１を標的とするＲＮＡ複合体を用いたアトピー性皮膚炎および喘息の治療
CA3011946A1 (en) 2016-03-07 2017-09-14 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
US10407469B2 (en) 2016-04-01 2019-09-10 Northwestern University Programmable polypeptide and nucleic acid nanoparticles
CA3020585A1 (en) 2016-04-11 2017-10-19 Arbutus Biopharma Corporation Targeted nucleic acid conjugate compositions
WO2017178656A1 (en) 2016-04-14 2017-10-19 Roche Innovation Center Copenhagen A/S TRITYL-MONO-GalNAc COMPOUNDS AND THEIR USE
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
BR112018071997A2 (pt) 2016-04-26 2019-02-12 Viaqua Therapeutics Ltd nanopartícula de quitosana-rna, nanopartícula de quitosana-dsrna, composição nutracêutica, crustáceo criado em cativeiro, e método de tratamento ou prevenção de uma doença ou condição em um crustáceo criado em cativeiro associado a um vírus patogênico
MX2018015109A (es) 2016-06-06 2019-04-22 Arrowhead Pharmaceuticals Inc Nucleotidos modificados con 5'-ciclo-fosfonato.
EP3484524B1 (en) 2016-07-15 2022-11-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
WO2018017814A1 (en) 2016-07-20 2018-01-25 President And Fellows Of Harvard College Peptidoglycan glycosyltransferase inhibitors of sed proteins for treating bacterial infections
JOP20190015A1 (ar) 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
SG11201901841TA (en) * 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
WO2018053142A2 (en) 2016-09-14 2018-03-22 President And Fellows Of Harvard College Methods and compositions for modulating erythropoiesis
US11400161B2 (en) 2016-10-06 2022-08-02 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
WO2018112032A1 (en) 2016-12-13 2018-06-21 President And Fellows Of Harvard College Methods and compositions for targeting tumor-infiltrating tregs using inhibitors of ccr8 and tnfrsf8
WO2018112364A1 (en) 2016-12-16 2018-06-21 Evelo Biosciences, Inc. Combination therapies for treating melanoma
WO2018112360A1 (en) 2016-12-16 2018-06-21 Evelo Biosciences, Inc. Combination therapies for treating cancer
US20190314427A1 (en) 2016-12-16 2019-10-17 Evelo Biosciences, Inc. Methods of treating cancer using parabacteroides
WO2018112365A2 (en) 2016-12-16 2018-06-21 Evelo Biosciences, Inc. Methods of treating colorectal cancer and melanoma using parabacteroides goldsteinii
CN110268060B (zh) 2017-01-10 2024-07-26 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
EP3570857A1 (en) 2017-01-18 2019-11-27 Evelo Biosciences, Inc. Methods of treating cancer
EP3570856A2 (en) 2017-01-18 2019-11-27 Evelo Biosciences, Inc. Methods of treating cancer
AU2018213379A1 (en) 2017-01-30 2019-07-18 Arrowhead Pharmaceuticals Inc. Compositions and methods for inhibition of factor XII gene expression
SG11201906728TA (en) 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
MX2019012280A (es) 2017-04-11 2020-01-23 Arbutus Biopharma Corp Composiciones dirigidas.
