CA2910760C - Targeting lipids - Google Patents

Targeting lipids Download PDF

Info

Publication number
CA2910760C
CA2910760C CA2910760A CA2910760A CA2910760C CA 2910760 C CA2910760 C CA 2910760C CA 2910760 A CA2910760 A CA 2910760A CA 2910760 A CA2910760 A CA 2910760A CA 2910760 C CA2910760 C CA 2910760C
Authority
CA
Canada
Prior art keywords
lipid
acid
nucleic acid
rna
gene
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.)
Active
Application number
CA2910760A
Other languages
English (en)
French (fr)
Other versions
CA2910760A1 (en
Inventor
Muthiah Manoharan
Kallanthottathil G. Rajeev
Jayaprakash K. Narayanannair
Muthusamy Jayaraman
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.)
Arbutus Biopharma Corp
Original Assignee
Arbutus Biopharma 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 Arbutus Biopharma Corp filed Critical Arbutus Biopharma Corp
Priority to CA3043911A priority Critical patent/CA3043911A1/en
Publication of CA2910760A1 publication Critical patent/CA2910760A1/en
Application granted granted Critical
Publication of CA2910760C publication Critical patent/CA2910760C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • 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
    • A61K47/554—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 the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • 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
    • 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
    • A61K31/7004—Monosaccharides having only carbon, hydrogen and oxygen atoms
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, 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
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02P—CLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00—Technologies relating to chemical industry
    • Y02P20/50—Improvements relating to the production of bulk chemicals
    • Y02P20/55—Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Botany (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Steroid Compounds (AREA)
CA2910760A 2007-12-04 2008-12-04 Targeting lipids Active CA2910760C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3043911A CA3043911A1 (en) 2007-12-04 2008-12-04 Targeting lipids

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US99230907P 2007-12-04 2007-12-04
US60/992,309 2007-12-04
US1359707P 2007-12-13 2007-12-13
US61/013,597 2007-12-13
US12775108P 2008-05-14 2008-05-14
US61/127,751 2008-05-14
US9109308P 2008-08-22 2008-08-22
US61/091,093 2008-08-22
US9726108P 2008-09-16 2008-09-16
US61/097,261 2008-09-16
CA2708173A CA2708173C (en) 2007-12-04 2008-12-04 Targeting lipids

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CA2708173A Division CA2708173C (en) 2007-12-04 2008-12-04 Targeting lipids

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA3043911A Division CA3043911A1 (en) 2007-12-04 2008-12-04 Targeting lipids

Publications (2)

Publication Number Publication Date
CA2910760A1 CA2910760A1 (en) 2009-07-02
CA2910760C true CA2910760C (en) 2019-07-09

Family

ID=40599687

Family Applications (6)

Application Number Title Priority Date Filing Date
CA2910760A Active CA2910760C (en) 2007-12-04 2008-12-04 Targeting lipids
CA3043911A Abandoned CA3043911A1 (en) 2007-12-04 2008-12-04 Targeting lipids
CA2708153A Active CA2708153C (en) 2007-12-04 2008-12-04 Carbohydrate conjugates as delivery agents for oligonucleotides
CA2708173A Active CA2708173C (en) 2007-12-04 2008-12-04 Targeting lipids
CA2930393A Active CA2930393C (en) 2007-12-04 2008-12-04 Carbohydrate conjugates as delivery agents for oligonucleotides
CA3146103A Pending CA3146103A1 (en) 2007-12-04 2008-12-04 Carbohydrate conjugates as delivery agents for oligonucleotides

Family Applications After (5)

Application Number Title Priority Date Filing Date
CA3043911A Abandoned CA3043911A1 (en) 2007-12-04 2008-12-04 Targeting lipids
CA2708153A Active CA2708153C (en) 2007-12-04 2008-12-04 Carbohydrate conjugates as delivery agents for oligonucleotides
CA2708173A Active CA2708173C (en) 2007-12-04 2008-12-04 Targeting lipids
CA2930393A Active CA2930393C (en) 2007-12-04 2008-12-04 Carbohydrate conjugates as delivery agents for oligonucleotides
CA3146103A Pending CA3146103A1 (en) 2007-12-04 2008-12-04 Carbohydrate conjugates as delivery agents for oligonucleotides

Country Status (7)

Country Link
US (15) US8106022B2 (https=)
EP (8) EP4074344A1 (https=)
JP (13) JP5635412B2 (https=)
CN (1) CN102006890A (https=)
AU (2) AU2008333811B2 (https=)
CA (6) CA2910760C (https=)
WO (2) WO2009082607A2 (https=)

