AU2008340355B2 - Targeting lipids - Google Patents

Targeting lipids Download PDF

Info

Publication number
AU2008340355B2
AU2008340355B2 AU2008340355A AU2008340355A AU2008340355B2 AU 2008340355 B2 AU2008340355 B2 AU 2008340355B2 AU 2008340355 A AU2008340355 A AU 2008340355A AU 2008340355 A AU2008340355 A AU 2008340355A AU 2008340355 B2 AU2008340355 B2 AU 2008340355B2
Authority
AU
Australia
Prior art keywords
lipid
independently
ligand
occurrence
formula
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
AU2008340355A
Other languages
English (en)
Other versions
AU2008340355A1 (en
Inventor
Muthusamy Jayaraman
Muthiah Manoharan
Jayaprakash K. Narayanannair
Kallanthottathil G. Rajeev
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
Tekmira Pharmaceuticals 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 Tekmira Pharmaceuticals Corp filed Critical Tekmira Pharmaceuticals Corp
Publication of AU2008340355A1 publication Critical patent/AU2008340355A1/en
Assigned to TEKMIRA PHARMACEUTICALS CORPORATION reassignment TEKMIRA PHARMACEUTICALS CORPORATION Request for Assignment Assignors: ALNYLAM PHARMACEUTICALS, INC.
Priority to AU2014277867A priority Critical patent/AU2014277867B2/en
Application granted granted Critical
Publication of AU2008340355B2 publication Critical patent/AU2008340355B2/en
Priority to AU2022201011A priority patent/AU2022201011A1/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
    • A61K47/50—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
    • 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
    • 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)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (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)
  • Obesity (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Endocrinology (AREA)
  • Botany (AREA)
  • Emergency Medicine (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)
AU2008340355A 2007-12-04 2008-12-04 Targeting lipids Active AU2008340355B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2014277867A AU2014277867B2 (en) 2007-12-04 2014-12-22 Targeting lipids
AU2022201011A AU2022201011A1 (en) 2007-12-04 2022-02-16 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
PCT/US2008/085582 WO2009082607A2 (en) 2007-12-04 2008-12-04 Targeting lipids

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2014277867A Division AU2014277867B2 (en) 2007-12-04 2014-12-22 Targeting lipids

Publications (2)

Publication Number Publication Date
AU2008340355A1 AU2008340355A1 (en) 2009-07-02
AU2008340355B2 true AU2008340355B2 (en) 2015-01-22

Family

ID=40599687

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2008340355A Active AU2008340355B2 (en) 2007-12-04 2008-12-04 Targeting lipids
AU2008333811A Active AU2008333811B2 (en) 2007-12-04 2008-12-04 Carbohydrate conjugates as delivery agents for oligonucleotides

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2008333811A Active AU2008333811B2 (en) 2007-12-04 2008-12-04 Carbohydrate conjugates as delivery agents for oligonucleotides

Country Status (7)

Country Link
US (15) US20090247608A1 (OSRAM)
EP (8) EP4223299A3 (OSRAM)
JP (13) JP5635412B2 (OSRAM)
CN (1) CN102006890A (OSRAM)
AU (2) AU2008340355B2 (OSRAM)
CA (6) CA2930393C (OSRAM)
WO (2) WO2009073809A2 (OSRAM)

Families Citing this family (693)

