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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (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)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Botany (AREA)
  • Emergency Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Endocrinology (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)
  • Steroid Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (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 (https=)
EP (8) EP3141265A1 (https=)
JP (13) JP5635412B2 (https=)
CN (1) CN102006890A (https=)
AU (2) AU2008340355B2 (https=)
CA (6) CA3146103A1 (https=)
WO (2) WO2009073809A2 (https=)

Families Citing this family (713)

* 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
ES2702942T3 (es) * 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
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
EP2066687A4 (en) 2006-09-21 2010-12-08 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF HAMP GENE
WO2009082606A2 (en) * 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Folate conjugates
WO2009073809A2 (en) * 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2721183C (en) * 2008-04-11 2019-07-16 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
KR101773551B1 (ko) 2008-10-15 2017-08-31 아이오니스 파마수티컬즈, 인코포레이티드 인자 １１ 발현의 조정
MX360460B (es) 2008-10-20 2018-11-05 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de transtiretina.
CN111808084A (zh) * 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
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
EP3243504A1 (en) * 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
AU2010221419B2 (en) 2009-03-02 2015-10-01 Alnylam Pharmaceuticals, Inc. Nucleic acid chemical modifications
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
JP5894913B2 (ja) * 2009-06-15 2016-03-30 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． Ｐｃｓｋ９遺伝子を標的とする、脂質で製剤化されたｄｓｒｎａ
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
WO2011102906A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion delivery system
WO2011102905A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion diagnostic delivery system
WO2011102904A1 (en) * 2010-02-19 2011-08-25 Robert Shorr Imageable lipid-oil-water nanoemulsion therapeutic delivery system
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
EP3560503B1 (en) 2010-03-24 2021-11-17 Phio Pharmaceuticals Corp. Rna interference in dermal and fibrotic indications
EP2555778A4 (en) 2010-04-06 2014-05-21 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING CD274 / PD-L1 GENE EXPRESSION
US9145556B2 (en) 2010-04-13 2015-09-29 Life Technologies Corporation Compositions and methods for inhibition of nucleic acids function
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
CA2800693A1 (en) * 2010-05-28 2011-12-01 Purdue Research Foundation Delivery of therapeutic agents to inflamed tissues using folate-targeted agents
EP2933333B1 (en) 2010-07-08 2018-02-21 Bonac Corporation Single-strand nucleic acid molecule for controlling gene expression
AU2011282977A1 (en) * 2010-07-28 2013-02-21 Smartcells, Inc. Drug-ligand conjugates, synthesis thereof, and intermediates thereto
EP2639307B1 (en) 2010-08-03 2015-03-04 Bonac Corporation Single-stranded nucleic acid molecule having nitrogen-containing alicyclic skeleton
US9243246B2 (en) * 2010-08-24 2016-01-26 Sirna Therapeutics, Inc. Single-stranded RNAi agents containing an internal, non-nucleic acid spacer
CA2812046A1 (en) * 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
CN101974152B (zh) * 2010-09-28 2012-07-11 山东大学 一种类脂-阳离子聚合物及其制备方法
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
CA2817960C (en) 2010-11-17 2020-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
AU2011343664B2 (en) 2010-12-17 2015-10-08 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for siRNA
CA3131967A1 (en) 2010-12-29 2012-07-05 F. Hoffman-La Roche Ag Small molecule conjugates for intracellular delivery of nucleic acids
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
PH12013501969B1 (en) 2011-03-29 2018-08-31 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of tmprss6 gene
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
EP2723861A4 (en) * 2011-06-21 2014-12-10 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING HEPCIDINE ANTIMICROBIAL PEPTIDE (HAMP) OR THE ASSOCIATED GENE EXPRESSION
EP3388068A1 (en) 2011-06-21 2018-10-17 Alnylam Pharmaceuticals, Inc. Composition and methods for inhibition of expression of protein c (proc) genes
EP2723758B1 (en) * 2011-06-21 2018-06-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof
MX390699B (es) 2011-06-21 2025-03-21 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3).
WO2012178033A2 (en) 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
EA024762B9 (ru) 2011-06-30 2017-04-28 Эрроухэд Рисерч Корпорейшн Композиции и способы ингибирования экспрессии генов вируса гепатита в
WO2013019874A1 (en) * 2011-08-01 2013-02-07 Mbc Pharma, Inc. Vitamin b6 derivatives of nucleotides, acyclonucleotides and acyclonucleoside phosphonates
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP3456317B1 (en) 2011-09-27 2025-09-24 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
EP2594575A1 (en) * 2011-11-15 2013-05-22 Université de Strasbourg Mannosylated compounds useful for the prevention and the treatment of infectious diseases
EP3730618A1 (en) * 2011-11-18 2020-10-28 Alnylam Pharmaceuticals, Inc. Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
SI3366775T2 (sl) * 2011-11-18 2025-12-31 Alnylam Pharmaceuticals, Inc. Modificirana sredstva RNAI
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
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
TW201808342A (zh) * 2012-05-02 2018-03-16 喜納製藥公司 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
WO2013180038A1 (ja) 2012-05-26 2013-12-05 株式会社ボナック デリバリー機能を有する遺伝子発現制御用の一本鎖核酸分子
EA032345B1 (ru) 2012-06-01 2019-05-31 Берг Ллк Способ лечения рака с использованием кофермента q10
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
EP2858679B2 (en) 2012-06-08 2024-06-05 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
EP2861256B1 (en) 2012-06-15 2019-10-23 The Brigham and Women's Hospital, Inc. Compositions for treating cancer and methods for making the same
AU2013299717B2 (en) * 2012-08-06 2018-06-28 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated RNA agents and process for their preparation
CN102824515B (zh) * 2012-09-18 2015-05-20 淄博齐鼎立专利信息咨询有限公司 热毒清片在制备抑制a-204细胞增殖药物中的应用
AU2013336582A1 (en) 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to cell types
CN104837996A (zh) * 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
US9506897B2 (en) 2012-11-16 2016-11-29 Agilent Technologies, Inc. Methods and compositions for improved ion-exchange chromatography
IL292159B1 (en) * 2012-12-05 2026-04-01 Alnylam Pharmaceuticals Inc PCSK9 iRNA compositions and methods of using them
US20150337318A1 (en) * 2013-01-11 2015-11-26 Phaserx Inc. Rna targeted to c-met
AU2014211406B2 (en) * 2013-01-30 2019-07-18 Roche Innovation Center Copenhagen A/S LNA oligonucleotide carbohydrate conjugates
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの精製方法
BR112015022855A2 (pt) 2013-03-14 2017-11-07 Shire Human Genetic Therapies composições e método para produção de um anticorpo in vitro
RS57739B1 (sr) 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
IL288931B2 (en) * 2013-03-14 2025-05-01 Alnylam Pharmaceuticals Inc Compositions comprising a complementary portion of C5 IRNA and methods of using them
EP3388834B1 (en) 2013-03-15 2020-04-15 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