WO2018215049A1 (en) 2017-05-23 2018-11-29 F. Hoffmann-La Roche Ag Process for galnac oligonucleotide conjugates
TN2019000308A1 (en) 2017-07-06 2021-05-07 Arrowhead Pharmaceuticals Inc RNAi AGENTS FOR INHIBITING EXPRESSION OF ALPHA-ENaC AND METHODS OF USE
CA3069451A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals, Inc. Methods for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
TW201920648A (zh) 2017-08-29 2019-06-01 美商艾弗洛生物科技股份有限公司 使用布勞特氏(blautia)菌株治療癌症
KR20240161202A (ko) 2017-09-11 2024-11-12 애로우헤드 파마슈티컬스 인코포레이티드 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물
TN2020000038A1 (en) 2017-09-14 2021-10-04 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
WO2019075419A1 (en) 2017-10-13 2019-04-18 Dicerna Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION
JP7473472B2 (ja) 2017-10-17 2024-04-23 アローヘッド ファーマシューティカルズ インコーポレイテッド アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物
CN110944675B9 (zh) 2017-12-01 2024-08-09 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制备方法和用途
CA3083970A1 (en) 2017-12-01 2019-06-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
DK3719128T3 (da) 2017-12-01 2025-03-03 Suzhou Ribo Life Science Co Ltd Dobbelt-strenget oligonukleotid, sammensætning og konjugat bestaende dobbelt-strenget oligonukleotid, fremgangsmåde herfor og anvendelse heraf
EP3719127A4 (en) 2017-12-01 2021-10-20 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE
EP3719126B1 (en) 2017-12-01 2025-01-01 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
US11021692B2 (en) 2017-12-19 2021-06-01 Janssen Sciences Ireland Unlimited Company Hepatitis B virus (HBV) vaccines and uses thereof
AU2018394875B2 (en) 2017-12-29 2023-08-03 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
AU2019206731A1 (en) 2018-01-15 2020-07-30 Ionis Pharmaceuticals, Inc. Modulators of DNM2 expression
IL276043B2 (en) * 2018-01-29 2024-06-01 Hoffmann La Roche Process for the preparation of galnac oligonucleotide conjugates
WO2019157531A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
WO2019169143A1 (en) 2018-02-28 2019-09-06 Evelo Biosciences, Inc. Compositions and methods for treating cancer using turicibacter sanguinis
WO2019169160A1 (en) 2018-02-28 2019-09-06 Evelo Biosciences, Inc. Compositions and methods for treating cancer using ruminococcus gnavus
WO2019169138A1 (en) 2018-02-28 2019-09-06 Evelo Biosciences, Inc. Compositions and methods for treating cancer using paraclostridium benzoelyticum
WO2019169168A1 (en) 2018-02-28 2019-09-06 Evelo Biosciences, Inc. Compositions and methods for treating cancer using agathobaculum
WO2019178057A1 (en) 2018-03-12 2019-09-19 Evelo Biosciences, Inc. Methods of treating cancer using burkholderia
KR102857148B1 (ko) 2018-04-05 2025-09-11 사일런스 테라퓨틱스 게엠베하 안티센스 가닥의 5' 단부에 비닐포스포네이트를 갖는 siRNA
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
US12005120B2 (en) 2018-05-08 2024-06-11 Regulus Therapeutics Inc. Galnac conjugated modified oligonucleotides as miR-122 inhibitor having HCV antiviral activity with reduced hyperbilirubinemia side-effect
CU20200082A7 (es) 2018-05-09 2021-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para la reducción de la expresión de fxi
US12496311B2 (en) 2018-07-17 2025-12-16 Aronora, Inc. Methods for safely reducing thrombopoietin
JP2021531278A (ja) 2018-07-20 2021-11-18 レグルス セラピューティクス インコーポレイテッド オリゴヌクレオチドの経口送達のための方法
EP3833397A4 (en) 2018-08-08 2023-06-14 Arcturus Therapeutics, Inc. COMPOSITIONS AND AGENTS AGAINST NON-ALCOHOLIC STEATOHEPATITIS
WO2020038377A1 (zh) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
US11273137B2 (en) 2018-09-04 2022-03-15 Board Of Trustees Of Michigan State University Methods and compositions to prevent and treat disorders associated with mutations in the ODC1 gene
KR102769565B1 (ko) 2018-09-19 2025-02-20 애로우헤드 파마슈티컬스 인코포레이티드 17베타-HSD 유형 13- (HSD17B13)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
TW202542311A (zh) 2018-09-19 2025-11-01 美商Ionis製藥公司 Pnpla3表現之調節劑
US11896674B2 (en) 2018-09-30 2024-02-13 Suzhou Ribo Life Science Co., Ltd. SiRNA conjugate, preparation method therefor and use thereof
CR20210395A (es) 2018-12-21 2021-11-05 Ionis Pharmaceuticals Inc Moduladores de la expresión de hsd17b13
WO2020135673A1 (zh) * 2018-12-28 2020-07-02 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
WO2020135581A1 (zh) * 2018-12-28 2020-07-02 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
SG11202105577TA (en) 2019-01-09 2021-06-29 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use
WO2020163747A1 (en) 2019-02-07 2020-08-13 Arrowhead Pharmaceuticals, Inc. Rnai agents for hepatitis b virus infection
CA3132199A1 (en) 2019-03-12 2020-09-17 President And Fellows Of Harvard College Methods and compositions for treating cancer
CA3132170A1 (en) 2019-04-18 2020-10-22 Michael BIERMER Combination therapy for treating hepatitis b virus infection
US20200332297A1 (en) 2019-04-18 2020-10-22 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
US11510939B1 (en) 2019-04-19 2022-11-29 Apellis Pharmaceuticals, Inc. RNAs for complement inhibition
US12590304B2 (en) 2019-05-22 2026-03-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
KR20220011689A (ko) 2019-05-22 2022-01-28 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 약제학적 조성물, 접합체, 제조 방법, 및 용도
AU2020280439B2 (en) 2019-05-22 2025-08-14 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
CN113795582B (zh) 2019-05-24 2024-05-24 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).