* 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
EP2660322A3 (en) * 2003-04-17 2013-11-13 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
EP2231194B1 (en) * 2007-12-04 2017-02-22 Alnylam Pharmaceuticals Inc. Folate-irna conjugates
JP5635412B2 (ja) * 2007-12-04 2014-12-03 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． 標的化脂質
EP2274425A2 (en) * 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
CN102405286A (zh) 2008-09-22 2012-04-04 阿克赛医药公司 减小大小的自递送RNAi化合物
EP2379084B1 (en) 2008-10-15 2017-11-22 Ionis Pharmaceuticals, Inc. Modulation of factor 11 expression
CA3222620A1 (en) 2008-10-20 2010-04-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
CN111909020A (zh) * 2008-11-10 2020-11-10 阿布特斯生物制药公司 用于递送治疗剂的脂质和组合物
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US20120101148A1 (en) * 2009-01-29 2012-04-26 Alnylam Pharmaceuticals, Inc. lipid formulation
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
CA2753975C (en) 2009-03-02 2017-09-26 Alnylam Pharmaceuticals, Inc. Nucleic acid chemical modifications
CA2764158A1 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
CN102458366B (zh) * 2009-06-15 2015-02-11 阿尔尼拉姆医药品有限公司 靶向pcsk9基因的脂质配制的dsrna
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
ES2666559T3 (es) 2009-12-01 2018-05-07 Translate Bio, Inc. Entrega del mrna para la aumentación de proteínas y enzimas en enfermedades genéticas humanas
WO2011102904A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion therapeutic delivery system
WO2011102905A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion diagnostic delivery system
WO2011102906A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion delivery system
KR20180044433A (ko) 2010-03-24 2018-05-02 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 ｒｎａ 간섭
RU2615143C2 (ru) * 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
US8507663B2 (en) 2010-04-06 2013-08-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of CD274/PD-L1 gene
US9145556B2 (en) 2010-04-13 2015-09-29 Life Technologies Corporation Compositions and methods for inhibition of nucleic acids function
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
US20130071321A1 (en) 2010-05-28 2013-03-21 Purdue Research Foundation Delivery of agents to inflamed tissues using folate-targeted liposomes
US8785121B2 (en) 2010-07-08 2014-07-22 Bonac Corporation Single-stranded nucleic acid molecule for controlling gene expression
CA2805739A1 (en) * 2010-07-28 2012-02-02 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
CN103052711B (zh) * 2010-08-03 2016-08-03 株式会社博纳克 具有含氮脂环式骨架的单链核酸分子
KR20130137160A (ko) 2010-08-24 2013-12-16 머크 샤프 앤드 돔 코포레이션 내부의 비－핵산 스페이서를 함유하는 단일－가닥 ＲＮＡｉ 작용제
AU2011302152B2 (en) * 2010-09-15 2015-06-11 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)
CA2817960C (en) 2010-11-17 2020-06-09 Isis 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
MX346144B (es) 2010-12-17 2017-03-09 Arrowhead Res Corp * Porcion activadora del modulador farmacocinetico del agregado de galactosa para arnsi.
CA2976966C (en) 2010-12-29 2021-11-09 F. Hoffmann-La Roche Ag Small molecule conjugates for intracellular delivery of nucleic acids
EP3202760B1 (en) 2011-01-11 2019-08-21 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
CN103813810B (zh) * 2011-03-29 2021-08-03 阿尔尼拉姆医药品有限公司 用于抑制tmprss6基因表达的组合物和方法
BR112013031553A2 (pt) 2011-06-08 2020-11-10 Shire Human Genetic Therapies, Inc. composições, mrna que codifica para uma hgla e seu uso, uso de pelo menos uma molécula de mrna e um veículo de transferência e uso de um mrna que codifica para proteína exógena
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
CN120555426A (zh) 2011-06-21 2025-08-29 阿尔尼拉姆医药品有限公司 血管生成素样3（ANGPTL3）iRNA组合物及其使用方法
US9068184B2 (en) 2011-06-21 2015-06-30 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein C (PROC) genes
WO2012177921A2 (en) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc Compositions and methods for inhibiting hepcidin antimicrobial peptide (hamp) or hamp-related gene expression
KR102540778B1 (ko) 2011-06-21 2023-06-07 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
WO2012178033A2 (en) 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
KR102167524B1 (ko) 2011-06-30 2020-10-20 애로우헤드 파마슈티컬스 인코포레이티드 B형 간염 바이러스의 유전자 발현 저해용 조성물 및 방법
US9334300B2 (en) 2011-08-01 2016-05-10 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
JP6250543B2 (ja) 2011-09-27 2017-12-20 アルニラム・ファーマシューティカルズ・インコーポレーテッド ジ脂肪族置換ｐｅｇ化脂質
EP2594575A1 (en) * 2011-11-15 2013-05-22 Université de Strasbourg Mannosylated compounds useful for the prevention and the treatment of infectious diseases
LT3366775T (lt) 2011-11-18 2022-08-10 Alnylam Pharmaceuticals, Inc. Modifikuoti rnri agentai
RU2678807C2 (ru) * 2011-11-18 2019-02-01 Элнилэм Фармасьютикалз, Инк. СРЕДСТВА ДЛЯ РНКи, КОМПОЗИЦИИ И СПОСОБЫ ИХ ПРИМЕНЕНИЯ ДЛЯ ЛЕЧЕНИЯ ЗАБОЛЕВАНИЙ, АССОЦИИРОВАННЫХ С ТРАНСТИРЕТИНОМ (TTR)
ES2921724T1 (es) 2011-12-07 2022-08-31 Alnylam Pharmaceuticals Inc Lípidos biodegradables para la administración de agentes activos
MX357392B (es) 2012-04-02 2018-07-06 Berg Llc Ensayos basados en interrogatorios celulares y uso 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
TWI595885B (zh) * 2012-05-02 2017-08-21 喜納製藥公司 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
WO2013173789A2 (en) 2012-05-17 2013-11-21 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
US9663784B2 (en) * 2012-05-26 2017-05-30 Bonac Corporation Single-stranded nucleic acid molecule for regulating expression of gene having delivering function
KR20200118233A (ko) 2012-06-01 2020-10-14 버그 엘엘씨 조효소 q10을 이용한 고형 종양의 치료
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
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
CA2876155C (en) 2012-06-08 2022-12-13 Ethris Gmbh 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
EP4253395A3 (en) * 2012-08-06 2023-11-29 Alnylam Pharmaceuticals, Inc. Processes for the preparation of carbohydrate conjugated rna agents
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
JP2016503394A (ja) 2012-10-26 2016-02-04 エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． 細胞型へのオリゴヌクレオチド分子の選択的送達のための組成物および方法
CN117126846A (zh) 2012-11-15 2023-11-28 罗氏创新中心哥本哈根有限公司 寡核苷酸缀合物
US9506897B2 (en) 2012-11-16 2016-11-29 Agilent Technologies, Inc. Methods and compositions for improved ion-exchange chromatography
SMT201800039T1 (it) * 2012-12-05 2018-03-08 Alnylam Pharmaceuticals Inc Composizioni di irna di pcsk9 e metodi di uso delle stesse
US20150337318A1 (en) * 2013-01-11 2015-11-26 Phaserx Inc. Rna targeted to c-met
CA2893801A1 (en) * 2013-01-30 2014-08-07 F. Hoffmann-La Roche Ag Lna oligonucleotide carbohydrate conjugates
CN120574828A (zh) * 2013-03-14 2025-09-02 阿尔尼拉姆医药品有限公司 补体组分C5 iRNA组合物及其使用方法
EP4446413A3 (en) 2013-03-14 2024-12-18 Translate Bio, Inc. Methods for purification of messenger rna
HUE055044T2 (hu) 2013-03-14 2021-10-28 Translate Bio Inc MRNS-kódolt ellenanyagok bejuttatására szolgáló eljárások és készítmények
WO2014153052A2 (en) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
KR20150131365A (ko) 2013-03-15 2015-11-24 미라젠 세러퓨틱스 인코포레이티드 브리지드 바이사이클릭 뉴클레오시드
ES2795249T3 (es) 2013-03-15 2020-11-23 Translate Bio Inc Mejora sinérgica de la administración de ácidos nucleicos a través de formulaciones mezcladas
MX380565B (es) 2013-04-08 2025-03-12 Berg Llc Terapias combinadas de coenzima q10 para el tratamiento de cáncer.
TWI680767B (zh) 2013-05-01 2020-01-01 美商雷格勒斯治療公司 用於增強的細胞攝取之化合物及方法
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
AR096203A1 (es) 2013-05-06 2015-12-16 Alnylam Pharmaceuticals Inc Dosificaciones y métodos para administrar moléculas de ácido nucleico formuladas en lípidos
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
PL2999785T3 (pl) 2013-05-22 2018-09-28 Alnylam Pharmaceuticals, Inc. Kompozycje iRNA Serpina1 i sposoby ich zastosowania
SG10202103166XA (en) * 2013-05-22 2021-04-29 Alnylam Pharmaceuticals Inc Tmprss6 irna compositions and methods of use thereof
EP3564374A1 (en) 2013-06-21 2019-11-06 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
SG11201510656PA (en) 2013-06-27 2016-01-28 Roche Innovation Ct Copenhagen As Antisense oligomers and conjugates targeting pcsk9
JP6422961B2 (ja) * 2013-07-05 2018-11-14 バイオニア コーポレーションＢｉｏｎｅｅｒ Ｃｏｒｐｏｒａｔｉｏｎ 改善された高効率ナノ粒子型オリゴヌクレオチド構造体およびその製造方法
CA3177846A1 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
AU2014315186B2 (en) 2013-09-04 2020-02-27 Berg Llc Methods of treatment of cancer by continuous infusion of coenzyme Q10
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
CA2925107A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
EP3052628B1 (en) * 2013-10-04 2020-02-26 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
EP4036241A1 (en) 2013-10-22 2022-08-03 Translate Bio, Inc. Cns delivery of mrna and uses thereof
CN105658242A (zh) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 用于苯丙酮尿症的mrna疗法
AU2014340155B2 (en) 2013-10-22 2018-11-01 Massachusetts Institute Of Technology Lipid formulations for delivery of messenger RNA
EP3060664B1 (en) 2013-10-25 2021-07-07 Sanofi Microrna compounds and methods for modulating mir-21 activity
US20160289677A1 (en) * 2013-11-14 2016-10-06 Roche Innovation Center Copenhagen A/S APOB Antisense Conjugate Compounds
CN113151180A (zh) 2013-12-02 2021-07-23 菲奥医药公司 癌症的免疫治疗
SG11201604692UA (en) 2013-12-12 2016-07-28 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
JP6486836B2 (ja) 2013-12-26 2019-03-20 学校法人東京医科大学 遺伝子発現制御のための人工ミミックｍｉＲＮＡおよびその用途
KR102357337B1 (ko) 2013-12-27 2022-01-28 가부시키가이샤 보낙 유전자 발현 제어를 위한 인공 매치형 miRNA 및 그 용도
WO2015106128A2 (en) * 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
WO2015107115A1 (en) * 2014-01-15 2015-07-23 Basf Se Saccharide-modified nucleic acid molecules
WO2015113922A1 (en) 2014-01-30 2015-08-06 Roche Innovation Center Copenhagen A/S Poly oligomer compound with biocleavable conjugates
CN106103718B (zh) 2014-02-11 2021-04-02 阿尔尼拉姆医药品有限公司 己酮糖激酶(KHK)iRNA组合物及其使用方法
RU2019130898A (ru) 2014-03-19 2019-11-11 Ионис Фармасьютикалз, Инк. Композиции для модуляции экспрессии атаксина 2
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
SG10201910844SA (en) 2014-04-01 2020-01-30 Biogen Ma Inc Compositions for modulating sod-1 expression
US10426753B2 (en) 2014-04-03 2019-10-01 Invictus Oncology Pvt. Ltd. Supramolecular combinatorial therapeutics
CN106164248B (zh) 2014-04-25 2019-10-15 川斯勒佰尔公司 信使rna的纯化方法
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
EP3137476B1 (en) 2014-04-28 2019-10-09 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
EP3137115B1 (en) * 2014-05-01 2020-10-14 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
WO2015168532A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating pkk expression
KR20200102553A (ko) 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
DK3137596T3 (da) 2014-05-01 2019-09-02 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af komplement faktor b-ekspression