DK2970968T3 (en) 2013-03-15 2018-03-05 Miragen Therapeutics Inc CONNECTED BICYCLIC Nucleosides
NZ713868A (en) 2013-04-08 2021-12-24 Berg Llc Treatment of cancer using coenzyme q10 combination therapies
SG10201803157XA (en) 2013-05-01 2018-05-30 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-122
US9506030B2 (en) 2013-05-01 2016-11-29 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
WO2014182661A2 (en) 2013-05-06 2014-11-13 Alnylam Pharmaceuticals, Inc Dosages and methods for delivering lipid formulated nucleic acid molecules
RU2535052C1 (ru) * 2013-05-21 2014-12-10 Владимир Николаевич Иванов Фармацевтическая композиция, содержащая производные лизина, пролина и тритерпеновой кислоты для лечения и профилактики вирусных инфекций, вызываемых рнк и днк-содержащими вирусами, такими как: грипп, герпес, опоясывающий лишай, папиллома человека, аденоновирусы, а также бактериальных инфекций, вызываемых грам-положительными и грам-отрицательными микроорганизмами
EA038792B1 (ru) * 2013-05-22 2021-10-20 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ RNAi Serpina1 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
KR102234623B1 (ko) 2013-05-22 2021-04-02 알닐람 파마슈티칼스 인코포레이티드 Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법
EP3564374A1 (en) 2013-06-21 2019-11-06 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
HUE048738T2 (hu) 2013-06-27 2020-08-28 Roche Innovation Ct Copenhagen As Antiszensz oligomerek és konjugátumok, amelyek a PCK9-t célozzák meg
BR112016000160B1 (pt) * 2013-07-05 2021-06-08 Bioneer Corporation estrutura de oligonucleotídeo tipo nanopartícula melhorada tendo alta eficiência e método para a preparação da mesma
WO2015006740A2 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
KR102370843B1 (ko) 2013-09-04 2022-03-04 버그 엘엘씨 코엔자임 q10의 연속주입에 의한 암치료 방법
CA2924343C (en) 2013-09-17 2025-09-16 Bolt Threads, Inc. PROCESSES AND COMPOSITIONS FOR SYNTHESIZING IMPROVED SILK FIBERS
WO2015042447A1 (en) 2013-09-20 2015-03-26 Isis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
WO2015042564A1 (en) 2013-09-23 2015-03-26 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing transthyretin (ttr) associated diseases
CA2925107A1 (en) * 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
LT3052628T (lt) * 2013-10-04 2020-09-10 Alnylam Pharmaceuticals, Inc. Kompozicijos ir būdai alas1 geno raiškai nuslopinti
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
CN106413811A (zh) 2013-10-22 2017-02-15 夏尔人类遗传性治疗公司 精氨基琥珀酸合成酶缺乏症的mrna疗法
JP6506749B2 (ja) 2013-10-22 2019-04-24 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 フェニルケトン尿症のためのｍＲＮＡ療法
US9994846B2 (en) 2013-10-25 2018-06-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
MX2016005855A (es) * 2013-11-14 2016-07-13 Roche Innovation Ct Copenhagen As Compuestos de conjugados antisentido de apolipoproteina b (apob).
CN106061488B (zh) 2013-12-02 2021-04-09 菲奥医药公司 癌症的免疫治疗
SG10201804960RA (en) * 2013-12-12 2018-07-30 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
WO2015099122A1 (ja) 2013-12-26 2015-07-02 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
CN106068324B (zh) 2013-12-27 2020-12-29 株式会社博纳克 控制基因表达的人工匹配型miRNA及其用途
US10119136B2 (en) 2014-01-09 2018-11-06 Alnylam Pharmaceuticals, Inc. RNAi agents modified at the 4′-C position
ES2742102T3 (es) * 2014-01-15 2020-02-13 Baseclick Gmbh Moléculas de ácido nucleico modificadas con sacárido
CA2935426C (en) * 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target
CN113057959B (zh) 2014-02-11 2024-07-16 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
EP3119888B1 (en) 2014-03-19 2021-07-28 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
HUE050704T2 (hu) 2014-04-01 2020-12-28 Biogen Ma Inc Összetételek a SOD-1 expressziójának modulálására
BR112016022499A2 (pt) 2014-04-03 2017-08-15 Invictus Oncology Pvt Ltd Produtos terapêuticos combinatórios supramoleculares
US9850269B2 (en) 2014-04-25 2017-12-26 Translate Bio, Inc. Methods for purification of messenger RNA
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
WO2015168172A1 (en) 2014-04-28 2015-11-05 Isis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
US9382540B2 (en) 2014-05-01 2016-07-05 Isis Pharmaceuticals, Inc Compositions and methods for modulating angiopoietin-like 3 expression
HUE052709T2 (hu) 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
US10098959B2 (en) 2014-05-01 2018-10-16 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
BR112016022742B1 (pt) 2014-05-01 2022-06-14 Ionis Pharmaceuticals, Inc Composto químico, composição compreendendo o composto e uso dos mesmos
EA036496B1 (ru) * 2014-05-01 2020-11-17 Ионис Фармасьютикалз, Инк. Конъюгированные олигонуклеотиды для модулирования экспрессии фактора комплемента b
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
WO2015179693A1 (en) * 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
AU2015264038B2 (en) 2014-05-22 2021-02-11 Alnylam Pharmaceuticals, Inc. Angiotensinogen (AGT) iRNA compositions and methods of use thereof
JP6557722B2 (ja) 2014-05-30 2019-08-07 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 核酸の送達のための生分解性脂質
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
KR102559979B1 (ko) 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
KR20170033439A (ko) 2014-08-07 2017-03-24 레굴루스 테라퓨틱스 인크 대사 장애에 대한 마이크로rna의 표적화
CN113599539A (zh) 2014-08-29 2021-11-05 阿尔尼拉姆医药品有限公司 治疗甲状腺素运载蛋白（ttr）介导的淀粉样变性的方法
WO2016030863A1 (en) 2014-08-29 2016-03-03 Glaxosmithkline Intellectual Property Development Limited Compounds and methods for treating viral infections
US10900039B2 (en) 2014-09-05 2021-01-26 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
CN106795200B (zh) 2014-10-10 2020-06-19 豪夫迈·罗氏有限公司 Galnac亚磷酰胺、其核酸缀合物及其用途
TW202503057A (zh) 2014-10-10 2025-01-16 美商艾爾妮蘭製藥公司 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法
WO2016057693A1 (en) * 2014-10-10 2016-04-14 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhalation delivery of conjugated oligonucleotide
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3212794B1 (en) 2014-10-30 2021-04-07 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
KR102545316B1 (ko) 2014-11-10 2023-06-22 알닐람 파마슈티칼스 인코포레이티드 Ｂ형 간염 바이러스(ｈｂｖ) ｉｒｎａ 조성물 및 그의 이용 방법
WO2016077567A1 (en) * 2014-11-12 2016-05-19 Tumorend, Llc Compositions, methods and treatments for inhibiting cell adhesion and virus binding and penetration
CA2964979A1 (en) 2014-11-14 2016-05-19 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
JP6767976B2 (ja) 2014-12-05 2020-10-14 トランスレイト バイオ， インコーポレイテッド 関節疾患の治療のためのメッセンジャーｒｎａ治療法
US10539566B2 (en) 2014-12-08 2020-01-21 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
JP2018504380A (ja) 2014-12-18 2018-02-15 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． Ｒｅｖｅｒｓｉｒ（商標）化合物
WO2016105499A1 (en) 2014-12-23 2016-06-30 Intellectual Property Associates, Llc Methods and formulations for transdermal administration
KR20170098914A (ko) 2014-12-27 2017-08-30 가부시키가이샤 보낙 유전자 발현 제어를 위한 천연형 miRNA 및 그의 용도
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
US20180002692A1 (en) * 2015-01-30 2018-01-04 Anushree Chatterjee Sequence Specific and Organism Specific Antimicrobials and Related Materials and Methods
CA2976445A1 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
CN104804463B (zh) * 2015-03-10 2016-08-24 西安交通大学第一附属医院 用于靶向肿瘤组织的近红外荧光染色剂及制备方法和应用
WO2016149508A1 (en) 2015-03-19 2016-09-22 Shire Human Genetic Therapies, Inc. Mrna therapy for pompe disease
WO2016158809A1 (ja) 2015-03-27 2016-10-06 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
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
MX2017012426A (es) 2015-04-03 2018-01-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tmprss6.