US20220226269A1 (en) 2019-06-12 2022-07-21 President And Fellows Of Harvard College Methods and compositions for modulation of an interspecies gut bacterial pathway for levodopa metabolism
KR20220042116A (ko) 2019-06-18 2022-04-04 얀센 사이언시즈 아일랜드 언리미티드 컴퍼니 B형 간염 바이러스(HBV) 백신 및 HBV-타케팅 RNAi의 조합
CA3143679A1 (en) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company Combination of hepatitis b virus (hbv) vaccines and hbv-targeting rnai
CN110218728A (zh) * 2019-06-28 2019-09-10 厦门甘宝利生物医药有限公司 一种新化合物及其应用
JP2022543191A (ja) * 2019-07-30 2022-10-11 エムペグ エルエイ リミテッド ライアビリティ カンパニー 増強された生理活性を有する多量体オリゴヌクレオチドの皮下送達
WO2021022110A1 (en) 2019-08-01 2021-02-04 Evelo Biosciences, Inc. Inducing immune effects using bacteria of the genus bifidobacterium
WO2021074772A1 (en) 2019-10-14 2021-04-22 Astrazeneca Ab Modulators of pnpla3 expression
US20230054711A1 (en) * 2019-12-31 2023-02-23 Xiamen University Multimerization delivery system for intracellular delivery of molecule
US20230070129A1 (en) 2020-01-06 2023-03-09 Seasun Therapeutics Composition for preventing or treating macular degeneration, containing cell permeable nucleic acid complex as active ingredient
CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
KR20230004456A (ko) 2020-03-04 2023-01-06 리제너론 파아마슈티컬스, 인크. 면역 요법에 대한 종양 세포의 감작화를 위한 방법 및 조성물
US20210285000A1 (en) 2020-03-05 2021-09-16 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
AR121672A1 (es) 2020-03-26 2022-06-29 Arrowhead Pharmaceuticals Inc AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3, COMPOSICIONES FARMACÉUTICAS DE ESTA, Y MÉTODOS DE USO
CN115916262A (zh) 2020-04-21 2023-04-04 旗舰创业股份有限公司 双官能分子及其使用方法
EP4157264A4 (en) 2020-05-27 2024-06-19 The Regents of the University of California COMPOSITIONS AND METHODS FOR CELL TRANSDIFFERENTIATION
EP4168547A1 (en) 2020-06-22 2023-04-26 Janssen Pharmaceuticals, Inc. Compositions and methods for treatment of hepatitis d virus infection
CA3189065A1 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
CN116348150A (zh) 2020-09-11 2023-06-27 箭头药业股份有限公司 用于递送治疗剂的脂质缀合物
MX2023002939A (es) 2020-09-11 2023-04-11 Arrowhead Pharmaceuticals Inc Plataformas para el transporte de farmacos hacia el musculo esqueletico y metodos de uso.
WO2022056454A2 (en) 2020-09-14 2022-03-17 President And Fellows Of Harvard College Methods and compositions for treating hpv-positive cancers
AU2021344422A1 (en) 2020-09-16 2023-05-04 Gordon J. Freeman Methods of treating an individual that has failed an anti-pd-1/anti-pd-l1 therapy
LT4136092T (lt) 2020-11-18 2024-09-25 Ionis Pharmaceuticals, Inc. Junginiai ir būdai, skirti angiotenzinogeno raiškos moduliavimui
TW202245809A (zh) 2020-12-18 2022-12-01 美商詹森藥物公司 用於治療b型肝炎病毒感染之組合療法
WO2022152869A1 (en) 2021-01-15 2022-07-21 Janssen Sciences Ireland Unlimited Company Use of oligonucleotides for individuals with hepatic impairment
CN114767704B (zh) * 2021-01-21 2024-06-14 圣诺制药公司 一种能够靶向乙型肝炎病毒的药物构造及药物组合物
JP2024516096A (ja) 2021-04-08 2024-04-12 アローヘッド ファーマシューティカルズ インコーポレイテッド 終末糖化産物受容体の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用方法
MX2023014026A (es) 2021-05-28 2023-12-12 Arrowhead Pharmaceuticals Inc Agentes de rnai para inhibir la expresion de mucina 5ac (muc5ac), composiciones de estos y metodos de uso.