AU2015252895B2 (en) 2014-05-01 2021-07-15 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
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
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2015179724A1 (en) 2014-05-22 2015-11-26 Alnylam Pharmaceuticals, Inc. Angiotensinogen (agt) irna compositions and methods of use thereof
CN106659731A (zh) 2014-05-30 2017-05-10 夏尔人类遗传性治疗公司 用于递送核酸的可生物降解脂质
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
JP6599373B2 (ja) 2014-06-24 2019-10-30 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立体化学的に濃縮した組成物
EP3160938B1 (en) 2014-06-25 2020-09-16 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
US9668980B2 (en) 2014-07-02 2017-06-06 Rana Therapeutics, Inc. Encapsulation of messenger RNA
BR112017001931A2 (pt) 2014-08-07 2017-11-28 Regulus Therapeutics Inc direcionamento de micrornas para distúrbios metabólicos
WO2016030863A1 (en) 2014-08-29 2016-03-03 Glaxosmithkline Intellectual Property Development Limited Compounds and methods for treating viral infections
US10060921B2 (en) 2014-08-29 2018-08-28 Alnylam Pharmaceuticals, Inc. Methods of treating transthyretin (TTR) mediated amyloidosis
KR102689262B1 (ko) 2014-09-05 2024-07-30 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
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
SG11201702682PA (en) 2014-10-03 2017-04-27 Cold Spring Harbor Lab Targeted augmentation of nuclear gene output
WO2016057693A1 (en) * 2014-10-10 2016-04-14 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhalation delivery of conjugated oligonucleotide
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
CA2961993A1 (en) 2014-10-10 2016-04-14 F. Hoffmann-La Roche Ag Galnac phosphoramidites, nucleic acid conjugates thereof and their use
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3904519A1 (en) 2014-10-30 2021-11-03 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
US11203776B2 (en) 2014-11-12 2021-12-21 Tumorend, Llc Compositions, methods and treatments for inhibiting cell adhesion and virus binding and penetration
US10822369B2 (en) 2014-11-14 2020-11-03 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of proteins
JP2017535552A (ja) 2014-11-17 2017-11-30 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． アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用方法
WO2016085852A1 (en) 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
EP3226912B1 (en) 2014-12-05 2021-01-20 Translate Bio, Inc. Messenger rna therapy for treatment of articular disease
WO2016094425A1 (en) 2014-12-08 2016-06-16 Berg Llc Use of markers including filamin a in the diagnosis and treatment of prostate cancer
US12359197B2 (en) 2014-12-12 2025-07-15 Etagen Pharma, Inc. Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
DK3569711T3 (da) * 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
AU2015364508A1 (en) 2014-12-18 2017-07-06 Alnylam Pharmaceuticals, Inc. ReversirTM compounds
CA2972065A1 (en) * 2014-12-23 2016-06-30 Intellectual Property Associates, Llc Methods and formulations for transdermal administration
MX381947B (es) 2014-12-27 2025-03-11 Bonac Corp Mirna natural para controlar la expresion de gen y uso del mismo.
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016115490A1 (en) 2015-01-16 2016-07-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
US20180002692A1 (en) * 2015-01-30 2018-01-04 Anushree Chatterjee Sequence Specific and Organism Specific Antimicrobials and Related Materials and Methods
JP2018510621A (ja) 2015-02-13 2018-04-19 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． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
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
JP6895892B2 (ja) 2015-03-19 2021-06-30 トランスレイト バイオ， インコーポレイテッド ポンペ病のｍＲＮＡ治療
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
CN107429250B (zh) 2015-04-03 2022-03-01 Ionis制药公司 用于调节tmprss6表达的化合物和方法
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
KR20250005546A (ko) 2015-04-13 2025-01-09 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(angptl3) irna 조성물 및 이의 이용 방법
US10407678B2 (en) 2015-04-16 2019-09-10 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
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
AU2016285852B2 (en) 2015-06-29 2020-12-17 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
GB201511459D0 (en) * 2015-06-30 2015-08-12 Cell Therapy Ltd Sirna microbubble
WO2017007825A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation 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
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
AU2016294347B2 (en) 2015-07-10 2022-07-28 Ionis Pharmaceuticals, Inc. Modulators of diacyglycerol acyltransferase 2 (DGAT2)
EP3324980B1 (en) 2015-07-17 2021-11-10 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
WO2017019660A1 (en) 2015-07-27 2017-02-02 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
JP6896703B2 (ja) 2015-07-31 2021-06-30 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． ＴＴＲ関連疾患を治療または予防するためのトランスサイレチン（ＴＴＲ）ｉＲＮＡ組成物およびその使用方法
NZ739902A (en) 2015-07-31 2022-09-30 Arcturus Therapeutics Inc Multiligand agent for drug delivery
AU2016306275A1 (en) 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
AU2016310494B2 (en) 2015-08-25 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating a proprotein convertase subtilisin kexin (PCSK9) gene-associated disorder
AU2016315584B2 (en) 2015-09-02 2022-07-14 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
RU2018113709A (ru) 2015-09-24 2019-10-30 Айонис Фармасьютикалз, Инк. Модуляторы экспрессии kras
PT3353303T (pt) 2015-09-25 2023-10-10 Academisch Ziekenhuis Leiden Composições e métodos para modulação da expressão de ataxina 3
JP2018530325A (ja) 2015-10-08 2018-10-18 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． アンジオテンシノーゲンの発現を調節するための化合物及び方法
JP2018535197A (ja) * 2015-10-09 2018-11-29 ダイス モレキュルズ エスヴィー， エルエルシーＤｉｃｅ Ｍｏｌｅｃｕｌｅｓ Ｓｖ， Ｌｌｃ タグ付きコンビナトリアルライブラリ化合物のＬｏｇＤを推定する方法
US10196639B2 (en) 2015-10-09 2019-02-05 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017066573A1 (en) 2015-10-14 2017-04-20 Shire Human Genetic Therapies, Inc. Modification of rna-related enzymes for enhanced production
EP3365446A4 (en) 2015-10-19 2019-06-26 Phio Pharmaceuticals Corp. SMALL SELF-LEANING NUCLEIC ACID COMPOUNDS TURNED AGAINST LONG NON-CODING RNA
FI3368507T3 (fi) 2015-10-28 2023-03-21 Acuitas Therapeutics Inc Uusia lipidejä ja lipidinanopartikkeliformulaatioita nukleiinihappojen annostelemiseksi
DK4119569T3 (da) 2015-11-06 2024-08-12 Ionis Pharmaceuticals Inc Konjugerede antisense-forbindelser til anvendelse i behandling
CA2999341A1 (en) 2015-11-06 2017-05-11 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
WO2017096395A1 (en) 2015-12-04 2017-06-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
TWI836281B (zh) 2015-12-07 2024-03-21 美商健贊公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法及組成物
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
JP7049248B2 (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 常染色体優性精神遅滞－５とドラベ症候群の処置のためのアンチセンスオリゴマー
EP3389671A4 (en) 2015-12-14 2019-07-17 Cold Spring Harbor Laboratory ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME
CA3005254A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13
EP4104867A3 (en) 2015-12-14 2023-03-01 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3389672A4 (en) 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER DISEASES
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
CA3006015A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
AU2017205462A1 (en) 2016-01-05 2018-06-07 Ionis Pharmaceuticals, Inc. Methods for reducing LRRK2 expression
ES2858090T3 (es) 2016-01-29 2021-09-29 Kyowa Kirin Co Ltd Complejo de ácidos nucleicos
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
CA3011946A1 (en) * 2016-03-07 2017-09-14 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
AU2017234678A1 (en) 2016-03-16 2018-08-16 Ionis Pharmaceuticals, Inc. Methods of modulating KEAP1
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
EP3436066A1 (en) 2016-04-01 2019-02-06 Checkmate Pharmaceuticals, Inc. Fc receptor-mediated drug delivery
CA3020343A1 (en) 2016-04-08 2017-10-12 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
MA45478A (fr) * 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
WO2017178656A1 (en) 2016-04-14 2017-10-19 Roche Innovation Center Copenhagen A/S TRITYL-MONO-GalNAc COMPOUNDS AND THEIR USE
WO2017184786A1 (en) 2016-04-19 2017-10-26 The Broad Institute Inc. Cpf1 complexes with reduced indel activity
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
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
SG11201809002RA (en) 2016-04-29 2018-11-29 Univ Nanyang Tech G-quadruplex-containing antisense oligonucleotides
US11040108B2 (en) 2016-04-29 2021-06-22 Brandeis University Amino acid- and peptide-steroid conjugates and use thereof
EP3452055A4 (en) 2016-05-06 2019-11-06 Tod M. Woolf IMPROVED METHODS FOR GENERIC MODIFICATION WITH AND WITHOUT PROGRAMMABLE NUCLEASES
WO2017214518A1 (en) 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
US10835583B2 (en) 2016-06-13 2020-11-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
CA3023514A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
JP7022687B2 (ja) * 2016-06-30 2022-02-18 協和キリン株式会社 核酸複合体
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
AU2017296195A1 (en) 2016-07-11 2019-01-24 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
US10781175B2 (en) 2016-07-15 2020-09-22 Am Chemicals Llc Solid supports and phosphoramidite building blocks for oligonucleotide conjugates
CA3030864A1 (en) 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
CA3033867A1 (en) 2016-08-17 2018-02-22 Solstice Biologics, Ltd. Polynucleotide constructs
UY37376A (es) 2016-08-26 2018-03-23 Amgen Inc Construcciones de arni para inhibir expresión de asgr1 y métodos para su uso
KR102403408B1 (ko) 2016-09-02 2022-05-30 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
KR101933767B1 (ko) 2016-09-05 2018-12-28 (의료)길의료재단 발기부전 환자를 위한 콘돔
WO2018067900A1 (en) * 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of 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
TWI788312B (zh) 2016-11-23 2023-01-01 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
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2018102745A1 (en) 2016-12-02 2018-06-07 Cold Spring Harbor Laboratory Modulation of lnc05 expression
EP3554561B1 (en) 2016-12-14 2023-06-28 Janssen Biotech, Inc. Cd137 binding fibronectin type iii domains
EP3554535A4 (en) 2016-12-14 2020-10-21 Janssen Biotech, Inc. PD-L1 BINDING FIBRONECTIN TYPE III DOMAINS
BR112019012154A2 (pt) 2016-12-14 2019-11-12 Janssen Biotech, Inc. domínios do tipo iii da fibronectina de ligação a cd8a
KR20190098181A (ko) 2016-12-16 2019-08-21 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물을 사용하여 TTR-관련 질병을 치료하거나 예방하는 방법