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
CN115927335A (zh) 2015-04-13 2023-04-07 阿尔尼拉姆医药品有限公司 类血管生成素3（ANGPTL3）iRNA组合物及其使用方法
US10407678B2 (en) 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2016196111A1 (en) 2015-06-01 2016-12-08 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
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. 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
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
GB201511459D0 (en) * 2015-06-30 2015-08-12 Cell Therapy Ltd Sirna microbubble
EP3862005A1 (en) 2015-07-06 2021-08-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
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
MX2018000412A (es) 2015-07-10 2018-03-14 Ionis Pharmaceuticals Inc Moduladores de diaciglicerol aciltransferasa 2 (dgat2).
CA3205381A1 (en) 2015-07-17 2017-01-26 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
KR20180031025A (ko) 2015-07-31 2018-03-27 아크투루스 쎄라퓨틱스, 인크. 약물 전달을 위한 다중 리간드 제제
PT3329002T (pt) 2015-07-31 2021-01-12 Alnylam Pharmaceuticals Inc Composições de iarn de transtirretina (ttr) e métodos de seu uso para tratamento ou prevenção de doenças associadas à ttr
AU2016306275A1 (en) 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
WO2017030973A1 (en) 2015-08-14 2017-02-23 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
CN108348541A (zh) 2015-08-25 2018-07-31 阿尔尼拉姆医药品有限公司 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物
CA2996873A1 (en) 2015-09-02 2017-03-09 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (pd-l1) irna compositions and methods of use thereof
CN108513588A (zh) 2015-09-24 2018-09-07 Ionis制药公司 Kras表达的调节剂
EP4285912A3 (en) 2015-09-25 2024-07-10 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
CN113817735A (zh) 2015-10-08 2021-12-21 Ionis制药公司 用于调节血管紧张素原表达的化合物和方法
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
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
JP6997704B2 (ja) 2015-10-14 2022-02-04 トランスレイト バイオ， インコーポレイテッド 生産性向上のためのｒｎａ関連酵素の修飾
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
KR20250078597A (ko) 2015-11-06 2025-06-02 아이오니스 파마수티컬즈, 인코포레이티드 아포리포프로테인 (a) 발현 조정
PL4119569T3 (pl) 2015-11-06 2024-11-18 Ionis Pharmaceuticals, Inc. Sprzężone związki antysensowne do zastosowania w terapii
EP3389670A4 (en) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. METHODS OF TREATING BREAST CANCER
KR20180095843A (ko) 2015-12-07 2018-08-28 젠자임 코포레이션 Serpinc1-연관 장애의 치료를 위한 방법 및 조성물
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
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
JP7049249B2 (ja) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 中枢神経系疾患の処置のための組成物および方法
JP2019500345A (ja) 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 肝臓病の処置のための組成物および方法
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
ES2903394T3 (es) 2015-12-14 2022-04-01 Cold Spring Harbor Laboratory Composiciones para el tratamiento de la retinitis pigmentosa 13
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
AU2016381174A1 (en) 2015-12-31 2018-05-31 Ionis Pharmaceuticals, Inc. Methods for reducing Ataxin-2 expression
CA3006599A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
AU2017213404A1 (en) 2016-01-29 2018-09-20 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
US10478503B2 (en) 2016-01-31 2019-11-19 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
EP3429690A4 (en) 2016-03-16 2019-10-23 Ionis Pharmaceuticals, Inc. METHOD FOR MODULATING KEAP1
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
WO2017173334A1 (en) 2016-04-01 2017-10-05 Checkmate Pharmaceuticals, Inc. Fc receptor-mediated drug delivery
EP3825400B1 (en) 2016-04-08 2024-12-25 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
CA3020585A1 (en) 2016-04-11 2017-10-19 Arbutus Biopharma Corporation Targeted nucleic acid conjugate compositions
WO2017178656A1 (en) 2016-04-14 2017-10-19 Roche Innovation Center Copenhagen A/S TRITYL-MONO-GalNAc COMPOUNDS AND THEIR USE
WO2017184689A1 (en) * 2016-04-19 2017-10-26 Alnylam Pharmaceuticals, Inc. High density lipoprotein binding protein (hdlbp/vigilin) irna compositions and methods of use thereof
EP3445853A1 (en) 2016-04-19 2019-02-27 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
US11390867B2 (en) 2016-04-29 2022-07-19 Nanyang Technological University G-quadruplex-containing antisense oligonucleotides
US11040108B2 (en) 2016-04-29 2021-06-22 Brandeis University Amino acid- and peptide-steroid conjugates and use thereof
CA3022319A1 (en) 2016-05-06 2017-11-09 Tod M. Woolf Improved methods for genome editing with and without programmable nucleases
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
EP3469074B1 (en) 2016-06-13 2020-12-09 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
AU2017286811A1 (en) 2016-06-17 2018-11-22 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
TWI746590B (zh) * 2016-06-30 2021-11-21 日商協和麒麟股份有限公司 核酸複合體
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
WO2018013525A1 (en) 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
EP3484524B1 (en) 2016-07-15 2022-11-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
US10781175B2 (en) 2016-07-15 2020-09-22 Am Chemicals Llc Solid supports and phosphoramidite building blocks for oligonucleotide conjugates
JOP20190015A1 (ar) 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
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
SG11201901841TA (en) 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
KR101933767B1 (ko) 2016-09-05 2018-12-28 (의료)길의료재단 발기부전 환자를 위한 콘돔
US11400161B2 (en) 2016-10-06 2022-08-02 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
CA3043768A1 (en) 2016-11-29 2018-06-07 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
CN110225770B (zh) 2016-12-14 2022-04-26 杨森生物科技公司 结合cd8a的纤连蛋白iii型结构域
EP3554535A4 (en) 2016-12-14 2020-10-21 Janssen Biotech, Inc. PD-L1 BINDING FIBRONECTIN TYPE III DOMAINS
EP3554561B1 (en) 2016-12-14 2023-06-28 Janssen Biotech, Inc. Cd137 binding fibronectin type iii domains
WO2018112320A1 (en) 2016-12-16 2018-06-21 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
WO2018127927A1 (en) 2017-01-09 2018-07-12 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
CN110268060B (zh) 2017-01-10 2024-07-26 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
CN106986942B (zh) * 2017-02-28 2020-12-25 国药中生生物技术研究院有限公司 一种包含蝙蝠肝炎病毒核心蛋白的重组融合蛋白及其制备方法和应用
EP3594346A4 (en) 2017-03-10 2020-12-16 National Center For Child Health And Development ANTISENS OLIGONUCLEOTIDE AND COMPOSITION FOR THE PREVENTION OR TREATMENT OF TYPE IA GLYCOGEN STORAGE DISEASE
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
EP3385272A1 (en) * 2017-04-05 2018-10-10 Silence Therapeutics GmbH Further novel oligonucleotide-ligand conjugates
DK3607069T3 (da) 2017-04-05 2022-11-21 Silence Therapeutics Gmbh Produkter og sammensætninger
WO2018185253A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
MX2019012280A (es) 2017-04-11 2020-01-23 Arbutus Biopharma Corp Composiciones dirigidas.