Families Citing this family (711)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8586781B2 (en) 1998-04-02 2013-11-19 Mbc Pharma, Inc. Bone targeted therapeutics and methods of making and using the same
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
DK1620544T3 (en) * 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
US8575327B2 (en) 2003-06-12 2013-11-05 Alnylam Pharmaceuticals, Inc. Conserved HBV and HCV sequences useful for gene silencing
US9393315B2 (en) 2011-06-08 2016-07-19 Nitto Denko Corporation Compounds for targeting drug delivery and enhancing siRNA activity
AU2007299629C1 (en) 2006-09-21 2012-05-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the HAMP gene
WO2009082607A2 (en) * 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
EP2231194B1 (en) * 2007-12-04 2017-02-22 Alnylam Pharmaceuticals Inc. Folate-irna conjugates
WO2009126933A2 (en) 2008-04-11 2009-10-15 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
CA2746527A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
MX2011004097A (es) 2008-10-15 2011-07-28 Isis Pharmaceuticals Inc Modulacion de la expresion del factor 11.
EA036772B1 (ru) 2008-10-20 2020-12-18 Элнилэм Фармасьютикалз, Инк. Композиции и способы для ингибирования экспрессии транстиретина
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
EP3243504A1 (en) * 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
EP2669290A1 (en) 2009-03-02 2013-12-04 Alnylam Pharmaceuticals Inc. Nucleic Acid Chemical Modifications
ES2804764T3 (es) 2009-06-01 2021-02-09 Halo Bio Rnai Therapeutics Inc Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos
NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
HUE042177T2 (hu) 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
WO2011102905A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion diagnostic delivery system
WO2011102904A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion therapeutic delivery system
WO2011102906A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion delivery system
US9080171B2 (en) * 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
BR112012024049A2 (pt) 2010-03-24 2017-03-01 Rxi Pharmaceuticals Corp interferência de rna em indicações dérmicas e fibróticas
CA2792561C (en) 2010-04-06 2021-10-26 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
DK2558578T3 (en) 2010-04-13 2016-01-25 Life Technologies Corp CONFIGURATIONS AND METHODS FOR INHIBITION OF nucleic acid function
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
CA2800693A1 (en) 2010-05-28 2011-12-01 Purdue Research Foundation Delivery of therapeutic agents to inflamed tissues using folate-targeted agents
US8785121B2 (en) 2010-07-08 2014-07-22 Bonac Corporation Single-stranded nucleic acid molecule for controlling gene expression
AU2011282977A1 (en) * 2010-07-28 2013-02-21 Smartcells, Inc. Drug-ligand conjugates, synthesis thereof, and intermediates thereto
US8691782B2 (en) 2010-08-03 2014-04-08 Bonac Corporation Single-stranded nucleic acid molecule having nitrogen-containing alicyclic skeleton
ES2527660T3 (es) * 2010-08-03 2015-01-28 Bonac Corporation Molécula de ARN monocatenario que tiene esqueleto alicíclico que contiene nitrógeno
WO2012027206A1 (en) 2010-08-24 2012-03-01 Merck Sharp & Dohme Corp. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
CN101974152B (zh) * 2010-09-28 2012-07-11 山东大学 一种类脂-阳离子聚合物及其制备方法
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
CA3077910A1 (en) 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
EP2652134B1 (en) 2010-12-17 2017-03-01 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
ES2605990T3 (es) 2010-12-29 2017-03-17 F. Hoffmann-La Roche Ag Conjugados de molécula pequeña para la administración intracelular de ácidos nucleicos
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
KR102365961B1 (ko) 2011-03-29 2022-02-23 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
EP2723861A4 (en) * 2011-06-21 2014-12-10 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING HEPCIDINE ANTIMICROBIAL PEPTIDE (HAMP) OR THE ASSOCIATED GENE EXPRESSION
ES2923573T3 (es) 2011-06-21 2022-09-28 Alnylam Pharmaceuticals Inc Composiciones de ARNi de proteína 3 de tipo angiopoyetina (ANGPTL3) y métodos de uso de las mismas
CN103649103A (zh) 2011-06-21 2014-03-19 阿尔尼拉姆医药品有限公司 用于抑制载脂蛋白c-iii(apoc3)基因表达的组合物与方法
WO2012177949A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
EP3366312A1 (en) * 2011-06-23 2018-08-29 Alnylam Pharmaceuticals, Inc. Serpina 1 sirnas: compositions of matter and methods of treatment
HRP20181564T1 (hr) 2011-06-30 2018-11-30 Arrowhead Pharmaceuticals, Inc. Pripravci i postupci za inhibiciju ekspresije gena virusa hepatitisa b
CA2843885C (en) 2011-08-01 2020-03-10 Mbc Pharma, Inc. Vitamin b6 derivatives of nucleotides, acyclonucleotides and acyclonucleoside phosphonates
EP3453761A1 (en) 2011-08-29 2019-03-13 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2760477B1 (en) 2011-09-27 2018-08-08 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
EP2594575A1 (en) * 2011-11-15 2013-05-22 Université de Strasbourg Mannosylated compounds useful for the prevention and the treatment of infectious diseases
EP2780454A2 (en) 2011-11-18 2014-09-24 Alnylam Pharmaceuticals, Inc. Modified rnai agents
HUE048622T2 (hu) * 2011-11-18 2020-08-28 Alnylam Pharmaceuticals Inc RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére
EP2788006B1 (en) 2011-12-07 2026-01-07 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
MX375673B (es) 2012-04-02 2025-03-06 Berg Llc Ensayos basados en interrogatorios celulares y usos de los mismos.
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
AR090906A1 (es) * 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y procedimientos para la administracion de oligonucleotidos
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
CN109810977A (zh) 2012-05-26 2019-05-28 株式会社博纳克 具有递送功能的基因表达调控用单链核酸分子
MX369768B (es) 2012-06-01 2019-11-21 Berg Llc Composición farmacéutica de coenzima q10 para usarse en un método para tratar cáncer.
WO2013183656A1 (ja) * 2012-06-04 2013-12-12 大日本住友製薬株式会社 Ｇタンパク質共役受容体結合リガンドと核酸分子とのコンジュゲート
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
US10245229B2 (en) 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
JP6427097B2 (ja) 2012-06-15 2018-11-21 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドＴｈｅ Ｂｒｉｇｈａｍ ａｎｄ Ｗｏｍｅｎ’ｓ Ｈｏｓｐｉｔａｌ， Ｉｎｃ． 癌を処置するための組成物および該組成物を製造するための方法
AU2013299717B2 (en) * 2012-08-06 2018-06-28 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated RNA agents and process for their preparation
CN102824515B (zh) * 2012-09-18 2015-05-20 淄博齐鼎立专利信息咨询有限公司 热毒清片在制备抑制a-204细胞增殖药物中的应用
MX2015005328A (es) 2012-10-26 2015-09-25 Nlife Therapeutics S L Composiciones y metodos para la administracion selectiva de moleculas de oligonucleotidos a tipos de celulas.
US10077443B2 (en) 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
US9506897B2 (en) 2012-11-16 2016-11-29 Agilent Technologies, Inc. Methods and compositions for improved ion-exchange chromatography
US10125369B2 (en) * 2012-12-05 2018-11-13 Alnylam Pharmaceuticals, Inc. PCSK9 iRNA compositions and methods of use thereof
US20150337318A1 (en) * 2013-01-11 2015-11-26 Phaserx Inc. Rna targeted to c-met
CN104955952A (zh) * 2013-01-30 2015-09-30 弗·哈夫曼-拉罗切有限公司 Lna寡核苷酸碳水化合物缀合物
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
CA3216595A1 (en) 2013-03-14 2014-10-02 Kevin Fitzgerald Complement component c5 irna compositions and methods of use thereof
DK2970456T3 (da) 2013-03-14 2021-07-05 Translate Bio Inc Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer
PL3467108T3 (pl) 2013-03-14 2024-09-30 Translate Bio, Inc. Sposoby oczyszczania informacyjnego rna
CA2902571A1 (en) 2013-03-15 2014-09-18 MiRagen Therapeutics, Inc. Bridged bicyclic nucleosides
US10130649B2 (en) 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
SG10201903112WA (en) 2013-04-08 2019-05-30 Berg Llc Treatment of cancer using coenzyme q10 combination therapies
CN105164261B (zh) * 2013-05-01 2022-03-18 莱古路斯治疗法股份有限公司 用于增强的细胞摄取的化合物和方法
SG11201508925WA (en) 2013-05-01 2015-11-27 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-122
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
WO2014182661A2 (en) 2013-05-06 2014-11-13 Alnylam Pharmaceuticals, Inc Dosages and methods for delivering lipid formulated nucleic acid molecules
RU2535052C1 (ru) * 2013-05-21 2014-12-10 Владимир Николаевич Иванов Фармацевтическая композиция, содержащая производные лизина, пролина и тритерпеновой кислоты для лечения и профилактики вирусных инфекций, вызываемых рнк и днк-содержащими вирусами, такими как: грипп, герпес, опоясывающий лишай, папиллома человека, аденоновирусы, а также бактериальных инфекций, вызываемых грам-положительными и грам-отрицательными микроорганизмами
KR102486617B1 (ko) * 2013-05-22 2023-01-12 알닐람 파마슈티칼스 인코포레이티드 Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법
TW201936624A (zh) * 2013-05-22 2019-09-16 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
EP3564374A1 (en) 2013-06-21 2019-11-06 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
ES2770667T3 (es) 2013-06-27 2020-07-02 Roche Innovation Ct Copenhagen As Oligómeros antisentido y conjugados que se dirigen a PCSK9
CN105705638B (zh) * 2013-07-05 2019-11-26 柏业公司 改善的具有高效能的纳米颗粒型寡核苷酸结构及其制备方法
JP6702862B2 (ja) 2013-07-11 2020-06-03 アルニラム ファーマスーティカルズ インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法
KR102370843B1 (ko) 2013-09-04 2022-03-04 버그 엘엘씨 코엔자임 q10의 연속주입에 의한 암치료 방법
EP3046585B1 (en) 2013-09-17 2021-08-25 Bolt Threads, Inc. Methods and compositions for synthesizing improved silk fibers
WO2015042447A1 (en) 2013-09-20 2015-03-26 Isis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
TW201610151A (zh) 2013-09-23 2016-03-16 阿尼拉製藥公司 治療或預防與甲狀腺素運載蛋白相關疾病之方法
US10077444B2 (en) * 2013-10-02 2018-09-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
TWI768330B (zh) 2013-10-04 2022-06-21 美國西奈山伊坎醫學院 抑制alas1基因表現的組合物及方法
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
US9994846B2 (en) 2013-10-25 2018-06-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
KR20160083876A (ko) * 2013-11-14 2016-07-12 로슈 이노베이션 센터 코펜하겐 에이/에스 ApoB 안티센스 접합체 화합물
WO2015084897A2 (en) 2013-12-02 2015-06-11 Mirimmune, Llc Immunotherapy of cancer
SG10201804960RA (en) 2013-12-12 2018-07-30 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
CN105899663B (zh) 2013-12-26 2019-07-26 学校法人东京医科大学 用于基因表达控制的人工模拟miRNA及其用途
SG10201913570XA (en) 2013-12-27 2020-03-30 Bonac Corp Artificial match-type mirna for controlling gene expression and use therefor
WO2015106128A2 (en) * 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
JP6584412B2 (ja) * 2014-01-15 2019-10-02 ベースクリック ゲーエムベーハー 糖修飾核酸分子
WO2015113922A1 (en) * 2014-01-30 2015-08-06 Roche Innovation Center Copenhagen A/S Poly oligomer compound with biocleavable conjugates
AU2015217301A1 (en) 2014-02-11 2016-08-25 Alnylam Pharmaceuticals, Inc. Ketohexokinase (KHK) iRNA compositions and methods of use thereof
CN106103717A (zh) 2014-03-19 2016-11-09 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
RS60707B1 (sr) 2014-04-01 2020-09-30 Biogen Ma Inc Kompozicije za modulaciju ekspresije sod-1
KR20160132496A (ko) 2014-04-03 2016-11-18 인빅터스 온콜로지 피비티. 엘티디. 초분자 조합 치료제
PT3134506T (pt) 2014-04-25 2019-10-31 Translate Bio Inc Métodos de purificação de rna mensageiro
DK3137476T3 (da) 2014-04-28 2019-11-18 Ionis Pharmaceuticals Inc Linker-modificerede oligomerforbindelser
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
SG11201608502TA (en) 2014-05-01 2016-11-29 Ionis Pharmaceuticals Inc Compositions and methods for modulating complement factor b expression
EP3862362A3 (en) 2014-05-01 2021-10-27 Ionis Pharmaceuticals, Inc. Conjugates of modified antisense oligonucleotides and their use for modulating pkk expression
PT3137605T (pt) 2014-05-01 2020-12-18 Ionis Pharmaceuticals Inc Composições e métodos para modulação da expressão de angiopoietina de tipo 3
CN110903337A (zh) * 2014-05-01 2020-03-24 Ionis制药公司 用于调节生长激素受体表达的组合物和方法
US10098959B2 (en) 2014-05-01 2018-10-16 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法和組成物
CA3215908A1 (en) 2014-05-22 2015-11-26 Alnylam Pharmaceuticals, Inc. Angiotensinogen (agt) irna compositions and methods of use thereof