JP7348064B2 (ja) 2017-01-09 2023-09-20 アポセンス リミテッド 分子を経膜送達するための化合物及び方法
KR102675026B1 (ko) 2017-01-10 2024-06-17 애로우헤드 파마슈티컬스 인코포레이티드 알파-1 항트립신 (AAT) RNAi 작용제, AAT RNAi 작용제를 포함하는 조성물, 및 사용 방법
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
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
EP3603648B1 (en) * 2017-03-29 2025-09-17 Shionogi & Co., Ltd Complex of nucleic acid medicine and multibranched lipid
RS63836B1 (sr) 2017-04-05 2023-01-31 Silence Therapeutics Gmbh Proizvodi i sastavi
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
KR20250145702A (ko) 2017-04-11 2025-10-13 아뷰터스 바이오파마 코포레이션 표적화 조성물
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
US11324820B2 (en) 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
WO2018195165A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
IL301115A (en) 2017-04-28 2023-05-01 Acuitas Therapeutics Inc Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
US11273222B2 (en) * 2017-05-26 2022-03-15 National Cerebral And Cardiovascular Center Antisense nucleic acid targeting PCSK9
CN110799647A (zh) 2017-06-23 2020-02-14 马萨诸塞大学 双尾自递送sirna及相关的方法
EP3645546A4 (en) 2017-06-30 2021-12-01 Solstice Biologics, Ltd. CHIRAL PHOSPHORAMIDITIS AUXILIARIES AND THEIR METHODS OF USE
US10195286B2 (en) * 2017-07-04 2019-02-05 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
JP2020530442A (ja) 2017-07-10 2020-10-22 ジェンザイム・コーポレーション 血友病を有する対象の出血事象を処置するための方法および組成物
US11261447B2 (en) 2017-07-13 2022-03-01 Alnylam Pharmaceuticals, Inc. Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
JP7277432B2 (ja) 2017-07-13 2023-05-19 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 乳酸脱水素酵素Ａ（ＬＤＨＡ）ｉＲＮＡ組成物及びその使用方法
WO2019027999A1 (en) * 2017-07-31 2019-02-07 Ohio State Innovation Foundation BIOMIMETIC NANOMATERIALS AND USES THEREOF
WO2019027015A1 (ja) * 2017-08-02 2019-02-07 協和発酵キリン株式会社 核酸複合体
WO2019027009A1 (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
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036000A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
CA3071033A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
CA3075219A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Hnf4a sarna compositions and methods of use
EP3679140B1 (en) * 2017-09-08 2022-11-16 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
WO2019051642A1 (zh) * 2017-09-12 2019-03-21 广州中科蓝华生物科技有限公司 一种转染细胞内寄生虫的试剂盒及其应用
US10995335B2 (en) 2017-09-14 2021-05-04 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
WO2019053661A2 (en) * 2017-09-14 2019-03-21 Alios Biopharma, Inc. GALNAC DERIVATIVES
US11806360B2 (en) 2017-09-19 2023-11-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
EP3700570B1 (en) 2017-10-23 2025-01-08 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
CA3081910A1 (en) 2017-11-13 2019-05-16 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
WO2019105419A1 (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
EP3719125B1 (en) 2017-12-01 2025-01-08 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing same, and preparation method and use
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
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
EP3718572B1 (en) 2017-12-01 2024-07-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method and use
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
EA202091520A1 (ru) 2017-12-18 2020-10-05 Элнилэм Фармасьютикалз, Инк. Композиции на основе irna против бокса-1 высокомобильной группы (hmgb1) и способы их применения
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
AU2018392782B2 (en) 2017-12-21 2023-08-31 Alnylam Pharmaceuticals, Inc. Chirally-enriched double-stranded RNA agents
US11459564B2 (en) 2017-12-21 2022-10-04 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
AU2017444369B2 (en) * 2017-12-26 2022-02-24 Guangzhou Ribobio Co., Ltd. Modified oligonucleotides and compound that can be used for synthesizing same
KR102617947B1 (ko) 2017-12-29 2023-12-27 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 접합체와 제조 및 그 용도
WO2019130319A1 (en) 2018-01-01 2019-07-04 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
EP3737424A4 (en) * 2018-01-10 2021-10-27 Translate Bio MA, Inc. COMPOSITIONS AND METHODS TO FACILITATE THE DELIVERY OF SYNTHETIC NUCLEIC ACIDS INTO CELLS
WO2019140452A1 (en) 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
CA3090901A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
WO2019160866A2 (en) 2018-02-13 2019-08-22 Checkmate Pharmaceuticals, Inc. Compositions and methods for tumor immunotherapy
SG11202007644PA (en) 2018-02-14 2020-09-29 Deep Genomics Incorporated Oligonucleotide therapy for wilson disease
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
CN115976028B (zh) 2018-03-09 2025-03-25 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
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
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
EP3768694A4 (en) 2018-03-22 2021-12-29 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
EP3775207A1 (en) 2018-04-05 2021-02-17 Silence Therapeutics GmbH Sirnas with vinylphosphonate at the 5' end of the antisense strand
WO2019193189A1 (en) * 2018-04-05 2019-10-10 Silence Therapeutics Gmbh siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS
EP3550021A1 (en) * 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions for inhibiting expression of a target gene
EP3775218A1 (en) 2018-04-09 2021-02-17 Checkmate Pharmaceuticals Packaging oligonucleotides into virus-like particles
KR20200141481A (ko) 2018-04-11 2020-12-18 아이오니스 파마수티컬즈, 인코포레이티드 Ezh2 발현의 조절제
EP4242307A3 (en) 2018-04-12 2023-12-27 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
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
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
BR112020020957B1 (pt) 2018-05-09 2022-05-10 Ionis Pharmaceuticals, Inc Compostos oligoméricos, população e composição farmacêutica dos mesmos e seus usos
CA3098144A1 (en) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
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
WO2019241648A1 (en) 2018-06-14 2019-12-19 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing stmn2 expression
US20210251994A1 (en) 2018-06-15 2021-08-19 Flagship Pioneering Innovations V, Inc. Increasing immune activity through modulation of postcellular signaling factors
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
WO2020018558A1 (en) 2018-07-17 2020-01-23 Aronora, Inc. Methods for safely reducing thrombopoietin
CN112424356A (zh) 2018-07-20 2021-02-26 莱古路斯治疗法股份有限公司 用于经口递送寡核苷酸的方法
EP3826645A4 (en) 2018-07-25 2023-05-17 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING ATXN2 EXPRESSION
US20210292768A1 (en) 2018-08-08 2021-09-23 Arcturus Therapeutics, Inc. Compositions and agents against nonalcoholic steatohepatitis
AU2019316640A1 (en) 2018-08-10 2021-03-18 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
TW202020157A (zh) 2018-08-16 2020-06-01 美商艾爾妮蘭製藥公司 用於抑制lect2基因表現之組合物及方法
CN111655849B (zh) 2018-08-21 2024-05-10 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
EP3840759A4 (en) 2018-08-23 2022-06-01 University Of Massachusetts O-METHYL RICH FULLY STABILIZED OLIGONUCLEOTIDES
KR20210060480A (ko) 2018-08-24 2021-05-26 트랜슬레이트 바이오 인코포레이티드 전령 rna의 정제 방법
WO2020060986A1 (en) 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
TW202542311A (zh) 2018-09-19 2025-11-01 美商Ionis製藥公司 Pnpla3表現之調節劑
IL322436A (en) 2018-09-21 2025-09-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
AU2019350765A1 (en) 2018-09-28 2021-05-27 Alnylam Pharmaceuticals, Inc. Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated ocular diseases
CN111655297A (zh) 2018-09-30 2020-09-11 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
GB201816554D0 (en) 2018-10-10 2018-11-28 Centauri Therapeutics Ltd Novel compounds and therapeutic uses thereof
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
WO2020102630A1 (en) 2018-11-15 2020-05-22 Ionis Pharmaceuticals, Inc. Modulators of irf5 expression
BR112021007476A2 (pt) 2018-11-21 2021-11-03 Ionis Pharmaceuticals Inc Compostos e métodos para reduzir a expressão de príon
CA3120647A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
US12416004B2 (en) 2018-11-23 2025-09-16 Sanofi RNA compositions and methods for inhibiting ANGPTL8
CA3121449A1 (en) 2018-11-30 2020-06-04 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
JP7109674B2 (ja) * 2018-11-30 2022-07-29 キロノヴァ （シアメン） バイオファーマ カンパニー リミテッド 肝臓標的化特異的リガンドと甲状腺ホルモン受容体アゴニストとを含有する医薬品
EP3894559A4 (en) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY
CN119061010A (zh) 2018-12-10 2024-12-03 美国安进公司 经化学修饰的RNAi构建体及其用途
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
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ， インコーポレイテッド Ｋｃｎｔ１関連障害の治療のための組成物及び方法
WO2020132564A1 (en) 2018-12-21 2020-06-25 Ionis Pharmaceuticals, Inc. Modulators of hsd17b13 expression
JP7507495B2 (ja) 2018-12-28 2024-06-28 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
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
CN113474328B (zh) 2019-01-11 2025-07-11 爱康泰生治疗公司 用于脂质纳米颗粒递送活性剂的脂质
BR112021013956A2 (pt) 2019-01-16 2021-09-21 Genzyme Corporation Composições de irna de serpinc1 e métodos de uso das mesmas
CN118562796A (zh) * 2019-01-18 2024-08-30 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
AU2020207935A1 (en) 2019-01-18 2021-08-26 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
EP3917540A1 (en) 2019-01-31 2021-12-08 Ionis Pharmaceuticals, Inc. Modulators of yap1 expression
CA3131700A1 (en) 2019-02-27 2020-09-03 Ionis Pharmaceuticals, Inc. Modulators of malat1 expression
HUE071494T2 (hu) 2019-03-29 2025-09-28 Ionis Pharmaceuticals Inc Vegyületek és eljárások az UBE3A-ATS modulálására
US20220211740A1 (en) 2019-04-12 2022-07-07 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
MA55805A (fr) 2019-05-03 2022-03-09 Flagship Pioneering Innovations V Inc Métodes de modulation de l'activité immunitaire
EP3969052A1 (en) 2019-05-17 2022-03-23 Alnylam Pharmaceuticals Inc. Oral delivery of oligonucleotides
US20240200060A1 (en) 2019-05-22 2024-06-20 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
EP3974530A4 (en) 2019-05-22 2023-07-12 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, METHOD FOR PREPARATION AND USE
EP3974532A4 (en) 2019-05-22 2024-01-24 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PROCESS OF PREPARATION AND USE
EP3974533A4 (en) 2019-05-22 2023-11-08 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PROCESS OF PREPARATION 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
CN118599833A (zh) 2019-05-24 2024-09-06 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
WO2020238763A1 (zh) 2019-05-24 2020-12-03 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
BR112021024080A2 (pt) 2019-05-30 2022-02-08 Amgen Inc Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos