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
CA3059446A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
US11324820B2 (en) 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
US12544344B2 (en) 2017-04-19 2026-02-10 Phio Pharmaceuticals Corp. Topical delivery of nucleic acid compounds
CN110799492B (zh) 2017-04-28 2023-06-27 爱康泰生治疗公司 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
US11273222B2 (en) * 2017-05-26 2022-03-15 National Cerebral And Cardiovascular Center Antisense nucleic acid targeting PCSK9
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
US10195286B2 (en) * 2017-07-04 2019-02-05 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
TW202434265A (zh) 2017-07-10 2024-09-01 美商健贊公司 於患有血友病之個體中治療出血事件之方法及組成物
EP3652317A1 (en) 2017-07-13 2020-05-20 Alnylam Pharmaceuticals, Inc. Lactate dehydrogenase a (ldha) irna compositions and methods of use thereof
CA3069451A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals, Inc. Methods for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
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
TW201920668A (zh) * 2017-08-02 2019-06-01 日商協和醱酵麒麟有限公司 核酸複合體
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
EP4501322A3 (en) 2017-08-17 2025-04-16 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036613A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. MODULATION OF THE NOTCH SIGNALING PATHWAY FOR THE TREATMENT OF RESPIRATORY DISORDERS
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
US20200208152A1 (en) * 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
WO2019048632A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE
CN110168093B (zh) * 2017-09-12 2023-08-15 中科蓝华(广州)生物医药技术有限公司 一种转染细胞内寄生虫的试剂盒及其应用
TN2020000038A1 (en) 2017-09-14 2021-10-04 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
EA202090720A1 (ru) * 2017-09-14 2020-07-07 Янссен Байофарма, Инк. ПРОИЗВОДНЫЕ GalNAc
BR112020005230A2 (pt) 2017-09-19 2020-09-24 Alnylam Pharmaceuticals, Inc. composições e métodos para o tratamento da amiloidose mediada por transtiretina (ttr)
HUE070436T2 (hu) 2017-10-23 2025-06-28 Stoke Therapeutics Inc Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére
SG11202002940QA (en) 2017-11-01 2020-04-29 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
CN111527206A (zh) 2017-11-13 2020-08-11 赛伦斯治疗有限责任公司 用于抑制靶标基因表达的包含二硫代磷酸酯键的核酸
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
DK3719128T3 (da) 2017-12-01 2025-03-03 Suzhou Ribo Life Science Co Ltd Dobbelt-strenget oligonukleotid, sammensætning og konjugat bestaende dobbelt-strenget oligonukleotid, fremgangsmåde herfor og anvendelse heraf
EP3719126B1 (en) 2017-12-01 2025-01-01 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
EP3719127A4 (en) 2017-12-01 2021-10-20 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE
CN110944675B9 (zh) 2017-12-01 2024-08-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CA3083970A1 (en) 2017-12-01 2019-06-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
UY38003A (es) 2017-12-12 2019-06-28 Amgen Inc Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas
EP3728593A1 (en) 2017-12-18 2020-10-28 Alnylam Pharmaceuticals, Inc. High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
EP3728281A1 (en) 2017-12-21 2020-10-28 Alnylam Pharmaceuticals Inc. Chirally-enriched double-stranded rna agents
US11459564B2 (en) 2017-12-21 2022-10-04 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
WO2019127004A1 (zh) 2017-12-26 2019-07-04 广州市锐博生物科技有限公司 修饰的寡核苷酸和可用于合成修饰的寡核苷酸的化合物
AU2018394875B2 (en) 2017-12-29 2023-08-03 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
WO2019130319A1 (en) 2018-01-01 2019-07-04 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
WO2019140102A1 (en) * 2018-01-10 2019-07-18 Translate Bio Ma, Inc. Compositions and methods for facilitating delivery of synthetic nucleic acids to cells
AU2019206731A1 (en) 2018-01-15 2020-07-30 Ionis Pharmaceuticals, Inc. Modulators of DNM2 expression
WO2019147743A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
WO2019157531A1 (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
PH12020551219A1 (en) 2018-02-14 2021-04-19 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
US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
KR102398295B1 (ko) 2018-03-09 2022-05-17 다이이찌 산쿄 가부시키가이샤 당원병 Ia형 치료약
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
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
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
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
WO2019183440A1 (en) 2018-03-22 2019-09-26 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
EP3550021A1 (en) * 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions for inhibiting expression of a target gene
KR102857148B1 (ko) 2018-04-05 2025-09-11 사일런스 테라퓨틱스 게엠베하 안티센스 가닥의 5' 단부에 비닐포스포네이트를 갖는 siRNA
EP3775209A1 (en) * 2018-04-05 2021-02-17 Silence Therapeutics GmbH Sirnas with at least two ligands at different ends
BR112020020479A2 (pt) 2018-04-09 2021-01-12 Checkmate Pharmaceuticals Empacotamento de oligonucleotídeos em partículas similares a vírus
SG11202008660TA (en) 2018-04-11 2020-10-29 Ionis Pharmaceuticals Inc Modulators of ezh2 expression
EP3775211B1 (en) 2018-04-12 2023-04-05 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
CA3099280A1 (en) 2018-05-04 2019-11-07 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.
US12005120B2 (en) 2018-05-08 2024-06-11 Regulus Therapeutics Inc. Galnac conjugated modified oligonucleotides as miR-122 inhibitor having HCV antiviral activity with reduced hyperbilirubinemia side-effect
CU20200082A7 (es) 2018-05-09 2021-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para la reducción de la expresión de fxi
EP4663758A3 (en) 2018-05-09 2026-04-29 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
CN120247995A (zh) * 2018-05-30 2025-07-04 诺华股份有限公司 脂质修饰的核酸化合物和方法
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
KR20210035805A (ko) 2018-06-15 2021-04-01 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 세포후 신호전달 인자의 조절을 통한 면역 활성의 증가
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
US12496311B2 (en) 2018-07-17 2025-12-16 Aronora, Inc. Methods for safely reducing thrombopoietin
JP2021531278A (ja) 2018-07-20 2021-11-18 レグルス セラピューティクス インコーポレイテッド オリゴヌクレオチドの経口送達のための方法
AR115847A1 (es) 2018-07-25 2021-03-03 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de la atxn2
EP3833397A4 (en) 2018-08-08 2023-06-14 Arcturus Therapeutics, Inc. COMPOSITIONS AND AGENTS AGAINST NON-ALCOHOLIC STEATOHEPATITIS
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
JP7625512B2 (ja) 2018-08-13 2025-02-03 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 Ｂ型肝炎ウイルス（ＨＢＶ）ｄｓＲＮＡ物質組成物およびその使用方法
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
WO2020038377A1 (zh) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
KR102950141B1 (ko) 2018-08-24 2026-04-07 트랜슬레이트 바이오 인코포레이티드 전령 rna의 정제 방법
US20210332367A1 (en) 2018-09-18 2021-10-28 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
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
WO2020069055A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
US11896674B2 (en) 2018-09-30 2024-02-13 Suzhou Ribo Life Science Co., Ltd. SiRNA conjugate, preparation method therefor and use thereof
GB201816554D0 (en) 2018-10-10 2018-11-28 Centauri Therapeutics Ltd Novel compounds and therapeutic uses thereof
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
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
CR20210311A (es) 2018-11-15 2021-07-22 Ionis Pharmaceuticals Inc Moduladores de la expresión de irf5
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
US12281305B2 (en) 2018-11-21 2025-04-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing prion expression
CN113166760A (zh) 2018-11-23 2021-07-23 赛诺菲 用于抑制angptl8的新型rna组合物和方法
AU2019390097A1 (en) 2018-11-30 2021-07-15 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
WO2020117706A1 (en) 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity
CR20210371A (es) 2018-12-10 2021-08-27 Amgen Inc Constructos de arni para inhibir la expresión de pnpla3
WO2020123410A1 (en) 2018-12-10 2020-06-18 Amgen Inc. CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF
AU2019406186A1 (en) 2018-12-20 2021-07-15 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of KCNT1 related disorders
CR20210395A (es) 2018-12-21 2021-11-05 Ionis Pharmaceuticals Inc Moduladores de la expresión de hsd17b13
WO2020135673A1 (zh) 2018-12-28 2020-07-02 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
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
PT3908568T (pt) 2019-01-11 2024-09-30 Acuitas Therapeutics Inc Lípidos para a administração de agentes ativos por nanopartículas lipídicas
MX2021008628A (es) 2019-01-16 2021-11-17 Genzyme Corp Composiciones de arni para serpinc1 y metodos de uso de las mismas.