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
JP6557722B2 (ja) 2014-05-30 2019-08-07 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 核酸の送達のための生分解性脂質
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
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
EP3160959B1 (en) 2014-06-24 2023-08-30 Translate Bio, Inc. Stereochemically enriched compositions for delivery of nucleic acids
SMT202200502T1 (it) 2014-06-25 2023-01-13 Acuitas Therapeutics Inc Nuovi lipidi e formulazioni di nanoparticelle lipidiche per l'erogazione di acidi nucleici
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
TW201613949A (en) 2014-08-07 2016-04-16 Regulus Therapeutics Inc Targeting microRNAs for metabolic disorders
WO2016030863A1 (en) 2014-08-29 2016-03-03 Glaxosmithkline Intellectual Property Development Limited Compounds and methods for treating viral infections
DK3185957T3 (da) 2014-08-29 2022-08-29 Alnylam Pharmaceuticals Inc Patisiran til anvendelse ved behandlingen af transthyretin-medieret amyloidosis
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
TWI864340B (zh) 2014-10-10 2024-12-01 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
WO2016055601A1 (en) 2014-10-10 2016-04-14 F. Hoffmann-La Roche Ag Galnac phosphoramidites, nucleic acid conjugates thereof and their use
US20170304459A1 (en) * 2014-10-10 2017-10-26 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhalation delivery of conjugated oligonucleotide
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3212794B1 (en) 2014-10-30 2021-04-07 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
TWI710633B (zh) 2014-11-10 2020-11-21 美商阿尼拉製藥公司 Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法
EP3240411A4 (en) 2014-11-12 2019-02-20 Tumorend LLC COMPOSITIONS, METHODS AND TREATMENTS FOR INHIBITING CELL ADHESION AND VIRUS BINDING AND PENETRATION
JP2017533721A (ja) 2014-11-14 2017-11-16 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． タンパク質の調節のための化合物及び方法
HK1244843A1 (zh) 2014-11-17 2018-08-17 Alnylam Pharmaceuticals, Inc. 载脂蛋白c3(apoc3)irna组合物及其使用方法
WO2016085852A1 (en) 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
SG10202012249YA (en) 2014-12-08 2021-01-28 Berg Llc Use of markers including filamin a in the diagnosis and treatment of prostate cancer
EP4372091A3 (en) 2014-12-12 2024-07-31 Tod M. Woolf Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
JP7105065B2 (ja) * 2014-12-15 2022-07-22 ダイセルナ ファーマシューティカルズ， インコーポレイテッド リガンド修飾二本鎖核酸
CA2970795A1 (en) 2014-12-18 2016-06-23 Alnylam Pharmaceuticals, Inc. Reversir compounds
EP4349414B1 (en) * 2014-12-23 2025-10-15 Dyve Biosciences, Inc. Formulations for transdermal administration
SG11201705223XA (en) 2014-12-27 2017-07-28 Bonac Corp NATURALLY OCCURING miRNA FOR CONTROLLING GENE EXPRESSION, AND USE OF SAME
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
WO2016123628A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of Colorado, A Body Corporate Sequence specific and organism specific antimicrobials and related materials and methods
WO2016130806A2 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
CN104804463B (zh) * 2015-03-10 2016-08-24 西安交通大学第一附属医院 用于靶向肿瘤组织的近红外荧光染色剂及制备方法和应用
US10172924B2 (en) 2015-03-19 2019-01-08 Translate Bio, Inc. MRNA therapy for pompe disease
JP6602847B2 (ja) 2015-03-27 2019-11-06 株式会社ボナック デリバリー機能と遺伝子発現制御能を有する一本鎖核酸分子
MX2017012426A (es) 2015-04-03 2018-01-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tmprss6.
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
WO2016164746A1 (en) 2015-04-08 2016-10-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
EA201792263A1 (ru) 2015-04-13 2018-08-31 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA ПРОТИВ АНГИОПОЭТИН-ПОДОБНОГО БЕЛКА 3 (ANGPTL3) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10709728B2 (en) 2015-06-01 2020-07-14 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting angiotensinogen (AGT) and methods of use thereof
CN104987360A (zh) 2015-06-04 2015-10-21 大连民族学院 一类双烷氧酰胺烷基阳离子肽脂质及其合成方法和应用
EP4365291A3 (en) 2015-06-12 2024-08-14 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
LT3313829T (lt) 2015-06-29 2024-08-12 Acuitas Therapeutics Inc. Lipidai ir lipidų nanodalelių sudėtys, skirtos nukleorūgščių tiekimui
GB201511459D0 (en) * 2015-06-30 2015-08-12 Cell Therapy Ltd Sirna microbubble
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
CN108135923B (zh) 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1（sod1）的核酸分子
CA2991894A1 (en) 2015-07-10 2017-01-19 Ionis Pharmaceuticals, Inc. Modulators of diacyglycerol acyltransferase 2 (dgat2)
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
EP3919619A1 (en) 2015-07-17 2021-12-08 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
TW201718620A (zh) 2015-07-27 2017-06-01 阿尼拉製藥公司 黃嘌呤脫氫酶（ＸＤＨ）ｉＲＮＡ組成物及其使用方法
CN108138182B (zh) 2015-07-31 2022-08-19 阿尔尼拉姆医药品有限公司 甲状腺素运载蛋白(TTR)iRNA组合物及其治疗或预防TTR相关疾病的使用方法
EP3328439B1 (en) 2015-07-31 2020-04-08 Arcturus Therapeutics, Inc. Multiligand agent for drug delivery
KR20180038465A (ko) 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 Ｂ형 간염 바이러스 감염에 대한 ｒｎａｉ 치료법
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP2018525037A (ja) 2015-08-24 2018-09-06 ハロー−バイオ・アールエヌエーアイ・セラピューティックス、インコーポレイテッド 遺伝子発現の調節のためのポリヌクレオチドナノ粒子及びその使用
CN118697894A (zh) 2015-08-25 2024-09-27 阿尔尼拉姆医药品有限公司 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物
KR20250145703A (ko) 2015-09-02 2025-10-13 알닐람 파마슈티칼스 인코포레이티드 프로그램된 세포사 1 리간드 1 (PD-L1) iRNA 조성물 및 그의 사용 방법
JP6877414B2 (ja) 2015-09-24 2021-05-26 アイオニス・ファーマシューティカルズ・インコーポレイテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｋｒａｓ発現のモジュレーター
US10533175B2 (en) 2015-09-25 2020-01-14 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Ataxin 3 expression
CN113817735A (zh) 2015-10-08 2021-12-21 Ionis制药公司 用于调节血管紧张素原表达的化合物和方法
EP3359640A4 (en) * 2015-10-09 2019-03-20 Dice Molecules SV, LLC LOGD ESTIMATING METHODS OF MARKED COMBINATORIAL LIBRARY COMPOUNDS
EP3359685B1 (en) 2015-10-09 2026-01-28 University Of Southampton Modulation of gene expression for deregulated protein expression
CN114686548A (zh) 2015-10-14 2022-07-01 川斯勒佰尔公司 用于增强生产的rna相关酶的修饰
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
IL307179A (en) 2015-10-28 2023-11-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
US20190046555A1 (en) 2015-11-06 2019-02-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
AU2016349625B2 (en) 2015-11-06 2022-07-07 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
US11058709B1 (en) 2015-12-04 2021-07-13 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
FI3386518T3 (fi) 2015-12-07 2025-09-25 Genzyme Corp Menetelmiä ja koostumuksia serpinc1:een liittyvän häiriön hoitamiseksi
WO2017100587A1 (en) * 2015-12-09 2017-06-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting programmed cell death 1 ligand 1 (pd-l1) and methods of use thereof
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
CA3005090A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
JP7054675B2 (ja) 2015-12-14 2022-04-14 コールド スプリング ハーバー ラボラトリー 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法
JP7049249B2 (ja) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 中枢神経系疾患の処置のための組成物および方法
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
US20190002887A1 (en) 2015-12-31 2019-01-03 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
CA3006599A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
AU2017213404A1 (en) 2016-01-29 2018-09-20 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
WO2017152149A1 (en) 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
KR102515329B1 (ko) * 2016-03-07 2023-03-29 애로우헤드 파마슈티컬스 인코포레이티드 치료용 화합물을 위한 표적화 리간드
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
AU2017234678A1 (en) 2016-03-16 2018-08-16 Ionis Pharmaceuticals, Inc. Methods of modulating KEAP1
WO2017173334A1 (en) 2016-04-01 2017-10-05 Checkmate Pharmaceuticals, Inc. Fc receptor-mediated drug delivery
WO2017177169A1 (en) 2016-04-08 2017-10-12 Rana Therapeutics, Inc. Multimeric coding nucleic acid and uses thereof
SG10202008301UA (en) * 2016-04-11 2020-10-29 Arbutus Biopharma Corp Targeted nucleic acid conjugate compositions
EP3442983A1 (en) 2016-04-14 2019-02-20 H. Hoffnabb-La Roche Ag TRITYL-MONO-Ga1NAc COMPOUNDS AND THEIR USE
CN110382692A (zh) 2016-04-19 2019-10-25 博德研究所 新型crispr酶以及系统
MA45295A (fr) * 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
WO2017189996A1 (en) * 2016-04-29 2017-11-02 Brandeis University Amino acid- and peptide-steroid conjugates and use thereof
SG10201913477UA (en) 2016-04-29 2020-02-27 Univ Nanyang Tech G-quadruplex-containing antisense oligonucleotides
JP6872560B2 (ja) 2016-05-06 2021-05-19 エム． ウルフ、トッド プログラム可能ヌクレアーゼのあるゲノム編集及びプログラム可能ヌクレアーゼのないゲノム編集のための改善された方法
EP3469083A1 (en) * 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
CA3027312A1 (en) 2016-06-13 2017-12-21 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
EP3471781A4 (en) 2016-06-17 2020-05-06 Ionis Pharmaceuticals, Inc. MODULATION OF GYS1 EXPRESSION
TWI746590B (zh) * 2016-06-30 2021-11-21 日商協和麒麟股份有限公司 核酸複合體
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
WO2018013525A1 (en) 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
ES2935658T3 (es) 2016-07-15 2023-03-09 Ionis Pharmaceuticals Inc Compuestos y métodos para la modulación de SMN2
IL291323B2 (en) 2016-07-15 2023-09-01 Am Chemicals Llc Solid non-nucleoside supports and phosphoramidite building blocks for oligonucleotide synthesis
JOP20190015A1 (ar) 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
JP2019532027A (ja) 2016-08-17 2019-11-07 ソルスティス バイオロジクス，リミティッド ポリヌクレオチド構築物
UY37376A (es) 2016-08-26 2018-03-23 Amgen Inc Construcciones de arni para inhibir expresión de asgr1 y métodos para su uso
MX2018009853A (es) 2016-09-02 2018-11-09 Arrowhead Pharmaceuticals Inc Ligandos de direccion.
KR101933767B1 (ko) * 2016-09-05 2018-12-28 (의료)길의료재단 발기부전 환자를 위한 콘돔
EP3522898A4 (en) * 2016-10-06 2020-05-27 Ionis Pharmaceuticals, Inc. METHOD FOR CONJUGATING OLIGOMERIC COMPOUNDS
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
WO2018102745A1 (en) 2016-12-02 2018-06-07 Cold Spring Harbor Laboratory Modulation of lnc05 expression
US10611823B2 (en) 2016-12-14 2020-04-07 Hanssen Biotech, Inc CD137 binding fibronectin type III domains
WO2018111976A1 (en) 2016-12-14 2018-06-21 Janssen Biotech, Inc. Pd-l1 binding fibronectin type iii domains
NZ754187A (en) 2016-12-14 2026-02-27 Janssen Biotech Inc Cd8a-binding fibronectin type iii domains
AU2017376950B2 (en) 2016-12-16 2024-02-22 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing TTR-associated diseases using transthyretin (TTR) iRNA compositions
IL267919B2 (en) 2017-01-09 2024-03-01 Aposense Ltd Compounds and methods for transmembrane transfer of molecules
BR112019014282A2 (pt) 2017-01-10 2020-03-03 Arrowhead Pharmaceuticals, Inc. Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
CN106986942B (zh) * 2017-02-28 2020-12-25 国药中生生物技术研究院有限公司 一种包含蝙蝠肝炎病毒核心蛋白的重组融合蛋白及其制备方法和应用
US11963974B2 (en) 2017-03-10 2024-04-23 National Center For Child Health And Development Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
WO2018181428A1 (ja) * 2017-03-29 2018-10-04 塩野義製薬株式会社 核酸医薬及び多分岐脂質の複合体
JP7155239B2 (ja) 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
WO2019193144A1 (en) 2018-04-05 2019-10-10 Silence Therapeutics Gmbh siRNAs WITH VINYLPHOSPHONATE AT THE 5' END OF THE ANTISENSE STRAND
EP3385272A1 (en) * 2017-04-05 2018-10-10 Silence Therapeutics GmbH Further novel oligonucleotide-ligand conjugates
WO2018185253A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
WO2018191278A2 (en) 2017-04-11 2018-10-18 Arbutus Biopharma Corporation Targeted compositions
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
IL269927B2 (en) 2017-04-18 2025-04-01 Alnylam Pharmaceuticals Inc Methods of treating patients with hepatitis B virus infection
US11324820B2 (en) 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
WO2018195355A1 (en) 2017-04-19 2018-10-25 Rxi Pharmaceuticals Corporation Topical delivery of nucleic acid compounds
WO2018200943A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
US11273222B2 (en) 2017-05-26 2022-03-15 National Cerebral And Cardiovascular Center Antisense nucleic acid targeting PCSK9