EP3990465A1 (en) 2019-06-25 2022-05-04 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
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021030213A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
EP4013871A1 (en) 2019-08-13 2022-06-22 Amgen Inc. Rnai constructs for inhibiting slc30a8 expression 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
WO2021037205A1 (zh) 2019-08-29 2021-03-04 苏州瑞博生物技术股份有限公司 化合物和药物缀合物及其制备方法和用途
JP7805286B2 (ja) 2019-09-03 2026-01-23 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 Ｌｅｃｔ２遺伝子の発現を阻害するための組成物および方法
CA3152529A1 (en) 2019-09-03 2021-03-11 Arcturus Therapeutics, Inc. Asialoglycoprotein receptor mediated delivery of therapeutically active conjugates
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
CN114728017B (zh) 2019-10-14 2025-10-28 阿斯利康(瑞典)有限公司 Pnpla3表达的调节剂
US11781138B2 (en) 2019-10-14 2023-10-10 Aro Biotherapeutics Company FN3 domain-siRNA conjugates and uses thereof
US12491255B2 (en) 2019-10-14 2025-12-09 Aro Biotherapeutics Company EPCAM binding fibronectin type III domains
EP4045061A4 (en) 2019-10-14 2024-04-17 ARO Biotherapeutics Company CD71-BINDING FIBRONECTIN TYPE III DOMAINS
WO2021076828A1 (en) 2019-10-18 2021-04-22 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
AU2020369515A1 (en) 2019-10-22 2022-04-21 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
AR120341A1 (es) 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
CA3160329A1 (en) * 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
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
WO2021096763A1 (en) 2019-11-13 2021-05-20 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
US20210322339A1 (en) 2019-11-20 2021-10-21 Berg Llc Combination therapy of coenzyme q10 and radiation for treatment of glioma
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
WO2021119034A1 (en) 2019-12-09 2021-06-17 Amgen Inc. RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
JP2023514000A (ja) 2019-12-09 2023-04-05 エンピリコ インク． アンジオポエチン様４（ａｎｇｐｔｌ４）関連疾患の処置のためのオリゴヌクレオチド
TWI897902B (zh) 2019-12-13 2025-09-21 美商阿尼拉製藥公司 人類染色體9開讀框72 (C9ORF72) iRNA劑組成物及其使用方法
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
EP4076434A1 (en) 2019-12-17 2022-10-26 Flagship Pioneering Innovations V, Inc. Combination anti-cancer therapies with inducers of iron-dependent cellular disassembly
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
JP7681525B2 (ja) 2020-01-30 2025-05-22 エーザイ・アール・アンド・ディー・マネジメント株式会社 核酸複合体及びそれを含む医薬組成物
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)
IL295445A (en) 2020-02-10 2022-10-01 Alnylam Pharmaceuticals Inc Preparations and methods for silencing vegf-a expression
KR20220143106A (ko) 2020-02-18 2022-10-24 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 C3 (APOC3) iRNA 조성물 및 이의 사용 방법
IL295605A (en) 2020-02-28 2022-10-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating smn2
WO2021178725A1 (en) 2020-03-04 2021-09-10 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
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
KR20220152270A (ko) 2020-03-06 2022-11-15 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴 (ttr)의 발현을 억제하기 위한 조성물 및 방법
IL296109A (en) 2020-03-06 2022-11-01 Alnylam Pharmaceuticals Inc Ketohexokinase (khk) irna compositions and methods of use thereof
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
JP2023519215A (ja) 2020-03-23 2023-05-10 アムジエン・インコーポレーテツド 化学修飾核酸に対するモノクローナル抗体及びその使用
US20230134550A1 (en) 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
CA3172572A1 (en) 2020-03-24 2021-09-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
JP2023519274A (ja) 2020-03-26 2023-05-10 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 コロナウイルスｉＲＮＡ組成物およびその使用方法
WO2021202443A2 (en) 2020-03-30 2021-10-07 Alnylam Pharmaceucticals, Inc. Compositions and methods for silencing dnajc15 gene expression
EP4127134A4 (en) 2020-04-01 2024-07-31 Alnylam Pharmaceuticals, Inc. COMPOSITIONS OF ALPHA-2A ADRENERGIC RECEPTOR (ADRA2A) ARNI AGENTS AND METHODS OF USE THEREOF
EP4133078A1 (en) 2020-04-06 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
KR20230008078A (ko) 2020-04-07 2023-01-13 알닐람 파마슈티칼스 인코포레이티드 Scn9a 발현을 사일런싱하기 위한 조성물 및 방법
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
EP4132478A1 (en) 2020-04-09 2023-02-15 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
JP2023522961A (ja) 2020-04-21 2023-06-01 フラッグシップ パイオニアリング， インコーポレイテッド 二機能性分子およびその使用方法
BR112022021813A2 (pt) 2020-04-27 2023-01-17 Alnylam Pharmaceuticals Inc Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas
US20240270780A1 (en) * 2020-04-29 2024-08-15 Tai Bi Di Pharmaceutical Technology (Shijiazhuang) Co. Ltd Proteolysis targeting compound with tissue targeting capability and use thereof
WO2021222549A1 (en) 2020-04-30 2021-11-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
BR112022021333A2 (pt) 2020-05-01 2022-12-13 Ionis Pharmaceuticals Inc Compostos e métodos para modular atxn1
IL298063A (en) 2020-05-11 2023-01-01 Stoke Therapeutics Inc opa1 antisense oligomers for the treatment of conditions and diseases
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
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)
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
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)
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)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
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
MX2022014606A (es) 2020-05-22 2023-03-08 Wave Life Sciences Ltd Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas.
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
IL298697A (en) 2020-06-01 2023-02-01 Amgen Inc Rnai constructs for inhibiting hsd17b13 expression and methods of use thereof
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
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
AU2021292296A1 (en) 2020-06-18 2023-01-19 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof
MX2022016338A (es) 2020-06-29 2023-01-24 Ionis Pharmaceuticals Inc Compuestos y metodos para modular la plp1.
CN116096906A (zh) 2020-06-29 2023-05-09 旗舰创业创新五公司 工程化以促进萨诺传递的病毒及其在治疗癌症中的用途
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
US20240018524A1 (en) 2020-07-10 2024-01-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for treating epilepsy
EP4182297B1 (en) 2020-07-16 2025-09-03 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
MX2023001222A (es) 2020-07-28 2023-04-26 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de app.
MX2023001486A (es) 2020-08-07 2023-03-27 Ionis Pharmaceuticals Inc Compuestos y metodos para modular scn2a.
MX2023001786A (es) 2020-08-13 2023-03-10 Amgen Inc Construcciones de iarn y metodos para inhibir la expresion de marc1.
US20230303618A1 (en) * 2020-08-21 2023-09-28 Reyoung Corporation Triptolide conjugates and uses thereof
UY39420A (es) * 2020-09-11 2022-03-31 Arrowhead Pharmaceuticals Inc Conjugados lipídicos para el transporte de agentes terapéuticos
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
EP4225917A1 (en) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
WO2022079221A1 (en) 2020-10-16 2022-04-21 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
US20230383294A1 (en) 2020-10-16 2023-11-30 Sanofi Novel rna compositions and methods for inhibiting angptl3
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 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
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc Coagulation factor iRNA compositions (F5) and methods of using them
EP4488371A3 (en) 2020-11-18 2025-04-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
AU2021393417A1 (en) 2020-12-01 2023-06-29 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
IL303800A (en) 2020-12-18 2023-08-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating factor xii
US20240058279A1 (en) 2020-12-21 2024-02-22 Flagship Pioneering Innovations V, Inc. Use of cell turnover factors for increasing tissue regeneration
US20240175020A1 (en) 2020-12-23 2024-05-30 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
CN116669746B (zh) 2020-12-29 2025-09-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
WO2022150260A1 (en) 2021-01-05 2022-07-14 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
AU2022213743A1 (en) * 2021-01-28 2023-06-15 Nanjing Chempion Biotechnology Co., Ltd. Conjugate and use thereof
EP4175965B1 (en) * 2021-01-30 2024-03-13 E-Therapeutics plc Conjugated oligonucleotide compounds, methods of making and uses thereof
JP2024504504A (ja) * 2021-01-30 2024-01-31 イー セラピューティクス パブリック リミテッド カンパニー コンジュゲートオリゴヌクレオチド化合物、その作製方法及び使用
TW202305131A (zh) 2021-02-12 2023-02-01 美商艾拉倫製藥股份有限公司 用於治療或預防超氧歧化酶１－（ＳＯＤ１－）相關的神經退化疾病的超氧歧化酶１（ＳＯＤ１）ｉＲＮＡ組成物及其使用方法
JP2024509783A (ja) 2021-02-25 2024-03-05 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 プリオンタンパク質（ｐｒｎｐ）ｉｒｎａ組成物およびその使用方法
EP4298218A1 (en) 2021-02-26 2024-01-03 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
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
DK4161942T3 (da) 2021-03-19 2023-10-23 Bachem Holding Ag Forbedret oligonukleotidsyntese der undertrykker depurinering
EP4316495A4 (en) * 2021-03-25 2025-12-31 Aptacure Therapeutics Ltd CONJUGATE OF NUCLEIC ACID MOLECULE HAVING A PROLONGED IN VIVO HALF-LIFE
AU2022246144A1 (en) 2021-03-26 2023-09-21 Mina Therapeutics Limited Tmem173 sarna compositions and methods of use
EP4314296A2 (en) 2021-03-29 2024-02-07 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
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
US20240402177A1 (en) 2021-04-06 2024-12-05 Bpgbio, Inc. Protein markers for estrogen receptor (er)-positive luminal a (la)-like and luminal b1 (lb1)-like breast cancer
US20240230651A1 (en) 2021-04-06 2024-07-11 Bpgbio, Inc. Protein markers for the prognosis of breast cancer progression
IL307531A (en) 2021-04-06 2023-12-01 Bpgbio Inc Protein markers for estrogen receptor (ER)-like and estrogen receptor (ER)-negative breast cancer
CN115197078B (zh) * 2021-04-08 2024-06-04 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
US20240197633A1 (en) * 2021-04-08 2024-06-20 Xiamen Sinopeg Biotech Co., Ltd. Pegylated lipid, liposome modified by the lipid, pharmaceutical composition containing the liposome, formulation and application thereof
WO2022214692A1 (en) 2021-04-09 2022-10-13 Bachem Holding Ag Pseudo solid phase protecting group and methods for the synthesis of oligonucleotides and oligonucleotide conjugates
CA3215367A1 (en) 2021-04-14 2022-10-20 Swapnil Kulkarni Fn3 domain-sirna conjugates and uses thereof
EP4323409A4 (en) 2021-04-14 2025-04-16 ARO Biotherapeutics Company CD71-BINDING FIBRONECTIN TYPE III DOMAINS
CA3217717A1 (en) 2021-04-23 2022-10-27 Ganna Bio, Inc. Glycan modified nucleic acids, methods of preparation, and therapeutic uses
CA3216106A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
EP4329734A4 (en) 2021-04-26 2025-04-02 Celanese EVA Performance Polymers LLC Implantable device for the delayed release of a macromolecular drug compound