US11629349B2 (en) 2021-06-21 2023-04-18 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
JP2024527380A (ja) 2021-07-09 2024-07-24 グラクソスミスクライン、インテレクチュアル、プロパティー、（ナンバー３）、リミテッド 腎機能障害を有する個体へのオリゴヌクレオチドの使用
US12042509B2 (en) 2021-10-01 2024-07-23 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
PE20241353A1 (es) 2021-11-09 2024-07-03 Viaqua Therapeutics Ltd Composiciones para acuicultura
US20250230412A1 (en) 2022-02-01 2025-07-17 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
JP2025522433A (ja) 2022-06-15 2025-07-15 アローヘッド ファーマシューティカルズ インコーポレイテッド スーパーオキシドジスムターゼ１（ＳＯＤ１）の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用の方法
WO2024151942A2 (en) 2023-01-13 2024-07-18 Hemoshear Therapeutics, Inc. Sirnas targeting slc10a1 transcripts, compositions and uses thereof
EP4684015A2 (en) 2023-03-21 2026-01-28 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement factor b (cfb), pharmaceutical compositions thereof, and methods of use
TW202448484A (zh) 2023-04-20 2024-12-16 美商雅迪克斯製藥公司 Ｍａｐｔ調節組合物及其使用方法
WO2024238396A1 (en) 2023-05-12 2024-11-21 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
CN121335980A (zh) 2023-05-26 2026-01-13 阿达尔克斯制药有限公司 Sod1调节组合物及其使用方法
EP4731763A1 (en) 2023-06-20 2026-04-29 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
WO2025038750A2 (en) 2023-08-14 2025-02-20 President And Fellows Of Harvard College Methods and compositions for treating cancer
IL326882A (en) 2023-08-30 2026-04-01 Arrowhead Pharmaceuticals Inc RNAi agents for inhibiting expression of inhibitory subunit beta E (INHBE), their pharmaceutical compositions and methods of use
WO2025072748A2 (en) 2023-09-29 2025-04-03 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease
WO2025090926A1 (en) * 2023-10-26 2025-05-01 The Penn State Research Foundation Ultrasound-sensitive peptide particles with surface-bound gene editing tools for spatially resolved molecule delivery
WO2025137384A1 (en) 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Methods and reagents for improved oligonucleotide synthesis
WO2025155911A1 (en) 2024-01-18 2025-07-24 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hurler syndrome
US12553050B2 (en) 2024-03-22 2026-02-17 Takeda Pharmaceutical Company Limited Compositions and methods for inhibiting cytochrome P450 family 7 subfamily a member 1 (CYP7A1) expression
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
WO2025226980A2 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease
WO2025242321A1 (en) 2024-05-24 2025-11-27 Glaxosmithkline Intellectual Property (No.3) Limited Novel use
WO2025265013A2 (en) 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
US20260015617A1 (en) 2024-07-12 2026-01-15 Novartis Ag DOUBLE STRANDED RNAi AGENTS, COMPOSITIONS AND METHODS OF USE
WO2026044017A2 (en) 2024-08-21 2026-02-26 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050239204A1 (en) * 1994-09-28 2005-10-27 Wyeth Multifunctional molecular complexes for gene transfer to cells
WO2008022309A2 (en) * 2006-08-18 2008-02-21 F. Hoffmann-La Roche Ag Polyconjugates for in vivo delivery of polynucleotides
US20080281041A1 (en) * 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5166320A (en) 1987-04-22 1992-11-24 University Of Connecticut Carrier system and method for the introduction of genes into mammalian cells
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
NZ244306A (en) 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
WO1993019768A1 (en) 1992-04-03 1993-10-14 The Regents Of The University Of California Self-assembling polynucleotide delivery system
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
JP2690276B2 (ja) 1995-01-10 1997-12-10 科学技術振興事業団 静電結合型高分子ミセル薬物担体とその薬剤
CN1120707C (zh) 1995-11-22 2003-09-10 约翰斯·霍普金斯大学 增强生物分子的细胞摄取的配体