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
US10195286B2 (en) * 2017-07-04 2019-02-05 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
UY37803A (es) 2017-07-10 2019-02-28 Genzyme Corp Métodos y composiciones para tratar un evento de sangrado en un sujeto que padece hemofilia
CA3069451A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals, Inc. Methods for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
WO2019027999A1 (en) * 2017-07-31 2019-02-07 Ohio State Innovation Foundation BIOMIMETIC NANOMATERIALS AND USES THEREOF
WO2019027009A1 (ja) * 2017-08-02 2019-02-07 協和発酵キリン株式会社 核酸複合体
WO2019027015A1 (ja) * 2017-08-02 2019-02-07 協和発酵キリン株式会社 核酸複合体
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
EP3668834B1 (en) 2017-08-17 2024-10-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11197884B2 (en) 2017-08-18 2021-12-14 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
WO2019048645A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
CN110168093B (zh) * 2017-09-12 2023-08-15 中科蓝华(广州)生物医药技术有限公司 一种转染细胞内寄生虫的试剂盒及其应用
MX2020001912A (es) 2017-09-14 2020-03-24 Arrowhead Pharmaceuticals Inc Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso.
EP3682007A2 (en) * 2017-09-14 2020-07-22 Janssen BioPharma, Inc. Galnac derivatives
KR20250046363A (ko) 2017-09-19 2025-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
GB2610100B (en) 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases
AU2018360697A1 (en) 2017-11-01 2020-05-14 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
IL274611B2 (en) 2017-11-13 2024-10-01 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
JP7365052B2 (ja) 2017-12-01 2023-10-19 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
CN110945130B (zh) 2017-12-01 2024-04-09 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
WO2019105437A1 (zh) 2017-12-01 2019-06-06 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
CA3083968C (en) 2017-12-01 2024-04-23 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
WO2019105435A1 (zh) 2017-12-01 2019-06-06 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
AR113490A1 (es) 2017-12-12 2020-05-06 Amgen Inc CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS
JP2021508491A (ja) 2017-12-18 2021-03-11 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 高移動度グループボックス−１（ＨＭＧＢ１）ｉＲＮＡ組成物及びその使用方法
EP3727428A1 (en) 2017-12-20 2020-10-28 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
US11459564B2 (en) 2017-12-21 2022-10-04 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
CA3086485A1 (en) 2017-12-21 2019-06-27 Alnylam Pharmaceuticals, Inc. Chirally-enriched double-stranded rna agents
WO2019127004A1 (zh) 2017-12-26 2019-07-04 广州市锐博生物科技有限公司 修饰的寡核苷酸和可用于合成修饰的寡核苷酸的化合物
EP3732185B1 (en) * 2017-12-29 2025-02-26 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
IL275773B2 (en) 2018-01-01 2025-07-01 Aposense Ltd Compounds and methods for trans-membrane delivery of molecules
WO2019140102A1 (en) * 2018-01-10 2019-07-18 Translate Bio Ma, Inc. Compositions and methods for facilitating delivery of synthetic nucleic acids to cells
BR112020014425A2 (pt) 2018-01-15 2020-12-29 Ionis Pharmaceuticals, Inc. Moduladores de expressão de dnm2
WO2019147743A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
US12246031B2 (en) 2018-02-13 2025-03-11 Checkmate Pharmaceuticals, Inc. Compositions and methods for tumor immunotherapy
CN112041439A (zh) 2018-02-14 2020-12-04 深度基因组学公司 用于威尔森病的寡核苷酸疗法
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
CN111511915A (zh) 2018-03-09 2020-08-07 第一三共株式会社 糖原病Ia型治疗药
CN110305137A (zh) * 2018-03-20 2019-10-08 鲁南制药集团股份有限公司 一种培美曲塞二钠中间体及其制备方法
CN110305135B (zh) * 2018-03-20 2022-02-22 鲁南制药集团股份有限公司 一种培美曲塞二钠中间体及其制备方法
CN110305134B (zh) * 2018-03-20 2022-02-22 鲁南制药集团股份有限公司 一种培美曲塞二钠中间体及其制备方法
CN110305136A (zh) * 2018-03-20 2019-10-08 鲁南制药集团股份有限公司 一种培美曲塞二钠中间体及其制备方法
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
EP3550021A1 (en) * 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions for inhibiting expression of a target gene
WO2019193189A1 (en) * 2018-04-05 2019-10-10 Silence Therapeutics Gmbh siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS
KR102890791B1 (ko) 2018-04-09 2025-11-24 체크메이트 파마슈티칼스, 인크. 바이러스-유사 입자 내로의 올리고뉴클레오타이드의 포장
CA3094020A1 (en) 2018-04-11 2019-10-17 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
EP3775211B1 (en) 2018-04-12 2023-04-05 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2019217459A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
EP3790972A1 (en) 2018-05-08 2021-03-17 Regulus Therapeutics Inc. Galnac conjugated modified oligonucleotide as mir-122 inhibitor having hcv antiviral activity with reduced hyperbilirubinemia side-effect
SG11202010215TA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression
JP7438135B2 (ja) 2018-05-09 2024-02-26 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｆｘｉの発現を低下させるための化合物及び方法
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
IL279102B2 (en) * 2018-05-30 2024-10-01 Dtx Pharma Inc Lipid-modified nucleic acid compounds and methods
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
CN112638375A (zh) 2018-06-15 2021-04-09 旗舰创业创新五公司 通过后细胞信号传导因子的调节来增加免疫活性
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
KR102059815B1 (ko) 2018-07-09 2019-12-27 삼성전기주식회사 안테나 기판 및 이를 포함하는 안테나 모듈
EP3823725A4 (en) 2018-07-17 2023-05-10 Aronora, Inc. METHODS FOR SAFELY REDUCING THROMBOPOIETIN
TW202019441A (zh) 2018-07-20 2020-06-01 美商雷格勒斯治療公司 用於經口遞送寡核苷酸之方法
BR112021000308A2 (pt) 2018-07-25 2021-04-13 Ionis Pharmaceuticals, Inc. Compostos e métodos para redução da expressão de atxn2
US20210292768A1 (en) 2018-08-08 2021-09-23 Arcturus Therapeutics, Inc. Compositions and agents against nonalcoholic steatohepatitis
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
CA3106701A1 (en) 2018-08-13 2020-02-20 Alnylam Pharmaceuticals, Inc. Hepatitis b virus (hbv) dsrna agent compositions and methods of use thereof
US11987792B2 (en) 2018-08-16 2024-05-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
US11918600B2 (en) 2018-08-21 2024-03-05 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
CA3105385A1 (en) 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
TWI856973B (zh) 2018-09-19 2024-10-01 美商Ionis製藥公司 Pnpla3表現之調節劑
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
WO2020069055A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
EP3862024A4 (en) 2018-09-30 2022-08-17 Suzhou Ribo Life Science Co., Ltd. SIRNA CONJUGATE, METHOD FOR ITS PRODUCTION AND ITS USE
GB201816554D0 (en) 2018-10-10 2018-11-28 Centauri Therapeutics Ltd Novel compounds and therapeutic uses thereof
JP7543259B2 (ja) 2018-10-18 2024-09-02 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
BR112021008967A2 (pt) 2018-11-15 2021-08-17 Ionis Pharmaceuticals, Inc. moduladores da expressão de irf5
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
SG11202103794QA (en) 2018-11-21 2021-05-28 Ionis Pharmaceuticals Inc Compounds and methods for reducing prion expression
EP3884051A2 (en) 2018-11-23 2021-09-29 Sanofi Novel rna compositions and methods for inhibiting angptl8
CA3121410C (en) * 2018-11-30 2023-08-08 Kylonova (Xiamen) Biopharma Co., Ltd. Drug containing liver targeting specific ligand and thyroid hormone receptor agonist
CA3121449A1 (en) 2018-11-30 2020-06-04 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
WO2020117706A1 (en) 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity
EP3894554A1 (en) 2018-12-10 2021-10-20 Amgen Inc. Chemically-modified rnai constructs and uses thereof
CN113166761B (zh) 2018-12-10 2025-09-09 美国安进公司 用于抑制pnpla3表达的rnai构建体
US20220056455A1 (en) 2018-12-20 2022-02-24 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
EP3899024A4 (en) 2018-12-21 2023-05-31 Ionis Pharmaceuticals, Inc. Modulators of hsd17b13 expression
US12496347B2 (en) 2018-12-28 2025-12-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
CN111377985B (zh) * 2018-12-29 2023-11-10 苏州瑞博生物技术股份有限公司 化合物和缀合物及其制备方法和用途
MY202837A (en) 2019-01-11 2024-05-24 Acuitas Therapeutics Inc Lipids for lipid nanoparticle delivery of active agents
KR20210116509A (ko) 2019-01-16 2021-09-27 젠자임 코포레이션 Serpinc1 irna 조성물 및 그의 이용 방법
CN113330117B (zh) * 2019-01-18 2024-05-28 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
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
SG11202107399WA (en) 2019-01-31 2021-08-30 Ionis Pharmaceuticals Inc Modulators of yap1 expression
TW202540426A (zh) 2019-02-27 2025-10-16 美商Ionis製藥公司 Malat1表現之調節劑
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
FI3947684T3 (fi) 2019-03-29 2025-05-27 Ionis Pharmaceuticals Inc Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
WO2020227159A2 (en) 2019-05-03 2020-11-12 Flagship Pioneering Innovations V, Inc. Methods of modulating immune activity
JP2022532652A (ja) 2019-05-17 2022-07-15 アルニラム ファーマスーティカルズ インコーポレイテッド オリゴヌクレオチドの経口送達
JP7610268B2 (ja) 2019-05-22 2025-01-08 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
WO2020233680A1 (zh) 2019-05-22 2020-11-26 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
WO2020233651A1 (zh) 2019-05-22 2020-11-26 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
AU2020280438B2 (en) 2019-05-22 2025-03-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
US20220315929A1 (en) 2019-05-24 2022-10-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate, preparation method therefor and use thereof
CA3140233A1 (en) 2019-05-24 2020-12-03 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate, preparation method and use
EP3978609A4 (en) 2019-05-24 2024-02-07 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
TW202111124A (zh) 2019-05-30 2021-03-16 美商安進公司 用於抑制ｓｃａｐ表現之ｒｎａｉ構建體及其使用方法
MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).
AU2020301419B2 (en) 2019-06-25 2026-03-19 Amgen Inc. Purification methods for carbohydrate-linked oligonucleotides
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
EP3956450B1 (en) 2019-07-26 2025-08-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
WO2021030613A1 (en) 2019-08-13 2021-02-18 Amgen Inc. Rnai constructs for inhibiting slc30a8 expression and methods of use thereof
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
CN114585633A (zh) 2019-08-19 2022-06-03 米纳治疗有限公司 寡核苷酸缀合物组合物和使用方法
US20220290156A1 (en) 2019-08-27 2022-09-15 Sanofi Compositions and methods for inhibiting pcsk9
TW202122093A (zh) 2019-08-29 2021-06-16 大陸商蘇州瑞博生物技術股份有限公司 化合物、藥物綴合物、試劑盒及其用途
EP4025252A4 (en) 2019-09-03 2023-10-18 Arcturus Therapeutics, Inc. ASIALOGLYCOPROTEIN RECEPTOR-MEDIATED ADMINISTRATION OF THERAPEUTICALLY ACTIVE CONJUGATES
AU2020343255A1 (en) 2019-09-03 2022-03-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
WO2021049504A1 (ja) 2019-09-10 2021-03-18 第一三共株式会社 肝臓送達用ＧａｌＮＡｃ－オリゴヌクレオチドコンジュゲートおよび製造方法
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
WO2021072330A1 (en) 2019-10-09 2021-04-15 Silverback Therapeutics, Inc. Galnac-tgfbr1 inhibitor conjugates for the treatment of liver diseases
WO2021076543A1 (en) 2019-10-14 2021-04-22 Aro Biotherapeutics Company Epcam binding fibronectin type iii domains
EP4045062A1 (en) 2019-10-14 2022-08-24 Astrazeneca AB Modulators of pnpla3 expression
CN114786682B (zh) 2019-10-14 2024-07-16 Aro生物疗法公司 结合cd71的纤维粘连蛋白iii型结构域
WO2021076574A2 (en) 2019-10-14 2021-04-22 Aro Biotherapeutics Company Fn3 domain-sirna conjugates and uses thereof
WO2021076828A1 (en) 2019-10-18 2021-04-22 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
TW202134435A (zh) 2019-10-22 2021-09-16 美商阿尼拉製藥公司 補體成分ｃ３ ｉｒｎａ組成物及其使用方法
CN119499394A (zh) 2019-11-01 2025-02-25 阿尔尼拉姆医药品有限公司 亨廷顿（HTT）iRNA药剂组合物及其使用方法
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
CA3160329A1 (en) * 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
KR20220115946A (ko) 2019-11-13 2022-08-19 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐 (agt) 관련 장애를 치료하기 위한 방법 및 조성물
WO2021102356A1 (en) 2019-11-20 2021-05-27 Berg Llc Combination therapy of a coenzyme q10 compound and radiation therapy for treatment of glioma
WO2021102373A1 (en) 2019-11-22 2021-05-27 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
US20230078200A1 (en) 2019-12-09 2023-03-16 Amgen Inc. RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
CN115175685B (zh) 2019-12-09 2024-12-13 艾姆皮瑞克公司 用于治疗血管生成素样4(angptl4)相关疾病的寡核苷酸