CN113330117B (zh) * 2019-01-18 2024-05-28 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
BR112021013369A2 (pt) 2019-01-31 2021-09-21 Ionis Pharmaceuticals, Inc. Moduladores de expressão de yap1
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
AU2020227824B2 (en) 2019-02-27 2025-07-10 Ionis Pharmaceuticals, Inc. Modulators of MALAT1 expression
PT3947684T (pt) 2019-03-29 2025-05-19 Ionis Pharmaceuticals Inc Compostos e métodos para modular ube3a‑ats
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
WO2020227159A2 (en) 2019-05-03 2020-11-12 Flagship Pioneering Innovations V, Inc. Methods of modulating immune activity
AU2020279101B2 (en) 2019-05-17 2025-07-24 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
KR20220011689A (ko) 2019-05-22 2022-01-28 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 약제학적 조성물, 접합체, 제조 방법, 및 용도
JP7606758B2 (ja) 2019-05-22 2024-12-26 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
AU2020280439B2 (en) 2019-05-22 2025-08-14 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
US12590304B2 (en) 2019-05-22 2026-03-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
CN113795582B (zh) 2019-05-24 2024-05-24 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
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
CN113795280B (zh) 2019-05-24 2024-04-05 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
CA3141902A1 (en) 2019-05-30 2020-12-03 Amgen Inc. Rnai constructs for inhibiting scap expression and methods of use thereof
MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).
JP7720788B2 (ja) 2019-06-25 2025-08-08 アムジエン・インコーポレーテツド 炭水化物に連結されたオリゴヌクレオチドのための精製方法
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
WO2021021673A1 (en) 2019-07-26 2021-02-04 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
EP4007811A2 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase b2 (cpb2) irna compositions and methods of use thereof
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
US20220340911A1 (en) 2019-08-13 2022-10-27 Amgen Inc. Rnai constructs for inhibiting slc30a8 expression and methods of use thereof
US20220281911A1 (en) 2019-08-19 2022-09-08 Mina Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
CN115176011B (zh) 2019-08-27 2025-11-11 赛诺菲 用于抑制pcsk9的组合物和方法
US20230076803A1 (en) * 2019-08-29 2023-03-09 Suzhou Ribo Life Science Co., Ltd. Compound and drug conjugate, and preparation method and use thereof
JP7672394B2 (ja) 2019-09-03 2025-05-07 アークトゥラス・セラピューティクス・インコーポレイテッド 治療的に活性な複合体のアシアロ糖タンパク質受容体媒介送達
BR112022003860A2 (pt) 2019-09-03 2022-08-16 Alnylam Pharmaceuticals Inc Composições e métodos para inibir a expressão do gene lect2
IL291251B1 (en) * 2019-09-10 2026-02-01 Daiichi Sankyo Co Ltd Oligonucleotide-galnac conjugate for targeted delivery to the liver and method for its production
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
EP4031662A1 (en) 2019-09-20 2022-07-27 Translate Bio, Inc. Mrna encoding engineered cftr
US12503699B2 (en) 2019-10-04 2025-12-23 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
US11781138B2 (en) 2019-10-14 2023-10-10 Aro Biotherapeutics Company FN3 domain-siRNA conjugates and uses thereof
CN119119240A (zh) 2019-10-14 2024-12-13 Aro生物疗法公司 结合cd71的纤维粘连蛋白iii型结构域
WO2021074772A1 (en) 2019-10-14 2021-04-22 Astrazeneca Ab Modulators of pnpla3 expression
WO2021076543A1 (en) 2019-10-14 2021-04-22 Aro Biotherapeutics Company Epcam binding fibronectin type iii domains
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
WO2021081026A1 (en) 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
EP4051795A1 (en) 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
BR112022007795A2 (pt) * 2019-11-06 2022-07-05 Alnylam Pharmaceuticals Inc Administração extra-hepática
WO2021092145A1 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna composition and methods of use thereof for treating or preventing ttr-associated ocular diseases
BR112022009216A2 (pt) 2019-11-13 2022-08-02 Alnylam Pharmaceuticals Inc Métodos e composições para tratar um distúrbio associado a angiotensinogênio (agt)
WO2021102356A1 (en) 2019-11-20 2021-05-27 Berg Llc Combination therapy of a coenzyme q10 compound and radiation therapy for treatment of glioma
US12565653B2 (en) 2019-11-22 2026-03-03 Alnylam Pharmaceuticals, Inc. ATAXIN3 (ATXN3) RNAi agent compositions and methods of use thereof
CA3164050A1 (en) 2019-12-09 2021-06-17 Empirico Inc. Oligonucleotides for treatment of angiopoietin like 4 (angptl4) related diseases
MX2022007005A (es) 2019-12-09 2022-08-25 Amgen Inc Construcciones de iarn y metodos para inhibir la expresion de lpa.