US6630351B1 (en) 1999-06-07 2003-10-07 Mirus Corporation Compositions and methods for drug delivery using pH sensitive molecules
US20020165183A1 (en) 1999-11-29 2002-11-07 Hans Herweijer Methods for genetic immunization
US8217015B2 (en) 2003-04-04 2012-07-10 Arrowhead Madison Inc. Endosomolytic polymers
US5994316A (en) 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
US6020457A (en) 1996-09-30 2000-02-01 Dendritech Inc. Disulfide-containing dendritic polymers
GB9623051D0 (en) 1996-11-06 1997-01-08 Schacht Etienne H Delivery of DNA to target cells in biological systems
DE19652586A1 (de) 1996-12-17 1998-06-18 Biotechnolog Forschung Gmbh Dhc-Peptid und Mittel
CA2294988C (en) 1997-07-01 2015-11-24 Isis Pharmaceuticals Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
US5877309A (en) 1997-08-13 1999-03-02 Isis Pharmaceuticals, Inc. Antisense oligonucleotides against JNK
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US8038984B2 (en) 1998-06-20 2011-10-18 Washington University Membrane-permeant peptide complexes for treatment of sepsis
US20050112470A1 (en) * 1998-06-26 2005-05-26 Johnson Controls Technology Company Alloy for battery grids
US6773920B1 (en) 1999-03-31 2004-08-10 Invitrogen Corporation Delivery of functional protein sequences by translocating polypeptides
US8211468B2 (en) 1999-06-07 2012-07-03 Arrowhead Madison Inc. Endosomolytic polymers
US7019113B2 (en) 1999-06-07 2006-03-28 Mirus Bio Corporation Reversible modification of membrane interaction
US7442764B2 (en) 1999-06-07 2008-10-28 Mirns Bio Corporation Reversible modification of amine-containing compounds
US20040072785A1 (en) 1999-11-23 2004-04-15 Wolff Jon A. Intravascular delivery of non-viral nucleic acid
US7098030B2 (en) 1999-12-31 2006-08-29 Mirus Bio Corporation Polyampholytes for delivering polyions to a cell
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20030072794A1 (en) 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
JP2002122707A (ja) 2000-10-13 2002-04-26 Canon Inc 非球面マイクロ構造体、及びその作製方法
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20030157030A1 (en) 2001-11-02 2003-08-21 Insert Therapeutics, Inc. Methods and compositions for therapeutic use of rna interference
US7176303B2 (en) 2003-11-06 2007-02-13 Isis Pharmaceuticals, Inc. Modulation of STAT5 expression
DE60328383D1 (de) 2002-05-24 2009-08-27 Mirus Bio Corp Zusammensetzungen zur zuführung von nukleinsäuren an zellen
US20050008617A1 (en) 2002-09-28 2005-01-13 Massachusetts Institute Of Technology Compositions and methods for delivery of short interfering RNA and short hairpin RNA
EP1585756B1 (en) 2002-11-26 2010-04-21 University of Massachusetts Delivery of sirnas
US7682626B2 (en) 2003-02-07 2010-03-23 Roche Madison Inc. Polyvinylethers for delivery of polynucleotides to mammalian cells
US7816337B2 (en) 2003-02-18 2010-10-19 Roche Madison Inc. Reversible attachment of a membrane active polymer to a polynucleotide
US8324365B2 (en) 2003-04-03 2012-12-04 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US8454566B2 (en) 2003-07-10 2013-06-04 Medtronic Minimed, Inc. Methods and compositions for the inhibition of biofilms on medical devices
US20060040882A1 (en) 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US20060008907A1 (en) 2004-06-09 2006-01-12 The Curators Of The University Of Missouri Control of gene expression via light activated RNA interference
CA2587411A1 (en) 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
US20060166234A1 (en) 2004-11-22 2006-07-27 Barbara Robertson Apparatus and system having dry control gene silencing compositions
US20080206869A1 (en) * 2005-01-24 2008-08-28 Avaris Ab Nucleic Acid Complex
MX2007012157A (es) 2005-04-01 2008-03-14 Intezyne Technologies Llc Micelas polimericas para suministro de farmacos.