EP4073251A1 (en) 2019-12-13 2022-10-19 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）ｉＲＮＡ組成物及其使用方法
CN111041025B (zh) 2019-12-17 2021-06-18 深圳市瑞吉生物科技有限公司 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法
JP2023509359A (ja) 2019-12-17 2023-03-08 フラグシップ パイオニアリング イノベーションズ ブイ，インコーポレーテッド 鉄依存性細胞分解の誘導物質との併用抗癌療法
EP4071163A4 (en) 2020-01-30 2024-03-20 Eisai R&D Management Co., Ltd. NUCLEIC ACID COMPLEX AND PHARMACEUTICAL COMPOSITION CONTAINING SAME
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
MX2022009763A (es) 2020-02-10 2022-09-09 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion del factor de crecimiento endotelial vascular a (vegf-a).
KR20220143106A (ko) 2020-02-18 2022-10-24 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 C3 (APOC3) iRNA 조성물 및 이의 사용 방법
CR20220485A (es) 2020-02-28 2022-11-10 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
KR20230011913A (ko) 2020-03-04 2023-01-25 버브 테라퓨틱스, 인크. 표적화된 rna 전달을 위한 조성물 및 방법
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
BR112022017822A2 (pt) 2020-03-06 2022-11-08 Alnylam Pharmaceuticals Inc Composições de irna de cetoexocinase (khk) e métodos de uso das mesmas
KR20220152270A (ko) 2020-03-06 2022-11-15 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴 (ttr)의 발현을 억제하기 위한 조성물 및 방법
EP4121534A1 (en) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
EP4126967A1 (en) 2020-03-23 2023-02-08 Amgen Inc. Monoclonal antibodies to chemically-modified nucleic acids and uses thereof
AU2021244559A1 (en) 2020-03-24 2022-11-17 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics
MX2022011805A (es) 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix.
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
WO2021202902A1 (en) 2020-04-01 2021-10-07 Alnylam Pharmaceuticals, Inc. ALPHA-2A ADRENERGIC RECEPTOR (ADRA2A) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
EP4133078A1 (en) 2020-04-06 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
CA3179678A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
EP4132478A1 (en) 2020-04-09 2023-02-15 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
AU2021260934A1 (en) 2020-04-21 2022-11-24 Flagship Pioneering, Inc. Bifunctional molecules and methods of using thereof
KR20230018377A (ko) 2020-04-27 2023-02-07 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 e (apoe) irna 제제 조성물 및 이의 사용 방법
CN113563414B (zh) * 2020-04-29 2022-08-12 泰比棣医药科技(石家庄)有限公司 一种组织靶向的蛋白靶向降解化合物及其用途
MX2022013606A (es) 2020-04-30 2023-01-16 Alnylam Pharmaceuticals Inc Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas.
MX2022013707A (es) 2020-05-01 2022-12-07 Ionis Pharmaceuticals Inc Compuestos y metodos para modular atxn1.
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021231673A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
CN113683651A (zh) * 2020-05-19 2021-11-23 上海京新生物医药有限公司 一种GalNAc中间体的制备方法
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
JP2023526533A (ja) 2020-05-22 2023-06-21 ウェイブ ライフ サイエンシズ リミテッド 二本鎖オリゴヌクレオチド組成物及びそれに関連する方法
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
MX2022015169A (es) 2020-06-01 2023-07-03 Amgen Inc Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos.
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
CN116075592A (zh) 2020-06-09 2023-05-05 阿尔尼拉姆医药品有限公司 用于沉默gpam(线粒体甘油-3-磷酸酰基转移酶1)表达的sirna组合物和方法
WO2021252557A1 (en) 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
TW202214856A (zh) 2020-06-18 2022-04-16 美商阿尼拉製藥公司 黃嘌呤脫氫酶（ＸＤＨ）ｉＲＮＡ組成物及其使用之方法
JP2023532339A (ja) 2020-06-29 2023-07-27 フラグシップ パイオニアリング イノベーションズ ブイ，インコーポレーテッド サノトランスミッションを促進するためにエンジニアリングされたウイルス及び癌の処置におけるそれらの使用
BR112022024206A2 (pt) 2020-06-29 2023-01-03 Ionis Pharmaceuticals Inc Compostos e métodos para modular plp1
CN111744019B (zh) * 2020-07-01 2023-08-04 深圳瑞吉生物科技有限公司 基于甘露糖的mRNA靶向递送系统及其应用
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
WO2022016070A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
AU2021315992A1 (en) 2020-07-28 2023-02-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
IL300258A (en) 2020-08-07 2023-03-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating SCN2A
PE20230993A1 (es) 2020-08-13 2023-06-23 Amgen Inc Construcciones de iarn y metodos para inhibir la expresion de marc1
CN116583303A (zh) * 2020-08-21 2023-08-11 瑞阳公司 雷公藤甲素缀合物和其用途
AU2021342158A1 (en) * 2020-09-11 2023-04-13 Arrowhead Pharmaceuticals, Inc. Lipid conjugates for the delivery of therapeutic agents
CZ310613B6 (cs) 2020-09-23 2026-01-28 Ústav organické chemie a biochemie AV ČR, v. v. i. Lipidoidy pro transfekci nukleových kyselin a jejich použití
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
JP2023544413A (ja) 2020-10-05 2023-10-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｇタンパク質共役受容体７５（ＧＰＲ７５）ｉＲＮＡ組成物およびその使用方法
US20240035029A1 (en) 2020-10-16 2024-02-01 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
JP2023546103A (ja) 2020-10-16 2023-11-01 サノフイ Ａｎｇｐｔｌ３を阻害するための新規のｒｎａ組成物および方法
EP4232581A1 (en) 2020-10-21 2023-08-30 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
WO2022098841A1 (en) 2020-11-05 2022-05-12 Amgen Inc. METHODS FOR TREATING ATHEROSCLEROTIC CARDIOVASCULAR DISEASE WITH LPA-TARGETED RNAi CONSTRUCTS
KR20230107625A (ko) 2020-11-13 2023-07-17 알닐람 파마슈티칼스 인코포레이티드 응고 인자 V(F5) iRNA 조성물 및 이의 사용 방법
EP4136092B1 (en) 2020-11-18 2024-07-31 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
WO2022119873A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
JP2023554346A (ja) 2020-12-18 2023-12-27 アイオーニス ファーマシューティカルズ， インコーポレーテッド 第ｘｉｉ因子を調節するための化合物及び方法
CN117015386A (zh) 2020-12-21 2023-11-07 旗舰创业创新五公司 细胞周转因子用于增加组织再生的用途
IL303886A (en) 2020-12-23 2023-08-01 Flagship Pioneering Inc Compositions of modified trems and uses thereof
EP4273245A4 (en) 2020-12-29 2024-12-11 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND SIRNA CONJUGATE WITH THE NUCLEIC ACID, MANUFACTURING PROCESS THEREOF AND USE THEREOF
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
WO2022172083A2 (en) * 2021-01-21 2022-08-18 Sirnaomics, Inc. Targeted nucleic acid therapy for hepatitis b
US20230390411A1 (en) * 2021-01-28 2023-12-07 Nanjing Chempion Biotechnology Co., Ltd. Conjugate and use thereof
CA3204340A1 (en) * 2021-01-30 2022-08-04 Ahmad Ali MORTAZAVI Conjugated oligonucleotide compounds, methods of making and uses thereof
EP4175964A1 (en) * 2021-01-30 2023-05-10 E-Therapeutics plc Conjugated oligonucleotide compounds, methods of making and uses thereof
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
CN117222739A (zh) 2021-02-25 2023-12-12 阿尔尼拉姆医药品有限公司 朊病毒蛋白(prnp)irna组合物和其使用方法
TW202302847A (zh) 2021-02-26 2023-01-16 美商艾拉倫製藥股份有限公司 己酮糖激酶（ＫＨＫ）ｉＲＮＡ組成物及其使用方法
IL305442A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
CA3207897A1 (en) 2021-03-19 2022-09-22 Ralf Eisenhuth Improved oligonucleotide synthesis
EP4316495A4 (en) * 2021-03-25 2025-12-31 Aptacure Therapeutics Ltd CONJUGATE OF NUCLEIC ACID MOLECULE HAVING A PROLONGED IN VIVO HALF-LIFE
EP4314292A1 (en) 2021-03-26 2024-02-07 MiNA Therapeutics Limited Tmem173 sarna compositions and methods of use
WO2022212231A2 (en) 2021-03-29 2022-10-06 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
JP2024512669A (ja) 2021-03-31 2024-03-19 フラグシップ パイオニアリング イノベーションズ ブイ，インコーポレーテッド タノトランスミッションポリペプチド及び癌の処置におけるそれらの使用
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
WO2022216841A1 (en) 2021-04-06 2022-10-13 Berg Llc Protein markers for estrogen receptor (er)-positive luminal a(la)-like and luminal b1 (lb1)-like breast cancer
EP4320443A1 (en) 2021-04-06 2024-02-14 BPGbio, Inc. Protein markers for the prognosis of breast cancer progression
EP4320441A1 (en) 2021-04-06 2024-02-14 BPGbio, Inc. Protein markers for estrogen receptor (er)-positive-like and estrogen receptor (er)-negative-like breast cancer
CN117304473A (zh) * 2021-04-08 2023-12-29 厦门赛诺邦格生物科技股份有限公司 一种聚乙二醇化脂质及其修饰的脂质体、含该脂质体的药物组合物及其制剂和应用
CN115197080A (zh) * 2021-04-08 2022-10-18 厦门赛诺邦格生物科技股份有限公司 一种聚乙二醇化脂质及其修饰的脂质体、含该脂质体的药物组合物及其制剂和应用
US20250282813A1 (en) 2021-04-09 2025-09-11 Bachem Holding Ag Pseudo solid phase protecting group and methods for the synthesis of oligonucleotides and oligonucleotide conjugates
CA3214552A1 (en) 2021-04-14 2022-10-20 Russell C. Addis Cd71 binding fibronectin type iii domains
BR112023021318A2 (pt) 2021-04-14 2023-12-19 Aro Biotherapeutics Company Conjugados de domínio fn3-sirna e usos dos mesmos
AU2022262794A1 (en) 2021-04-23 2023-11-02 Beth Israel Deaconess Medical Center, Inc. Glycan modified nucleic acids, methods of preparation, and therapeutic uses
MX2023012586A (es) 2021-04-26 2023-10-31 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de serina 6, proteasa transmembranaria (tmprss6) y sus metodos de uso.
BR112023022439A2 (pt) 2021-04-26 2023-12-26 Celanese Eva Performance Polymers Llc Dispositivo implantável para liberação sustentada de um composto de fármaco macromolecular
CN117881786A (zh) 2021-04-27 2024-04-12 世代生物公司 表达治疗性抗体的非病毒dna载体及其用途
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
JP2024519293A (ja) 2021-04-29 2024-05-10 アルナイラム ファーマシューティカルズ， インコーポレイテッド シグナル伝達兼転写活性化因子６（ＳＴＡＴ６）ｉＲＮＡ組成物およびその使用方法
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
EP4341405A1 (en) 2021-05-20 2024-03-27 Korro Bio, Inc. Methods and compositions for adar-mediated editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
CN117561334A (zh) 2021-06-04 2024-02-13 阿尔尼拉姆医药品有限公司 人染色体9开放阅读框72（C9ORF72）iRNA药剂组合物和其使用方法
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
KR20240023602A (ko) 2021-06-18 2024-02-22 아이오니스 파마수티컬즈, 인코포레이티드 Ifnar1 발현을 감소시키기 위한 화합물 및 방법
CN115572241B (zh) * 2021-06-21 2024-08-30 润佳（苏州）医药科技有限公司 双价化合物、偶联物及其用途
CA3224904A1 (en) 2021-06-21 2022-12-29 Shanghai Junshi Biosciences Co., Ltd. Sirna inhibiting angptl3 gene expression and use thereof
AU2022299169A1 (en) 2021-06-23 2024-02-08 Beth Israel Deaconess Medical Center, Inc. Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20240316104A1 (en) 2021-06-29 2024-09-26 Flagship Pioneering Innovations V, Inc. Immune cells engineered to promote thanotransmission and uses thereof
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
IL309296A (en) 2021-06-30 2024-02-01 Alnylam Pharmaceuticals Inc Methods and compositions for the treatment of angiotensinogen-related disorder (AGT-)
US20240285547A1 (en) 2021-07-08 2024-08-29 Nippon Shinyaku Co., Ltd. Nephrotoxicity reducing agent
US20240285770A1 (en) 2021-07-08 2024-08-29 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
CN117580953B (zh) 2021-07-16 2025-07-18 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
US20240309378A1 (en) 2021-07-16 2024-09-19 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate containing double-stranded oligonucleotide, and preparation methods and uses
WO2023003805A1 (en) 2021-07-19 2023-01-26 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder
CZ310443B6 (cs) 2021-07-19 2025-06-25 Ústav organické chemie a biochemie AV ČR, v. v. i. Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití
TW202421169A (zh) 2021-07-21 2024-06-01 美商艾拉倫製藥股份有限公司 代謝疾患相關的標靶基因ｉＲＮＡ組成物及其使用方法
CA3226878A1 (en) 2021-07-23 2023-01-26 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
JP2024529437A (ja) 2021-07-29 2024-08-06 アルナイラム ファーマシューティカルズ， インコーポレイテッド ３－ヒドロキシ－３－メチルグリタル－ＣＯＡレダクターゼ（ＨＭＧＣＲ）ｉＲＮＡ組成物およびその使用方法
CN115920065A (zh) * 2021-08-02 2023-04-07 南方科技大学 一种可靶向去唾液酸糖蛋白受体的配体及其缀合物与用途
JP2024530647A (ja) * 2021-08-03 2024-08-23 ヴァーヴ・セラピューティクス，インコーポレーテッド 標的ｒｎａ送達のための組成物および方法
CA3227852A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
JP2024528270A (ja) * 2021-08-04 2024-07-26 ヘパジーン セラピューティクス（エイチケー）リミティド 治療活性剤の送達のためのリガンド共役体
IL310295A (en) 2021-08-04 2024-03-01 Alnylam Pharmaceuticals Inc iRNA compositions and methods for silencing angiotensinogen (AGT)
AU2022324471A1 (en) 2021-08-05 2024-02-15 Sanegene Bio Usa Inc. 1'-alkyl modified ribose derivatives and methods of use
CN117858883A (zh) 2021-08-09 2024-04-09 莱尔纳生物制药私人有限公司 GalNAc单体和GalNAc寡核苷酸缀合物
KR20240045300A (ko) 2021-08-13 2024-04-05 알닐람 파마슈티칼스 인코포레이티드 인자 XII (F12) iRNA 조성물 및 이의 사용 방법
WO2023034837A2 (en) 2021-08-31 2023-03-09 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
WO2023039594A1 (en) * 2021-09-13 2023-03-16 Ganna Bio, Inc. Glycan conjugate compositions and methods