KR20220115995A (ko) 2019-12-13 2022-08-19 알닐람 파마슈티칼스 인코포레이티드 인간 염색체 ９ 개방 판독 프레임 72 (C9orf72) iRNA 제제 조성물 및 이의 사용 방법
WO2021126734A1 (en) 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
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
US20230114107A1 (en) 2019-12-17 2023-04-13 Flagship Pioneering Innovations V, Inc. Combination anti-cancer therapies with inducers of iron-dependent cellular disassembly
CN114929729B (zh) 2020-01-30 2024-12-03 卫材R&D管理有限公司 核酸复合体及包含该核酸复合体的医药组合物
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
JP7735288B2 (ja) 2020-02-18 2025-09-08 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 アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用法
CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
GB2613225B (en) 2020-03-04 2024-01-03 Verve Therapeutics Inc Compositions and methods for targeted RNA delivery
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
CN115461460A (zh) 2020-03-06 2022-12-09 阿尔尼拉姆医药品有限公司 用于抑制转甲状腺素蛋白(ttr)的表达的组合物和方法
BR112022017822A2 (pt) 2020-03-06 2022-11-08 Alnylam Pharmaceuticals Inc Composições de irna de cetoexocinase (khk) e métodos de uso das mesmas
EP4121534A1 (en) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
EP4126967A1 (en) 2020-03-23 2023-02-08 Amgen Inc. Monoclonal antibodies to chemically-modified nucleic acids and uses thereof
US20230138409A1 (en) 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
JP2023520763A (ja) 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー ゴーシェ治療薬を発現するための非ウイルス性ｄｎａベクター及びその使用
TW202204615A (zh) 2020-03-26 2022-02-01 美商阿尼拉製藥公司 冠狀病毒ｉＲＮＡ組成物及其使用方法
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
US12534731B2 (en) 2020-04-01 2026-01-27 Alnylam Pharmaceuticals, Inc. Alpha-2A adrenergic receptor (ADRA2A) iRNA agent compositions and methods of use thereof
KR20230008729A (ko) 2020-04-06 2023-01-16 알닐람 파마슈티칼스 인코포레이티드 Myoc 발현을 사일런싱하기 위한 조성물 및 방법
JP2023521094A (ja) 2020-04-07 2023-05-23 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 Ｓｃｎ９ａ発現をサイレンシングするための組成物および方法
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
FI20215508A1 (en) 2020-04-09 2021-10-10 Niemelae Erik Johan Mimetic nanoparticles to prevent the spread of new coronaviruses and reduce the rate of infection
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
CN115916262A (zh) 2020-04-21 2023-04-04 旗舰创业股份有限公司 双官能分子及其使用方法
IL297702A (en) 2020-04-27 2022-12-01 Alnylam Pharmaceuticals Inc Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
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
JP2023523790A (ja) 2020-04-30 2023-06-07 アルナイラム ファーマシューティカルズ， インコーポレイテッド 補体因子Ｂ（ＣＦＢ）ｉＲＮＡ組成物およびその使用方法
TWI901676B (zh) 2020-05-01 2025-10-21 美商Ionis製藥公司 調節ａｔｘｎ１之化合物及方法
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
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)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
EP4150087A1 (en) 2020-05-15 2023-03-22 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)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
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
US20230183707A1 (en) 2020-05-21 2023-06-15 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
KR20240004092A (ko) 2020-06-01 2024-01-11 암젠 인크 Hsd17b13 발현을 억제하기 위한 rnai 작제물 및 이의 사용 방법
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
WO2021252649A2 (en) 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
WO2021257782A1 (en) 2020-06-18 2021-12-23 Alnylam Pharmaceuticals, Inc. XANTHINE DEHYDROGENASE (XDH) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US20230355804A1 (en) 2020-06-29 2023-11-09 Flagship Pioneering Innovations V, Inc. Viruses engineered to promote thanotransmission and their use in treating cancer
EP4172338A4 (en) 2020-06-29 2025-06-11 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING PLP1
CN111744019B (zh) * 2020-07-01 2023-08-04 深圳瑞吉生物科技有限公司 基于甘露糖的mRNA靶向递送系统及其应用
KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
WO2022011214A1 (en) 2020-07-10 2022-01-13 Alnylam Pharmaceuticals, Inc. Circular sirnas
ES3054438T3 (en) 2020-07-16 2026-02-03 Acuitas Therapeutics Inc Cationic lipids for use in lipid nanoparticles
CA3189922A1 (en) 2020-07-28 2022-02-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
IL300258A (en) 2020-08-07 2023-03-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating SCN2A
KR20230046319A (ko) 2020-08-13 2023-04-05 암젠 인크 MARC1 발현을 억제하기 위한 RNAi 작제물 및 방법
US20230303618A1 (en) * 2020-08-21 2023-09-28 Reyoung Corporation Triptolide conjugates and uses thereof
CN116348150A (zh) * 2020-09-11 2023-06-27 箭头药业股份有限公司 用于递送治疗剂的脂质缀合物
CZ310613B6 (cs) 2020-09-23 2026-01-28 Ústav organické chemie a biochemie AV ČR, v. v. i. Lipidoidy pro transfekci nukleových kyselin a jejich použití
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
WO2022079222A1 (en) 2020-10-16 2022-04-21 Sanofi Novel rna compositions and methods for inhibiting angptl3
WO2022079221A1 (en) 2020-10-16 2022-04-21 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
AU2021373780A1 (en) 2020-11-05 2023-06-08 Amgen Inc. METHODS FOR TREATING ATHEROSCLEROTIC CARDIOVASCULAR DISEASE WITH LPA-TARGETED RNAi CONSTRUCTS
KR20230107625A (ko) 2020-11-13 2023-07-17 알닐람 파마슈티칼스 인코포레이티드 응고 인자 V(F5) iRNA 조성물 및 이의 사용 방법
LT4136092T (lt) 2020-11-18 2024-09-25 Ionis Pharmaceuticals, Inc. Junginiai ir būdai, skirti angiotenzinogeno raiškos moduliavimui
TW202237150A (zh) 2020-12-01 2022-10-01 美商艾拉倫製藥股份有限公司 用於抑制ｈａｏ１（羥基酸氧化酶１（乙醇酸氧化酶））基因表現的方法及組成物
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
WO2022133278A2 (en) 2020-12-18 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor xii
EP4262976A1 (en) 2020-12-21 2023-10-25 Flagship Pioneering Innovations V, Inc. Use of cell turnover factors for increasing tissue regeneration
WO2022140365A1 (en) * 2020-12-22 2022-06-30 Empirico Inc. Galnac compositions for improving sirna bioavailability
IL303886A (en) 2020-12-23 2023-08-01 Flagship Pioneering Inc Compositions of modified trems and uses thereof
EP4273245A4 (en) 2020-12-29 2024-12-11 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND SIRNA CONJUGATE WITH THE NUCLEIC ACID, MANUFACTURING PROCESS THEREOF AND USE THEREOF
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
WO2022172083A2 (en) * 2021-01-21 2022-08-18 Sirnaomics, Inc. Targeted nucleic acid therapy for hepatitis b
CA3198230A1 (en) * 2021-01-28 2022-08-04 Jian Chen Conjugate and use 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
CA3204340A1 (en) * 2021-01-30 2022-08-04 Ahmad Ali MORTAZAVI Conjugated oligonucleotide compounds, methods of making and uses thereof
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
EP4298220A1 (en) 2021-02-25 2024-01-03 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods of use thereof
AU2022226098A1 (en) 2021-02-26 2023-08-24 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
JP2024508896A (ja) 2021-03-04 2024-02-28 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 アンジオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびその使用方法
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
EP4161942B1 (en) 2021-03-19 2023-08-02 Bachem Holding AG Improved oligonucleotide synthesis suppressing depurination