US9139850B2 (en) 2005-05-19 2015-09-22 L'oreal Vectorization of dsRNA by cationic particles and topical use
JP2007112768A (ja) * 2005-10-24 2007-05-10 Kyoto Univ 肝指向性リポソーム組成物
US8017109B2 (en) 2006-08-18 2011-09-13 Roche Madison Inc. Endosomolytic poly(acrylate) polymers
SI2539451T1 (sl) * 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050239204A1 (en) * 1994-09-28 2005-10-27 Wyeth Multifunctional molecular complexes for gene transfer to cells
US20080281041A1 (en) * 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
WO2008022309A2 (en) * 2006-08-18 2008-02-21 F. Hoffmann-La Roche Ag Polyconjugates for in vivo delivery of polynucleotides
US20080152661A1 (en) * 2006-08-18 2008-06-26 Rozema David B Polyconjugates for In Vivo Delivery of Polynucleotides

Also Published As

Publication number Publication date
CA2788600A1 (en) 2011-09-01
SMT201600079B (it) 2016-04-29
EP2539451B1 (en) 2016-01-06
SG183374A1 (en) 2012-09-27
PL2539451T3 (pl) 2016-08-31
IL221396A0 (en) 2012-10-31
JP2013520460A (ja) 2013-06-06
RS54578B1 (sr) 2016-06-30
EP3023495B1 (en) 2019-05-08
US8313772B2 (en) 2012-11-20
US20130245091A1 (en) 2013-09-19
US9345775B2 (en) 2016-05-24
AU2011219941A1 (en) 2012-08-16
KR20130012112A (ko) 2013-02-01
SI2539451T1 (sl) 2016-04-29
IL221396A (en) 2016-11-30
US9011919B2 (en) 2015-04-21
BR112012021256A2 (pt) 2018-03-06
US20110207799A1 (en) 2011-08-25
AU2011219941C1 (en) 2015-05-07
US20150202298A1 (en) 2015-07-23
CN102869774A (zh) 2013-01-09
US20170204408A9 (en) 2017-07-20
CN102869774B (zh) 2018-02-09
JP2016074671A (ja) 2016-05-12
WO2011104169A1 (en) 2011-09-01
KR101956623B1 (ko) 2019-03-12
US10316316B2 (en) 2019-06-11
EA024534B1 (ru) 2016-09-30
DK2539451T3 (da) 2016-04-04
AU2011219941B2 (en) 2014-10-23
JP6137834B2 (ja) 2017-05-31
CA2788600C (en) 2019-12-03
HUE026811T2 (en) 2016-07-28
US20160222384A1 (en) 2016-08-04
JP6290848B2 (ja) 2018-03-07
EP3023495A1 (en) 2016-05-25
MX2012009178A (es) 2012-11-30
EA201290602A1 (ru) 2013-04-30
ZA201205685B (en) 2013-09-25
PH12012501596A1 (en) 2017-02-03
AP2012006453A0 (en) 2012-08-31
PT2539451E (pt) 2016-03-28
ES2562817T3 (es) 2016-03-08
NZ601737A (en) 2013-06-28
HRP20160093T1 (hr) 2016-02-26
EP2539451A1 (en) 2013-01-02

Similar Documents

Publication Publication Date Title
AP3284A (en) Compositions for targeted delivery of SIRNA
GB201121070D0 (en) composition for delivery of biotherapeutics
SI2791160T1 (sl) Sestave modificirane MRNA
HUE042850T2 (hu) Készítmények hatóanyag szállításra
IL224390B (en) Compositions and methods for targeted thermal modulation
LT2640370T (lt) Kompozicijos, skirtos aktyvių agentų įvedimui po oda
ZA201304687B (en) Biodegradable drug delivery compositions
EP2470019A4 (en) COMPOSITIONS FOR THE ADMINISTRATION OF NON-SOLUBLE AGENTS
GB201308796D0 (en) Customisation of mobile-application delivery
PT2768864T (pt) Composições com concentrações aumentadas de carboximetilcelulose
EP2794879A4 (en) COMPOSITIONS AND METHODS FOR RELEASING BIOLOGICALLY ACTIVE RNA
ZA201003844B (en) Delivery of functional compounds
ZA201402166B (en) Compositions of efavirenz
GB201407689D0 (en) Pharmaceutical compositions of antihypertensives
GB201014935D0 (en) Enhanced delivery of siRNA
GB201015091D0 (en) Enhanced delivery of sirna
GB201014931D0 (en) Enhanced delivery of nucleic
IL226237B (en) Pharmaceutical preparations for administration of active substances through the skin
GB201001900D0 (en) Transdermal delivery composition