JP2024535850A (ja) 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド 補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
EP4405368A1 (en) 2021-09-22 2024-07-31 Sanegene Bio USA Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
MX2024004011A (es) 2021-10-01 2024-07-01 Adarx Pharmaceuticals Inc Composiciones moduladoras de precalicreína y métodos de uso de estas.
CN118076621A (zh) 2021-10-05 2024-05-24 美国圣因生物股份有限公司 多羟基化的环戊烷衍生物及使用方法
CN118556125A (zh) 2021-10-05 2024-08-27 美国安进公司 用于增强寡核苷酸化合物的基因沉默活性的组合物和方法
WO2023064891A1 (en) 2021-10-14 2023-04-20 Hemoshear Therapeutics, Inc. Compositions and methods of treating diseases associated with bile acid transporter
MX2024004437A (es) 2021-10-15 2024-07-01 Alnylam Pharmaceuticals Inc Composiciones de inhibidor de la angiotensina-receptor neprilisina (arni) de administracion extrahepatica y metodos de uso de las mismas.
CA3235262A1 (en) 2021-10-22 2023-04-27 Amgen Inc. Rnai constructs for inhibiting gpam expression and methods of use thereof
US20250352667A1 (en) 2021-10-22 2025-11-20 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
TW202333749A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 補體因子ｂ（ｃｆｂ）ｉｒｎａ組成物及其使用方法
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
US20250066790A1 (en) 2021-11-10 2025-02-27 University Of Rochester Antisense oligonucleotides for modifying protein expression
US20250019702A1 (en) 2021-11-10 2025-01-16 University Of Rochester Gata4-targeted therapeutics for treatment of cardiac hypertrophy
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
EP4471140A1 (en) 2022-01-30 2024-12-04 Rona Bioscience, Limited Targeting ligand containing n-acetylgalactosamine
IL314850A (en) 2022-02-22 2024-10-01 Sanegene Bio Usa Inc Ribonucleotide derivatives substituted at the 5' position in the carboxyl group and methods of use
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
CN120129745A (zh) 2022-03-10 2025-06-10 日本新药株式会社 抗病毒反义低聚物
WO2023177655A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
EP4493570A2 (en) 2022-03-16 2025-01-22 Empirico Inc. Galnac compositions for improving sirna bioavailability
JPWO2023176863A1 (https=) 2022-03-16 2023-09-21
KR20240161967A (ko) 2022-03-16 2024-11-13 다이이찌 산쿄 가부시키가이샤 트랜스페린 수용체 2 의 발현을 억제하는 siRNA
KR20240163743A (ko) 2022-03-28 2024-11-19 엠피리코 인크. 변형된 올리고뉴클레오티드
CN119213006A (zh) * 2022-04-26 2024-12-27 康涅狄格大学 用于增强肝脏和肾脏特异性靶向的遗传序列-糖缀合物
US20240002857A1 (en) 2022-05-09 2024-01-04 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
EP4522742A2 (en) 2022-05-13 2025-03-19 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
US20230372508A1 (en) 2022-05-19 2023-11-23 Olix Us, Inc. Linkers coupling functional ligands to macromolecules
JP2025520520A (ja) * 2022-06-14 2025-07-03 大睿生物 ＰＣＳＫ９遺伝子活性を調節するｓｉＲＮＡ分子
WO2023241587A1 (zh) 2022-06-14 2023-12-21 大睿生物医药科技(上海)有限公司 环膦酸酯修饰的核苷酸
US20250179492A1 (en) 2022-06-22 2025-06-05 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
JP2025520796A (ja) 2022-06-27 2025-07-03 大睿生物 アポリポタンパク質Ｃ３発現を阻害するｓｉＲＮＡ
CN119487192A (zh) * 2022-06-27 2025-02-18 中美瑞康核酸技术(南通)研究院有限公司 小激活核酸分子及其在治疗遗传性血管性水肿中的用途
IL317824A (en) 2022-07-11 2025-02-01 Sanegene Bio Usa Inc Ribose derivatives modified at the '2' position and methods of use
IL317825A (en) 2022-07-25 2025-02-01 Amgen Inc RNA interference constructs and methods for inhibiting fam13a expression
KR20250046303A (ko) 2022-08-05 2025-04-02 사네진 바이오 유에스에이 인크. 안지오텐시노겐 (agt)을 표적화하는 이중 가닥 rna 및 그의 사용 방법
JP2025527531A (ja) 2022-08-18 2025-08-22 アルナイラム ファーマシューティカルズ， インコーポレイテッド ユニバーサル非標的ｓｉｒｎａ組成物およびその使用方法
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
EP4581143A1 (en) 2022-08-29 2025-07-09 University of Rochester Antisense oligonucleotide-based anti-fibrotic therapeutics
JP2025532593A (ja) 2022-09-15 2025-10-01 リジェネロン・ファーマシューティカルズ・インコーポレイテッド １７ｂ－ヒドロキシステロイドデヒドロゲナーゼ１３型（ｈｓｄ１７ｂ１３）ｉｒｎａ組成物およびその使用方法
US20260001902A1 (en) 2022-09-19 2026-01-01 Bachem Holding Ag Improved oligonucleotide synthesis
JP2025532127A (ja) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｍｅｃｐ２発現を低減する化合物及び方法
JP2025533501A (ja) 2022-09-28 2025-10-07 サレプタ セラピューティクス， インコーポレイテッド ポンペ病マウスモデルの生成、特徴決定および使用方法
JP2025532985A (ja) 2022-09-30 2025-10-03 アルナイラム ファーマシューティカルズ， インコーポレイテッド 修飾二本鎖ｒｎａ剤
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
CN120265773A (zh) 2022-10-14 2025-07-04 美国圣因生物股份有限公司 靶向c3的小干扰rna及其用途
WO2024083746A1 (en) 2022-10-17 2024-04-25 Bachem Holding Ag Method and composition for oligonucleotide synthesis
CN120225676A (zh) 2022-12-02 2025-06-27 上海舶望制药有限公司 双环脱碱基核酸类似物以及由它们制备的寡聚化合物
EP4630061A1 (en) * 2022-12-08 2025-10-15 Nanovation Therapeutics Inc. Lipid nanoparticles comprising elevated neutral lipid and a targeting moiety for targeted delivery of nucleic acid
WO2024129710A1 (en) * 2022-12-12 2024-06-20 University Of Massachusetts Carbohydrate conjugates for the delivery of therapeutic oligonucleotides
AR131389A1 (es) 2022-12-16 2025-03-12 Amgen Inc CONSTRUCTOS DE ARNi PARA INHIBIR LA EXPRESIÓN DE TTR Y MÉTODOS DE USO DE LOS MISMOS
AU2023408479A1 (en) 2022-12-19 2025-05-01 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar)
EP4638743A2 (en) 2022-12-19 2025-10-29 Sanegene Bio USA Inc. Small interfering rna targeting cfb and uses thereof
EP4592432A3 (en) 2022-12-19 2025-10-29 Arnatar Therapeutics Arnatar compounds and methods for enhanced cellular uptake
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
CN117720590A (zh) * 2022-12-30 2024-03-19 天津药明康德新药开发有限公司 一种改进的n-乙酰半乳糖胺中间体的制备方法
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
US20240254485A1 (en) 2023-01-13 2024-08-01 Hemoshear Therapeutics, Inc. Sirnas targeting slc10a1 transcripts, compositions and uses thereof
EP4662311A2 (en) 2023-02-09 2025-12-17 Alnylam Pharmaceuticals, Inc. Reversir molecules and methods of use thereof
US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
WO2024186673A2 (en) 2023-03-03 2024-09-12 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
WO2024188174A1 (zh) 2023-03-10 2024-09-19 苏州瑞博生物技术股份有限公司 一种药物组合物及其用途
WO2024206230A1 (en) * 2023-03-24 2024-10-03 Trustees Of Tufts College Tissue targeting lipids and lipid nanoparticles
AU2024259356A1 (en) * 2023-04-19 2025-11-06 Prime Medicine, Inc. Lipid nanoparticle (lnp) delivery systems and formulations
WO2024220930A2 (en) 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
KR20260005960A (ko) 2023-05-03 2026-01-12 사네진 바이오 유에스에이 인크. 전신, 중추 신경계, 말초 신경계, 및 안구 전달을 위한 지질 기반 접합체
AU2024269205A1 (en) 2023-05-11 2025-12-11 Keymed Biosciences (Us) Inc. Dsrna molecules for regulating masp2 gene activity
AU2024269116A1 (en) 2023-05-11 2025-11-20 Sunshine Lake Pharma Co., Ltd. Novel targeting compound and use thereof, and nucleic acid conjugate and use thereof
CN121398851A (zh) 2023-05-12 2026-01-23 阿达尔克斯制药有限公司 Nmda配体缀合的化合物及其用途
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
CN118993970B (zh) * 2023-05-17 2025-12-23 苏州富士莱医药股份有限公司 一种l96侧链化合物的制备方法
WO2024240212A1 (zh) 2023-05-24 2024-11-28 云合智药（苏州）生物科技有限公司 用于抑制angptl3表达的rnai剂及其应用
IL324720A (en) 2023-05-26 2026-01-01 Adarx Pharmaceuticals Inc SOD1 modulators and methods of using them
WO2024249889A1 (en) * 2023-05-31 2024-12-05 University Of Connecticut Genetic sequence/small molecule-carbohydrate conjugates for enhanced lung-specific targeting
EP4562158A1 (en) 2023-06-13 2025-06-04 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar) for angiotensinogen
CN121712891A (zh) 2023-06-20 2026-03-20 阿达尔克斯制药有限公司 Lrrk2调节组合物及其使用方法
CN121866332A (zh) 2023-06-21 2026-04-14 美国圣因生物股份有限公司 靶向前蛋白转化酶枯草杆菌蛋白酶kexin 9（pcsk9）的双链rna及其使用方法
US12508320B2 (en) 2023-06-29 2025-12-30 SeeCure Taiwan Co., Ltd. Drug conjugate, pharmaceutical composition and method of treating hepatitis
WO2025002208A1 (zh) * 2023-06-30 2025-01-02 苏州时安生物技术有限公司 一种核酸缀合物、制备方法及用途
CN121399258A (zh) 2023-07-06 2026-01-23 大睿生物医药科技(上海)有限公司 调控AGT表达的dsRNA分子
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025015338A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
TW202517784A (zh) 2023-07-14 2025-05-01 大陸商云合智藥（蘇州）生物科技有限公司 用於抑制己酮糖激酶表達的ＲＮＡｉ劑及其應用
GB202311324D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
GB202311334D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
US20250051771A1 (en) 2023-07-24 2025-02-13 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
US20250064939A1 (en) 2023-07-24 2025-02-27 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
AU2024321710A1 (en) 2023-08-04 2026-03-05 Chia Tai Tianqing Pharmaceutical Group Co., Ltd. Double-stranded ribonucleic acid targeting pd-l1
KR20260049597A (ko) 2023-08-04 2026-04-14 알닐람 파마슈티칼스 인코포레이티드 Ctnnb1-관련 질환을 치료하기 위한 방법 및 조성물
AU2024321098A1 (en) 2023-08-09 2026-03-19 Beijing Foyou Pharma Co., Ltd Sirna for inhibiting dgat2 gene expression, sirna conjugate or prodrug and pharmaceutical composition thereof, and use thereof
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025040139A1 (zh) 2023-08-23 2025-02-27 云合智药（苏州）生物科技有限公司 用于抑制二酰甘油-O-酰基转移酶2表达的RNAi剂及其应用
CN121729496A (zh) 2023-08-31 2026-03-24 正大天晴药业集团股份有限公司 靶向凝血因子xi的双链核糖核酸
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
GB202314724D0 (en) 2023-09-26 2023-11-08 Astrazeneca Ab compounds and methods for reducing psd3 expression
WO2025072672A2 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
WO2025072699A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Aminoglycosides for delivery of agents to the kidney
WO2025072713A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Polymyxins for delivery of agents to the kidney
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
AU2024360411A1 (en) 2023-10-10 2026-02-26 Avanti Polar Lipids, Llc Liver asialoglycoprotein receptor targeted lipid and uses thereof
EP4695265A1 (en) 2023-10-16 2026-02-18 Bachem Holding AG Method and composition for oligonucleotide synthesis
WO2025085732A1 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Humanized dnm2 mouse model generation, characterization and methods of use
WO2025090796A1 (en) * 2023-10-24 2025-05-01 University Of Connecticut Carbohydrate ligands and uses thereof
CN121909203A (zh) 2023-10-31 2026-04-21 科罗生物公司 包含氨基磷酸酯核苷酸间键的寡核苷酸
WO2025097126A1 (en) * 2023-11-03 2025-05-08 Vionelix Pharmaceuticals, Inc. Mutation-resistant anti-viral compositions
WO2025077949A1 (zh) * 2023-11-06 2025-04-17 杭州天龙药业有限公司 提升双链寡核苷酸靶基因抑制效果的修饰模板及其组合与应用
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
TW202546227A (zh) 2024-01-18 2025-12-01 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療賀勒氏症候群（ｈｕｒｌｅｒ ｓｙｎｄｒｏｍｅ）的反義寡核苷酸
WO2025157273A1 (en) * 2024-01-25 2025-07-31 Shanghai Rona Therapeutics Co., Ltd. Two or more nucleic acid molecules connected by a linker
WO2025165891A1 (en) 2024-01-29 2025-08-07 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof
EP4671372A3 (en) 2024-01-29 2026-03-11 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof
WO2025193807A1 (en) 2024-03-12 2025-09-18 Sarepta Therapeutics, Inc. Humanized umod mouse model generation, characterization and methods of use
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025224036A1 (en) 2024-04-22 2025-10-30 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2025260068A1 (en) 2024-06-14 2025-12-18 Tune Therapeutics, Inc. Lipid nanoparticle formulation for delivery of nucleic acids to cells
WO2025260042A1 (en) 2024-06-14 2025-12-18 Amgen Inc. Rnai constructs and methods for inhibiting cnr1 expression
WO2026006436A1 (en) 2024-06-25 2026-01-02 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of tar dna binding protein 43 kda (tdp43)
WO2026006275A2 (en) 2024-06-26 2026-01-02 Amgen Inc. Rnai constructs and methods for inhibiting expression of inhbe
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
WO2026017140A2 (en) 2024-07-18 2026-01-22 Sanegene Bio Usa Inc. Small interfering rna targeting fxi and uses thereof
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use
WO2026041067A2 (en) 2024-08-20 2026-02-26 Sanegene Bio Usa Inc. Small interfering rna targeting myostatin (mstn) and uses thereof
WO2026044153A1 (en) 2024-08-21 2026-02-26 Bpgbio, Inc. Use of biomarkers in the diagnosis of pancreatic cancer
WO2026050243A1 (en) 2024-08-26 2026-03-05 Korro Bio, Inc. Galnac conjugated oligonucleotides for rna editing
WO2026046411A1 (en) 2024-09-02 2026-03-05 Sanegene Bio Usa Inc. Small interfering rna targeting plasminogen (plg) and uses thereof