EP4316495A4 (en) * 2021-03-25 2025-12-31 Aptacure Therapeutics Ltd CONJUGATE OF NUCLEIC ACID MOLECULE HAVING A PROLONGED IN VIVO HALF-LIFE
TW202305133A (zh) 2021-03-26 2023-02-01 英商米納治療有限公司 Tmem173 sarna組合物及其使用方法
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
EP4313109A1 (en) 2021-03-31 2024-02-07 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
CA3214833A1 (en) 2021-04-06 2022-10-13 Bpgbio, Inc. Protein markers for the prognosis of breast cancer progression
WO2022216846A1 (en) 2021-04-06 2022-10-13 Berg Llc Protein markers for estrogen receptor (er)-positive-like and estrogen receptor (er)-negative-like breast cancer
WO2022216841A1 (en) 2021-04-06 2022-10-13 Berg Llc Protein markers for estrogen receptor (er)-positive luminal a(la)-like and luminal b1 (lb1)-like breast cancer
CN117510830A (zh) * 2021-04-08 2024-02-06 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
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
US20250282813A1 (en) 2021-04-09 2025-09-11 Bachem Holding Ag Pseudo solid phase protecting group and methods for the synthesis of oligonucleotides and oligonucleotide conjugates
EP4323008A4 (en) 2021-04-14 2026-02-25 Aro Biotherapeutics Company FN3-ARNSI DOMAIN CONJUGATES AND THEIR USES
CA3214552A1 (en) 2021-04-14 2022-10-20 Russell C. Addis Cd71 binding fibronectin type iii domains
TW202304472A (zh) 2021-04-23 2023-02-01 美商格納生物公司 經聚醣修飾之核酸、製備方法及治療用途
BR112023022439A2 (pt) 2021-04-26 2023-12-26 Celanese Eva Performance Polymers Llc Dispositivo implantável para liberação sustentada de um composto de fármaco macromolecular
EP4330392A1 (en) 2021-04-26 2024-03-06 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
US20240216535A1 (en) 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
AU2022264509A1 (en) 2021-04-27 2023-12-14 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4341401A1 (en) 2021-05-18 2024-03-27 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
EP4341405A1 (en) 2021-05-20 2024-03-27 Korro Bio, Inc. Methods and compositions for adar-mediated editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
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
AR126070A1 (es) 2021-06-08 2023-09-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para tratar o prevenir la enfermedad de stargardt y/o trastornos asociados con la proteína transportadora de retinol 4 (rbp4)
AU2022293556A1 (en) 2021-06-18 2024-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
CA3224904A1 (en) 2021-06-21 2022-12-29 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
CN117677699A (zh) 2021-06-23 2024-03-08 马萨诸塞大学 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
EP4363574A1 (en) 2021-06-29 2024-05-08 Korro Bio, Inc. Methods and compositions for adar-mediated editing
AU2022303363A1 (en) 2021-06-29 2024-01-18 Flagship Pioneering Innovations V, Inc. Immune cells engineered to promote thanotransmission and uses thereof
AU2022303164A1 (en) 2021-06-30 2024-01-18 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
CA3225573A1 (en) 2021-07-08 2023-01-12 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
TW202308664A (zh) 2021-07-08 2023-03-01 日商日本新藥股份有限公司 腎毒性減輕劑
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
WO2023284763A1 (zh) 2021-07-16 2023-01-19 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
EP4372085A4 (en) 2021-07-16 2026-01-21 Suzhou Ribo Life Science Co Ltd Nucleic acid, composition and conjugate, container, preparation process and use
CZ310443B6 (cs) 2021-07-19 2025-06-25 Ústav organické chemie a biochemie AV ČR, v. v. i. Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití
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
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
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
WO2023015223A2 (en) * 2021-08-03 2023-02-09 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
CA3227852A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
EP4381071A1 (en) 2021-08-04 2024-06-12 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing angiotensinogen (agt)
EP4380623A4 (en) * 2021-08-04 2026-05-06 Hepagene Therapeutics Hk Ltd Ligand conjugates for delivery of therapeutically active agents
US12465647B2 (en) 2021-08-05 2025-11-11 Sanegene Bio Usa Inc. 1′-alkyl modified ribose derivatives and methods of use
WO2023016999A2 (en) 2021-08-09 2023-02-16 Cargene Therapeutics Pte. Ltd. Compounds
CN118076737A (zh) 2021-08-13 2024-05-24 阿尔尼拉姆医药品有限公司 因子XII（F12）iRNA组合物及其使用方法
JP2024538859A (ja) 2021-08-31 2024-10-24 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 細胞死誘導ＤＦＦＡ様エフェクターＢ（ＣＩＤＥＢ）ｉＲＮＡ組成物およびその使用方法
WO2023039594A1 (en) * 2021-09-13 2023-03-16 Ganna Bio, Inc. Glycan conjugate compositions and methods
JP2024535850A (ja) 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド 補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
EP4405368A1 (en) 2021-09-22 2024-07-31 Sanegene Bio USA Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
TW202328449A (zh) 2021-09-24 2023-07-16 美商艾拉倫製藥公司 微管相關蛋白ＴＡＵ（ＭＡＰＴ）ｉＲＮＡ試劑組合物及其使用方法
US12042509B2 (en) 2021-10-01 2024-07-23 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
EP4413015A1 (en) 2021-10-05 2024-08-14 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
TW202334415A (zh) 2021-10-14 2023-09-01 美商止血治療股份有限公司 治療與膽汁酸轉運體相關之疾病的組合物及方法
CN118369427A (zh) 2021-10-15 2024-07-19 阿尔尼拉姆医药品有限公司 肝外递送irna组合物及其使用方法
WO2023069754A2 (en) 2021-10-22 2023-04-27 Amgen Inc. Rnai constructs for inhibiting gpam expression and methods of use thereof
WO2023069603A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
CN118302525A (zh) 2021-10-29 2024-07-05 阿尔尼拉姆医药品有限公司 补体因子B（CFB）iRNA组合物及其使用方法
EP4423272A2 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
CA3237770A1 (en) 2021-11-10 2023-05-19 University Of Rochester Antisense oligonucleotides for modifying protein expression
US20250019702A1 (en) 2021-11-10 2025-01-16 University Of Rochester Gata4-targeted therapeutics for treatment of cardiac hypertrophy
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
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
US20230346819A1 (en) 2022-02-22 2023-11-02 Sanegene Bio Usa Inc. 5'-modified carbocyclic ribonucleotide derivatives and methods of use
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023171804A1 (ja) 2022-03-10 2023-09-14 日本新薬株式会社 抗ウイルスアンチセンスオリゴマー
CN119072330A (zh) 2022-03-14 2024-12-03 世代生物公司 异源初免加强疫苗组合物和使用方法
EP4495240A1 (en) 2022-03-16 2025-01-22 Daiichi Sankyo Company, Limited Sirna for suppressing expression of transferrin receptor-2
US11879125B2 (en) 2022-03-16 2024-01-23 Empirico Inc. GalNAc compositions for improving siRNA bioavailability
US20250188477A1 (en) 2022-03-16 2025-06-12 Daiichi Sankyo Company, Limited CHEMICALLY MODIFIED OLIGONUCLEOTIDE HAVING RNAi ACTIVITY
AU2023245603A1 (en) 2022-03-28 2024-11-07 Empirico Inc. Modified oligonucleotides
EP4514970A2 (en) * 2022-04-26 2025-03-05 University of Connecticut Genetic sequence-carbohydrate conjugates for enhanced liver- and kidney-specific targeting