JP2024515788A (ja) 2021-04-27 2024-04-10 ジェネレーション バイオ カンパニー 治療抗体を発現する非ウイルスｄｎａベクター及びその使用
US20240216535A1 (en) 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
US20240263177A1 (en) 2021-05-20 2024-08-08 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
EP4347822A2 (en) 2021-06-04 2024-04-10 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
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
BR112023026050A2 (pt) 2021-06-18 2024-03-05 Ionis Pharmaceuticals Inc Compostos e métodos para reduzir expressão de ifnar1
AU2022298603A1 (en) 2021-06-21 2024-02-01 Shanghai Junshi Biosciences Co., Ltd. Sirna inhibiting angptl3 gene expression, and use thereof
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
MX2023015523A (es) 2021-06-23 2024-03-11 Univ Massachusetts Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogenicos.
CA3224374A1 (en) 2021-06-29 2023-01-05 Flagship Pioneering Innovations V, Inc. Immune cells engineered to promote thanotransmission and uses thereof
EP4363574A1 (en) 2021-06-29 2024-05-08 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
EP4363580A1 (en) 2021-06-30 2024-05-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
JPWO2023282345A1 (OSRAM) 2021-07-08 2023-01-12
TW202317147A (zh) 2021-07-08 2023-05-01 日商日本新藥股份有限公司 析出抑制劑
US20240285547A1 (en) 2021-07-08 2024-08-29 Nippon Shinyaku Co., Ltd. Nephrotoxicity reducing agent
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
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
CN120989078A (zh) 2021-07-16 2025-11-21 瑞博泰克(山东)生物医药科技有限公司 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
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
MX2024000981A (es) 2021-07-21 2024-02-12 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de gen diana asociado con trastorno metabolico y sus metodos de uso.
AU2022316139A1 (en) 2021-07-23 2024-01-18 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
EP4380576A2 (en) * 2021-08-03 2024-06-12 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
CN117795074A (zh) 2021-08-03 2024-03-29 阿尔尼拉姆医药品有限公司 转甲状腺素蛋白（TTR）iRNA组合物和其使用方法
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
PE20241132A1 (es) 2021-08-04 2024-05-24 Alnylam Pharmaceuticals Inc Composiciones de arni y metodos para silenciar el angiotensinogeno (agt)
CA3227661A1 (en) 2021-08-05 2023-02-09 Weimin Wang 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
JP2024534766A (ja) 2021-08-13 2024-09-26 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 第ＸＩＩ因子（Ｆ１２）ｉＲＮＡ組成物およびその使用方法
EP4395790A2 (en) 2021-08-31 2024-07-10 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
CA3232387A1 (en) * 2021-09-13 2023-03-16 Ganna Bio, Inc. Glycan conjugate compositions and methods
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
CA3232420A1 (en) 2021-09-20 2023-03-23 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
US20230241224A1 (en) 2021-09-22 2023-08-03 Sanegene Bio Usa Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives and methods of use
MX2024003519A (es) 2021-09-24 2024-04-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) de proteina tau asociada a microtubulos (mapt) y sus metodos de uso.
WO2023056440A1 (en) 2021-10-01 2023-04-06 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
WO2023059695A1 (en) 2021-10-05 2023-04-13 Sanegene Bio Usa Inc. Polyhydroxylated cyclopentane derivatives and methods of use
WO2023059629A1 (en) 2021-10-05 2023-04-13 Amgen Inc. Compositions and methods for enhancing gene silencing activity of oligonucleotide compounds
EP4416287A1 (en) 2021-10-14 2024-08-21 Hemoshear Therapeutics, Inc. Compositions and methods of treating diseases associated with bile acid transporter
KR20240101590A9 (ko) 2021-10-15 2025-12-10 알닐람 파마슈티칼스 인코포레이티드 간외 전달 irna 조성물 및 이를 이용하는 방법
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
JP2024539097A (ja) 2021-10-22 2024-10-28 アムジェン インコーポレイテッド Ｇｐａｍ発現を阻害するためのｒｎａｉコンストラクト及びその使用方法
EP4423273A1 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
US20250019702A1 (en) 2021-11-10 2025-01-16 University Of Rochester Gata4-targeted therapeutics for treatment of cardiac hypertrophy
CA3237770A1 (en) 2021-11-10 2023-05-19 University Of Rochester Antisense oligonucleotides for modifying protein expression
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
WO2023114943A2 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
CN117756866A (zh) 2022-01-30 2024-03-26 大睿生物医药科技(上海)有限公司 含有n-乙酰半乳糖胺的靶向配体
CA3252536A1 (en) 2022-02-22 2023-08-31 Sanegene Bio Usa Inc. CARBOCYCLIC RIBONUCLEOTIDE DERIVATIVES MODIFIED AT THE 5' POSITION AND METHODS OF USE
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
TW202346589A (zh) 2022-03-10 2023-12-01 日商日本新藥股份有限公司 抗病毒反義寡聚物
CA3245737A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. PRIME-BOOST HETEROLOGICAL VACCINE COMPOSITIONS AND METHODS OF USE
TW202345867A (zh) 2022-03-16 2023-12-01 日商第一三共股份有限公司 抑制運鐵蛋白受體2表現的siRNA
WO2023176863A1 (ja) 2022-03-16 2023-09-21 第一三共株式会社 ＲＮＡｉ活性を有する化学修飾オリゴヌクレオチド
TW202400787A (zh) 2022-03-16 2024-01-01 美商安彼瑞可股份有限公司 改良siRNA生物可利用性之GalNAc組合物
US12258563B2 (en) 2022-03-28 2025-03-25 Empirico Inc. Modified oligonucleotides
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
WO2023220561A1 (en) 2022-05-09 2023-11-16 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
JP2025516680A (ja) 2022-05-13 2025-05-30 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 一本鎖ループオリゴヌクレオチド
US20230372508A1 (en) 2022-05-19 2023-11-23 Olix Us, Inc. Linkers coupling functional ligands to macromolecules
JP2025522453A (ja) 2022-06-14 2025-07-15 大睿生物 環状ホスホン酸エステル修飾ヌクレオチド
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
EP4544043A1 (en) 2022-06-22 2025-04-30 Flagship Pioneering Innovations VI, LLC Compositions of modified trems and uses thereof
WO2024001170A1 (en) * 2022-06-27 2024-01-04 Ractigen Therapeutics Small activating nucleic acid molecule and use thereof in treatment of hereditary angioedema
CN119421952A (zh) 2022-06-27 2025-02-11 大睿生物医药科技(上海)有限公司 抑制载脂蛋白C3表达的siRNA
AU2023307152A1 (en) 2022-07-11 2025-01-09 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
CN119677851A (zh) 2022-07-25 2025-03-21 安进公司 用于抑制FAM13A表达的RNAi构建体和方法
US20240052348A1 (en) 2022-08-05 2024-02-15 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
JP2025530741A (ja) 2022-08-29 2025-09-17 ユニヴァーシティ オヴ ロチェスター アンチセンスオリゴヌクレオチドベースの抗線維化治療剤
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
WO2024061842A1 (en) 2022-09-19 2024-03-28 Bachem Holding Ag Improved oligonucleotide synthesis
WO2024064858A2 (en) 2022-09-23 2024-03-28 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing mecp2 expression
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
AU2023358547A1 (en) 2022-10-14 2025-04-24 Sanegene Bio Usa Inc. Small interfering rna targeting c3 and uses thereof
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
AU2023387789A1 (en) * 2022-12-08 2025-06-26 Nanovation Therapeutics Inc. Lipid nanoparticles comprising elevated neutral lipid and a targeting moiety for targeted delivery of nucleic acid
US20240293555A1 (en) * 2022-12-12 2024-09-05 University Of Massachusetts Carbohydrate conjugates for the delivery of therapeutic oligonucleotides
EP4634385A2 (en) 2022-12-16 2025-10-22 Amgen Inc. Rnai constructs for inhibiting ttr expression and methods of use thereof
AU2023408479A1 (en) 2022-12-19 2025-05-01 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar)
CN120569477A (zh) 2022-12-19 2025-08-29 美国圣因生物股份有限公司 靶向cfb的小干扰rna及其用途
ES3030929T3 (en) 2022-12-19 2025-07-02 Arnatar Therapeutics Inc 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
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
CN120813691A (zh) 2023-02-09 2025-10-17 阿尔尼拉姆医药品有限公司 Reversir分子及其使用方法
WO2024175111A2 (en) 2023-02-24 2024-08-29 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
EP4678746A1 (en) 2023-03-10 2026-01-14 Suzhou Ribo Life Science Co., Ltd. Pharmaceutical composition and use thereof
WO2024206230A1 (en) * 2023-03-24 2024-10-03 Trustees Of Tufts College Tissue targeting lipids and lipid nanoparticles
CN121399143A (zh) * 2023-04-19 2026-01-23 普拉姆医药公司 脂质纳米颗粒(lnp)递送系统和制剂
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
US20250018045A1 (en) 2023-05-03 2025-01-16 Sanegene Bio Usa Inc. Lipid-based conjugates for systemic, central nervous system, peripheral nervous system, and ocular delivery
AU2024269205A1 (en) 2023-05-11 2025-12-11 Keymed Biosciences (Us) Inc. Dsrna molecules for regulating masp2 gene activity
CN118930593A (zh) 2023-05-11 2024-11-12 广东东阳光药业股份有限公司 一种新型的靶向化合物和核酸缀合物，及其用途
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2024238396A1 (en) 2023-05-12 2024-11-21 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
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
CN121335980A (zh) 2023-05-26 2026-01-13 阿达尔克斯制药有限公司 Sod1调节组合物及其使用方法
WO2024249889A1 (en) * 2023-05-31 2024-12-05 University Of Connecticut Genetic sequence/small molecule-carbohydrate conjugates for enhanced lung-specific targeting
WO2024259134A1 (en) 2023-06-13 2024-12-19 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar) for angiotensinogen
AU2024313301A1 (en) 2023-06-20 2025-12-11 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
US20250027089A1 (en) 2023-06-21 2025-01-23 Sanegene Bio Usa Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
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
WO2025007961A1 (zh) 2023-07-06 2025-01-09 大睿生物 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
GB202311334D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
GB202311324D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025024523A1 (en) 2023-07-24 2025-01-30 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
US20250051771A1 (en) 2023-07-24 2025-02-13 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
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
WO2025072713A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Polymyxins for delivery of agents to the kidney
WO2025072699A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Aminoglycosides 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
WO2025080572A1 (en) 2023-10-10 2025-04-17 Avanti Polar Lipids, Llc Liver asialoglycoprotein receptor targeted lipid and uses thereof
WO2025082632A1 (en) 2023-10-16 2025-04-24 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
AU2024369609A1 (en) 2023-10-31 2025-12-11 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
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
TW202523695A (zh) 2023-11-22 2025-06-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於治療非酒精性脂肪性肝病之方法及組成物
WO2025155911A1 (en) 2024-01-18 2025-07-24 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hurler syndrome
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
EP4665854A1 (en) 2024-01-29 2025-12-24 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