Family Cites Families (134)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
BE795866A (fr) 1972-02-29 1973-08-23 Basf Ag Procede de preparation de chlorure de choline solide a ecoulement libre
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
CH624011A5 (https=) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
DE3013839A1 (de) 1979-04-13 1980-10-30 Freunt Ind Co Ltd Verfahren zur herstellung einer aktivierten pharmazeutischen zusammensetzung
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
JPS5921613A (ja) 1982-07-28 1984-02-03 Takeda Chem Ind Ltd 直腸投与製剤
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4486435A (en) 1983-05-16 1984-12-04 Basf Wyandotte Corporation Spray-dried vitamin powders using hydrophobic silica
JPH0818982B2 (ja) 1983-11-09 1996-02-28 昭和電工株式会社 乳量の増加方法
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
JP3022967B2 (ja) 1985-03-15 2000-03-21 アンチバイラルズ インコーポレイテッド 立体規則性ポリヌクレオチド結合ポリマー
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
FR2594693B1 (fr) 1986-02-24 1990-01-05 Farah Nabil Nouveaux procedes de preparation a partir d'emulsions seches de formes orales solides a liberation modifiee et retardee de leur principes actifs
EP0243686B1 (en) * 1986-03-27 1992-07-15 Sumitomo Pharmaceuticals Company, Limited Beta-lactam compounds, and their production
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
JPH081881B2 (ja) 1987-03-31 1996-01-10 日立エーアイシー株式会社 電気二重層コンデンサ
SE8701479L (sv) 1987-04-09 1988-10-10 Carbomatrix Ab Metod foer inneslutning av biologiskt verksamma preparat samt anvaendning daerav
JPH081884B2 (ja) 1987-04-10 1996-01-10 松下電子工業株式会社 レジストパタ−ンの形成方法
DE3881755T2 (de) * 1987-05-01 1993-10-21 Fujisawa Pharmaceutical Co Pyrrolidin-Derivate.
JPH081883B2 (ja) 1987-05-12 1996-01-10 松下電器産業株式会社 半導体プロセスフロ−作成システム
CH672048A5 (https=) 1987-09-16 1989-10-31 Nestle Sa
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
AU637800B2 (en) 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5000888A (en) 1990-05-23 1991-03-19 Basf Corporation Process for spray drying riboflavin to produce a granulate product having low binder content
JPH0436233A (ja) 1990-05-29 1992-02-06 Biomaterial Universe Kk 生理活性物質含有生体内分解吸収性の徐放性製剤
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
BR9106702A (pt) 1990-07-27 1993-06-08 Isis Pharmaceuticals Inc Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo
US5223618A (en) 1990-08-13 1993-06-29 Isis Pharmaceuticals, Inc. 4'-desmethyl nucleoside analog compounds
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー．ファウ． 修飾リボザイム
US5110455A (en) 1990-12-13 1992-05-05 Cyprus Minerals Company Method for achieving enhanced copper flotation concentrate grade by oxidation and flotation
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
JPH07508410A (ja) 1992-06-18 1995-09-21 ジェンファーム　インターナショナル　インコーポレイテッド 酵母人工染色体を有するトランスジェニック非ヒト動物の製造方法
WO1994002595A1 (en) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304620D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
JPH07101882A (ja) 1993-09-30 1995-04-18 Sanei Gen F F I Inc 水溶性ヘミセルロースを含有する製剤
JPH07101881A (ja) 1993-09-30 1995-04-18 Sanei Gen F F I Inc 水溶性ヘミセルロースを含有する製剤
JP3342550B2 (ja) 1993-10-01 2002-11-11 三栄源エフ・エフ・アイ株式会社 水溶性ヘミセルロースを含有する製剤
JPH07101884A (ja) 1993-10-01 1995-04-18 Sanei Gen F F I Inc 水溶性ヘミセルロースを含有する製剤
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5539083A (en) 1994-02-23 1996-07-23 Isis Pharmaceuticals, Inc. Peptide nucleic acid combinatorial libraries and improved methods of synthesis
JPH07242568A (ja) 1994-03-04 1995-09-19 Eisai Co Ltd 苦味隠蔽製剤
US5534499A (en) 1994-05-19 1996-07-09 The University Of British Columbia Lipophilic drug derivatives for use in liposomes
JPH086766A (ja) 1994-06-23 1996-01-12 Matsushita Electric Ind Co Ltd 正弦余弦演算装置
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
JPH11508231A (ja) 1995-05-26 1999-07-21 ソマティックス セラピー コーポレイション 安定な脂質／核酸複合体を含む送達ビヒクル
ATE285477T1 (de) 1995-06-07 2005-01-15 Inex Pharmaceutical Corp Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
AU1039397A (en) * 1995-11-22 1997-06-27 Johns Hopkins University, The Ligands to enhance cellular uptake of biomolecules
DE69729958T2 (de) * 1996-05-16 2005-07-28 Nissin Food Products Co., Ltd Verbindungen mit antiviraler aktivität
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
JP4036233B2 (ja) 1996-08-09 2008-01-23 ヤマハ株式会社 楽音発生装置および楽音発生方法、並びに該方法に係るプログラムを記憶した記憶媒体
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US6034135A (en) 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
IL123998A (en) * 1998-04-08 2004-09-27 Galmed Int Ltd Conjugates of bile salts and pharmaceutical preparations containing them
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
EP2314700A1 (en) 1999-01-28 2011-04-27 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
US7601494B2 (en) * 1999-03-17 2009-10-13 The University Of North Carolina At Chapel Hill Method of screening candidate compounds for susceptibility to biliary excretion
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
US7491805B2 (en) * 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) * 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
US6906182B2 (en) * 2000-12-01 2005-06-14 Cell Works Therapeutics, Inc. Conjugates of glycosylated/galactosylated peptide, bifunctional linker, and nucleotidic monomers/polymers, and related compositions and method of use
US20040259247A1 (en) * 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
EP1389209B1 (en) 2001-04-24 2009-04-08 Purdue Research Foundation Folate mimetics and folate-receptor binding conjugates thereof
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US7109165B2 (en) * 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
EP1572067A4 (en) 2001-05-18 2009-05-13 Sirna Therapeutics Inc CONJUGATES AND COMPOSITIONS FOR CELLULAR RELEASE
WO2003008628A2 (en) * 2001-07-20 2003-01-30 Ribozyme Pharmacuticals, Inc. Enzymatic nucleic acid peptide conjugates
EP1543019A2 (en) 2002-09-11 2005-06-22 Santaris Pharma A/S Modified pna molecules
WO2004080406A2 (en) 2003-03-07 2004-09-23 Alnylam Pharmaceuticals Therapeutic compositions
AU2004227414A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals iRNA conjugates
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
WO2005014655A2 (en) 2003-08-08 2005-02-17 Fresenius Kabi Deutschland Gmbh Conjugates of hydroxyalkyl starch and a protein
NZ592917A (en) * 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
AU2005325262B2 (en) * 2004-04-27 2011-08-11 Alnylam Pharmaceuticals, Inc. Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
WO2005116226A2 (en) * 2004-05-24 2005-12-08 Midatech Ltd Nanoparticles comprising rna ligands
EP1791567B1 (en) * 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
US20060148740A1 (en) * 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
WO2009082607A2 (en) * 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
DE102009039097B3 (de) 2009-08-27 2010-11-25 Siemens Aktiengesellschaft Verfahren zum Übertragen von Daten in einem Sensornetzwerk, Sensorknoten und Zentral-Rechner
WO2012005769A1 (en) 2010-07-09 2012-01-12 Telecommunication Systems, Inc. Location privacy selector
US10833934B2 (en) 2015-06-30 2020-11-10 British Telecommunications Public Limited Company Energy management in a network
KR102146393B1 (ko) 2016-08-30 2020-08-20 삼성에스디아이 주식회사 리튬 이차 전지용 분리막, 이의 제조 방법, 및 이를 포함하는 리튬 이차 전지
FR3067929B1 (fr) 2017-06-23 2019-11-22 Produits Dentaires Pierre Rolland Adhesif dentaire
DE102018125079B4 (de) 2018-10-10 2023-12-28 Schaeffler Technologies AG & Co. KG Spannungswellengetriebe und Übertragungselement hierfür sowie Roboterarm und Verfahren zum Messen eines Drehmomentes
EP3868181B1 (en) 2018-10-18 2022-06-08 Signify Holding B.V. Determining light settings and/or daylight blocker settings based on data signal quality
MX2021009290A (es) 2019-02-04 2021-10-13 Detnet South Africa Pty Ltd Bomba de impulsion.
CN111845757A (zh) 2019-04-30 2020-10-30 通用汽车环球科技运作有限责任公司 分心驾驶消除系统
DE112019007355B4 (de) 2019-05-23 2025-07-31 Mitsubishi Electric Corporation Numerische steuervorrichtung
CN110649043B (zh) 2019-09-30 2021-11-19 厦门天马微电子有限公司 阵列基板、显示面板、显示装置及阵列基板的制备方法
KR102318555B1 (ko) 2020-03-19 2021-10-29 한국과학기술연구원 광소자용 역나노콘과 그 제조방법