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 (OSRAM) 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
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
DE3687030T2 (de) 1985-03-15 1993-03-11 Eugene Stirchak Stereoregulare polynukleotiden bindende polymere.
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
ES2051700T3 (es) * 1986-03-27 1994-07-01 Sumitomo Pharma Un proceso para la preparacion de un compuesto beta-lactama.
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
ES2054729T3 (es) * 1987-05-01 1994-08-16 Fujisawa Pharmaceutical Co Derivados de pirrolidina.
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 (OSRAM) 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
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
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
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
CA2088258C (en) 1990-07-27 2004-09-14 Phillip Dan Cook Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
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
DE552178T1 (de) 1990-10-12 1994-02-03 Max Planck Gesellschaft Abgeänderte ribozyme.
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.
WO1993025673A1 (en) 1992-06-04 1993-12-23 The Regents Of The University Of California In vivo gene therapy with intron-free sequence of interest
CA2135313A1 (en) 1992-06-18 1994-01-06 Theodore Choi Methods for producing transgenic non-human animals harboring a yeast artificial chromosome
EP1251170A3 (en) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of NF-kappaB dependent 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
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
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
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
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
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
EP0833613A1 (en) 1995-05-26 1998-04-08 Somatix Therapy Corporation Delivery vehicles comprising stable lipid/nucleic acid complexes
JP4335310B2 (ja) 1995-06-07 2009-09-30 ザ ユニバーシティ オブ ブリティッシュ コロンビア 疎水性脂質−核酸複合中間体を通して調製される脂質−核酸粒子、及び遺伝子移送のための使用
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
US6337390B1 (en) * 1996-05-16 2002-01-08 Nissan Food Products Co., Ltd. Compounds comprising sulfated nonulonic acid having antiviral activity
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
AU733310C (en) 1997-05-14 2001-11-29 University Of British Columbia, The 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
WO2000044914A1 (en) 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
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
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) * 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
NZ522045A (en) * 2000-03-30 2007-05-31 Whitehead Biomedical Inst RNA sequence-specific mediators of RNA interference
IL155991A0 (en) 2000-12-01 2003-12-23 Max Planck Gesellschaft Rna interference mediating small rna molecules
CA2431839A1 (en) 2000-12-01 2002-06-06 Paul O. P. Ts'o Conjugates of glycosylated/galactosylated peptide
US7875612B2 (en) 2001-04-24 2011-01-25 Purdue Research Foundation Folate mimetics and folate-receptor binding conjugates thereof
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
US7109165B2 (en) * 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
EP3231445A1 (en) 2001-05-18 2017-10-18 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20030148928A1 (en) * 2001-07-20 2003-08-07 Leonid Beigelman Enzymatic nucleic acid peptide conjugates
EP1543019A2 (en) 2002-09-11 2005-06-22 Santaris Pharma A/S Modified pna molecules
US8110674B2 (en) 2003-03-07 2012-02-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
EP1608735A4 (en) 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES
EP2660322A3 (en) 2003-04-17 2013-11-13 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
US7803397B2 (en) 2003-09-15 2010-09-28 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
EP1768998A2 (en) * 2004-04-27 2007-04-04 Alnylam Pharmaceuticals Inc. Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
US20080213177A1 (en) * 2004-05-24 2008-09-04 Thomas William Rademacher Nanoparticles Comprising Rna Ligands
JP5192234B2 (ja) * 2004-08-10 2013-05-08 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 化学修飾オリゴヌクレオチド
US20060148740A1 (en) * 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
JP5635412B2 (ja) * 2007-12-04 2014-12-03 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． 標的化脂質
US20120101148A1 (en) 2009-01-29 2012-04-26 Alnylam Pharmaceuticals, Inc. lipid formulation
DE102009039097B3 (de) 2009-08-27 2010-11-25 Siemens Aktiengesellschaft Verfahren zum Übertragen von Daten in einem Sensornetzwerk, Sensorknoten und Zentral-Rechner
US8315599B2 (en) 2010-07-09 2012-11-20 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
US11405999B2 (en) 2018-10-18 2022-08-02 Signify Holding B.V. Determining light settings and/or daylight blocker settings based on data signal quality
WO2020163883A1 (en) 2019-02-04 2020-08-13 Detnet South Africa (Pty) Ltd Boost pump
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