Also Published As

Publication number Publication date
US8106022B2 (en) 2012-01-31
JP2014139232A (ja) 2014-07-31
US20090239814A1 (en) 2009-09-24
JP2022190116A (ja) 2022-12-22
US9370582B2 (en) 2016-06-21
WO2009073809A3 (en) 2009-12-30
EP4321177A3 (en) 2024-05-08
AU2008340355A1 (en) 2009-07-02
JP5635412B2 (ja) 2014-12-03
JP2023010881A (ja) 2023-01-20
CA3043911A1 (en) 2009-07-02
US20140179761A1 (en) 2014-06-26
CA2708153C (en) 2017-09-26
US20220143189A1 (en) 2022-05-12
US11110174B2 (en) 2021-09-07
US20150119444A1 (en) 2015-04-30
EP4074344A1 (en) 2022-10-19
US20220008541A1 (en) 2022-01-13
EP3141265A1 (en) 2017-03-15
EP4321177A2 (en) 2024-02-14
JP2017002082A (ja) 2017-01-05
US20160051691A1 (en) 2016-02-25
WO2009073809A2 (en) 2009-06-11
US20150119445A1 (en) 2015-04-30
JP5519523B2 (ja) 2014-06-11
CA2708173C (en) 2016-02-02
US20150011615A1 (en) 2015-01-08
US20200297853A1 (en) 2020-09-24
JP6850827B2 (ja) 2021-03-31
US9867882B2 (en) 2018-01-16
CN102006890A (zh) 2011-04-06
EP3705125A1 (en) 2020-09-09
EP4223299A2 (en) 2023-08-09
EP2231195B1 (en) 2017-03-29
JP7590395B2 (ja) 2024-11-26
AU2008340355B2 (en) 2015-01-22
JP2016034974A (ja) 2016-03-17
US20190184018A1 (en) 2019-06-20
US9352048B2 (en) 2016-05-31
US20120136042A1 (en) 2012-05-31
CA3146103A1 (en) 2009-06-11
WO2009082607A2 (en) 2009-07-02
AU2008333811A1 (en) 2009-06-11
CA2910760A1 (en) 2009-07-02
US20090247608A1 (en) 2009-10-01
US20130178512A1 (en) 2013-07-11
CA2708153A1 (en) 2009-06-11
JP2018029599A (ja) 2018-03-01
WO2009082607A3 (en) 2009-12-17
CA2930393A1 (en) 2009-06-11
US8450467B2 (en) 2013-05-28
JP2019147846A (ja) 2019-09-05
CA2708173A1 (en) 2009-07-02
JP2011505425A (ja) 2011-02-24
US10806791B2 (en) 2020-10-20
US11666653B2 (en) 2023-06-06
JP2015025007A (ja) 2015-02-05
AU2008333811B2 (en) 2014-05-01
JP2021020928A (ja) 2021-02-18
EP2231195A2 (en) 2010-09-29
JP2021080299A (ja) 2021-05-27
EP3705125B1 (en) 2023-07-05
WO2009082607A8 (en) 2010-01-28
US9370581B2 (en) 2016-06-21
JP6215284B2 (ja) 2017-10-18
CA2930393C (en) 2022-11-29
US20160375137A9 (en) 2016-12-29
JP2011505426A (ja) 2011-02-24
JP2019023241A (ja) 2019-02-14
US20190099493A1 (en) 2019-04-04
JP6542853B2 (ja) 2019-07-10
EP3156077B1 (en) 2022-03-09
US12059468B2 (en) 2024-08-13
EP4223299A3 (en) 2023-08-30
US8828956B2 (en) 2014-09-09
EP3156077A1 (en) 2017-04-19
US9814777B2 (en) 2017-11-14
US20180326070A1 (en) 2018-11-15
JP5843914B2 (ja) 2016-01-13
EP2229186A2 (en) 2010-09-22

Similar Documents

Publication Publication Date Title
CA2910760C (en) Targeting lipids
US10835612B2 (en) Lipids and compositions for the delivery of therapeutics
CA2764609C (en) Improved cationic lipid of formula i
AU2020200082A1 (en) Targeting lipids
HK40075255A (en) Targeting lipids
AU2014277867B2 (en) Targeting lipids

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20151008

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 16TH ANNIV.) - STANDARD

Year of fee payment: 16

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241107

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20241107

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241107

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-4-4-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260226