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Biessen E A et al.; Journal of Medicinal Chemistry. 1995; 38(11): 1846-1852. *
Rensen Patrick C. N. et al. Journal of Medicinal Chemistry. 2004; 47(23): 5798-5808. *
Sliedregt Leo A. J. M. et al.; Journal of Medicinal Chemistry, 1999; 42(4):609-618. *
Zimmermann et al. Nature .2006; 441(7089):111-114. *

Also Published As

Publication number Publication date
US20150119445A1 (en) 2015-04-30
US10806791B2 (en) 2020-10-20
JP2014139232A (ja) 2014-07-31
WO2009082607A8 (en) 2010-01-28
EP3156077B1 (en) 2022-03-09
WO2009073809A2 (en) 2009-06-11
CA2708153C (en) 2017-09-26
US20090247608A1 (en) 2009-10-01
EP4321177A3 (en) 2024-05-08
CA2910760A1 (en) 2009-07-02
US11666653B2 (en) 2023-06-06
CN102006890A (zh) 2011-04-06
JP2022190116A (ja) 2022-12-22
US20130178512A1 (en) 2013-07-11
US20190099493A1 (en) 2019-04-04
JP2018029599A (ja) 2018-03-01
JP2021020928A (ja) 2021-02-18
CA3043911A1 (en) 2009-07-02
JP2019023241A (ja) 2019-02-14
EP4321177A2 (en) 2024-02-14
EP4074344A1 (en) 2022-10-19
US8828956B2 (en) 2014-09-09
JP7590395B2 (ja) 2024-11-26
CA3146103A1 (en) 2009-06-11
WO2009073809A3 (en) 2009-12-30
CA2708173A1 (en) 2009-07-02
CA2930393A1 (en) 2009-06-11
US20150011615A1 (en) 2015-01-08
JP2017002082A (ja) 2017-01-05
EP3156077A1 (en) 2017-04-19
JP2023010881A (ja) 2023-01-20
US20220143189A1 (en) 2022-05-12
CA2910760C (en) 2019-07-09
CA2930393C (en) 2022-11-29
CA2708153A1 (en) 2009-06-11
EP3705125B1 (en) 2023-07-05
JP6850827B2 (ja) 2021-03-31
US9814777B2 (en) 2017-11-14
US9370581B2 (en) 2016-06-21
US11110174B2 (en) 2021-09-07
JP2015025007A (ja) 2015-02-05
JP2021080299A (ja) 2021-05-27
CA2708173C (en) 2016-02-02
JP5843914B2 (ja) 2016-01-13
WO2009082607A2 (en) 2009-07-02
US20190184018A1 (en) 2019-06-20
US20200297853A1 (en) 2020-09-24
EP4223299A3 (en) 2023-08-30
EP4223299A2 (en) 2023-08-09
EP2231195B1 (en) 2017-03-29
AU2008340355A1 (en) 2009-07-02
US20160375137A9 (en) 2016-12-29
EP2231195A2 (en) 2010-09-29
US9352048B2 (en) 2016-05-31
JP2019147846A (ja) 2019-09-05
US9867882B2 (en) 2018-01-16
US20220008541A1 (en) 2022-01-13
US8106022B2 (en) 2012-01-31
US9370582B2 (en) 2016-06-21
US20090239814A1 (en) 2009-09-24
US12059468B2 (en) 2024-08-13
AU2008333811A1 (en) 2009-06-11
JP5519523B2 (ja) 2014-06-11
US20180326070A1 (en) 2018-11-15
JP2011505426A (ja) 2011-02-24
US20150119444A1 (en) 2015-04-30
JP6215284B2 (ja) 2017-10-18
US20120136042A1 (en) 2012-05-31
JP2011505425A (ja) 2011-02-24
US8450467B2 (en) 2013-05-28
AU2008333811B2 (en) 2014-05-01
US20140179761A1 (en) 2014-06-26
JP6542853B2 (ja) 2019-07-10
US20160051691A1 (en) 2016-02-25
JP2016034974A (ja) 2016-03-17
EP3705125A1 (en) 2020-09-09
EP2229186A2 (en) 2010-09-22
JP5635412B2 (ja) 2014-12-03
WO2009082607A3 (en) 2009-12-17
EP3141265A1 (en) 2017-03-15

Similar Documents

Publication Publication Date Title
AU2008340355B2 (en) Targeting lipids
AU2021201075B2 (en) Novel lipids and compositions for the delivery of therapeutics
AU2020200082A1 (en) Targeting lipids
AU2022201011A1 (en) Targeting lipids
HK40075255A (en) Targeting lipids

Legal Events

Date Code Title Description
PC1 Assignment before grant (sect. 113)

Owner name: TEKMIRA PHARMACEUTICALS CORPORATION

Free format text: FORMER APPLICANT(S): ALNYLAM PHARMACEUTICALS, INC.

FGA Letters patent sealed or granted (standard patent)