US20240002857A1 (en) 2022-05-09 2024-01-04 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
EP4522742A2 (en) 2022-05-13 2025-03-19 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
US20230372508A1 (en) 2022-05-19 2023-11-23 Olix Us, Inc. Linkers coupling functional ligands to macromolecules
CN119487191A (zh) 2022-06-14 2025-02-18 大睿生物医药科技(上海)有限公司 调控PCSK9基因活性的siRNA分子
CN119421887A (zh) 2022-06-14 2025-02-11 大睿生物医药科技(上海)有限公司 环膦酸酯修饰的核苷酸
US20250179492A1 (en) 2022-06-22 2025-06-05 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
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
CN119487192A (zh) * 2022-06-27 2025-02-18 中美瑞康核酸技术(南通)研究院有限公司 小激活核酸分子及其在治疗遗传性血管性水肿中的用途
EP4555088A1 (en) 2022-07-11 2025-05-21 Sanegene Bio USA Inc. Optimized 2'- modified ribose derivatives and methods of use
US20240084301A1 (en) 2022-07-25 2024-03-14 Amgen Inc. Rnai constructs and methods for inhibiting fam13a expression
KR20250046303A (ko) 2022-08-05 2025-04-02 사네진 바이오 유에스에이 인크. 안지오텐시노겐 (agt)을 표적화하는 이중 가닥 rna 및 그의 사용 방법
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
US20260055426A1 (en) 2022-08-19 2026-02-26 Generation Bio Co. CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF
WO2024050261A1 (en) 2022-08-29 2024-03-07 University Of Rochester Antisense oligonucleotide-based anti-fibrotic therapeutics
EP4569113A1 (en) 2022-09-15 2025-06-18 Regeneron Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
US20260001902A1 (en) 2022-09-19 2026-01-01 Bachem Holding Ag Improved oligonucleotide synthesis
JP2025532127A (ja) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｍｅｃｐ２発現を低減する化合物及び方法
WO2024073404A1 (en) 2022-09-28 2024-04-04 Sarepta Therapeutics, Inc. Pompe disease mouse model generation, characterization and methods of use
WO2024073732A1 (en) 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
US20240174732A1 (en) 2022-10-05 2024-05-30 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additional polypeptides and their use in treating cancer
KR20250088573A (ko) 2022-10-14 2025-06-17 사네진 바이오 유에스에이 인크. C3을 표적화하는 소형 간섭 rna 및 그의 용도
WO2024083746A1 (en) 2022-10-17 2024-04-25 Bachem Holding Ag Method and composition for oligonucleotide synthesis
CR20250257A (es) 2022-12-02 2025-08-28 Shanghai Argo Biopharmaceutical Co Ltd Análogos de ácidos nucleicos bicíclicos abásicos y compuestos oligoméricos preparados a partir de estos
EP4630061A1 (en) * 2022-12-08 2025-10-15 Nanovation Therapeutics Inc. Lipid nanoparticles comprising elevated neutral lipid and a targeting moiety for targeted delivery of nucleic acid
US20240293555A1 (en) * 2022-12-12 2024-09-05 University Of Massachusetts Carbohydrate conjugates for the delivery of therapeutic oligonucleotides
JP2025541245A (ja) 2022-12-16 2025-12-18 アムジェン インコーポレイテッド Ｔｔｒ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
WO2024137590A2 (en) 2022-12-19 2024-06-27 Sanegene Bio Usa Inc. Small interfering rna targeting cfb and uses thereof
ES3030929T3 (en) 2022-12-19 2025-07-02 Arnatar Therapeutics Inc Arnatar compounds and methods for enhanced cellular uptake
AU2023408479A1 (en) 2022-12-19 2025-05-01 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar)
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
CN117720590A (zh) * 2022-12-30 2024-03-19 天津药明康德新药开发有限公司 一种改进的n-乙酰半乳糖胺中间体的制备方法
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
WO2024151942A2 (en) 2023-01-13 2024-07-18 Hemoshear Therapeutics, Inc. Sirnas targeting slc10a1 transcripts, compositions and uses thereof
TW202449152A (zh) 2023-02-09 2024-12-16 美商艾拉倫製藥股份有限公司 Ｒｅｖｅｒｓｉｒ分子及其使用方法
US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
WO2024186673A2 (en) 2023-03-03 2024-09-12 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
WO2024188174A1 (zh) 2023-03-10 2024-09-19 苏州瑞博生物技术股份有限公司 一种药物组合物及其用途
WO2024206230A1 (en) * 2023-03-24 2024-10-03 Trustees Of Tufts College Tissue targeting lipids and lipid nanoparticles
AU2024259356A1 (en) * 2023-04-19 2025-11-06 Prime Medicine, Inc. Lipid nanoparticle (lnp) delivery systems and formulations
TW202448484A (zh) 2023-04-20 2024-12-16 美商雅迪克斯製藥公司 Ｍａｐｔ調節組合物及其使用方法
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
MX2025013094A (es) 2023-05-03 2026-02-03 Sanegene Bio Usa Inc Conjugados basados en lipidos para la administracion sistemica en el sistema nervioso central, el sistema nervioso periferico y ocular
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
AU2024269205A1 (en) 2023-05-11 2025-12-11 Keymed Biosciences (Us) Inc. Dsrna molecules for regulating masp2 gene activity
WO2024238396A1 (en) 2023-05-12 2024-11-21 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
EP4709855A2 (en) 2023-05-12 2026-03-18 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
CN118993970B (zh) * 2023-05-17 2025-12-23 苏州富士莱医药股份有限公司 一种l96侧链化合物的制备方法
CN119343449A (zh) 2023-05-24 2025-01-21 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
EP4562158A1 (en) 2023-06-13 2025-06-04 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar) for angiotensinogen
EP4731763A1 (en) 2023-06-20 2026-04-29 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
WO2024263915A2 (en) 2023-06-21 2024-12-26 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
KR20260016971A (ko) * 2023-06-30 2026-02-04 쑤저우 스란 바이오테크놀로지 씨오., 엘티디. 핵산 접합체, 그의 제조 방법 및 용도
WO2025007961A1 (zh) 2023-07-06 2025-01-09 大睿生物 调控AGT表达的dsRNA分子
WO2025015338A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
TW202517784A (zh) 2023-07-14 2025-05-01 大陸商云合智藥（蘇州）生物科技有限公司 用於抑制己酮糖激酶表達的ＲＮＡｉ劑及其應用
GB202311324D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
GB202311334D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025021831A1 (en) 2023-07-24 2025-01-30 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
US20250064939A1 (en) 2023-07-24 2025-02-27 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
KR20260049597A (ko) 2023-08-04 2026-04-14 알닐람 파마슈티칼스 인코포레이티드 Ctnnb1-관련 질환을 치료하기 위한 방법 및 조성물
AU2024321710A1 (en) 2023-08-04 2026-03-05 Chia Tai Tianqing Pharmaceutical Group Co., Ltd. Double-stranded ribonucleic acid targeting pd-l1
CN119464281A (zh) 2023-08-09 2025-02-18 北京福元医药股份有限公司 抑制DGAT2基因表达的siRNA、siRNA缀合物或其前药和药物组合物及用途
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025040139A1 (zh) 2023-08-23 2025-02-27 云合智药（苏州）生物科技有限公司 用于抑制二酰甘油-O-酰基转移酶2表达的RNAi剂及其应用
CN121729496A (zh) 2023-08-31 2026-03-24 正大天晴药业集团股份有限公司 靶向凝血因子xi的双链核糖核酸
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
GB202314724D0 (en) 2023-09-26 2023-11-08 Astrazeneca Ab compounds and methods for reducing psd3 expression
WO2025072672A2 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
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
AU2024360411A1 (en) 2023-10-10 2026-02-26 Avanti Polar Lipids, Llc Liver asialoglycoprotein receptor targeted lipid and uses thereof
EP4695265A1 (en) 2023-10-16 2026-02-18 Bachem Holding AG Method and composition for oligonucleotide synthesis
WO2025085732A1 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Humanized dnm2 mouse model generation, characterization and methods of use
WO2025090796A1 (en) * 2023-10-24 2025-05-01 University Of Connecticut Carbohydrate ligands and uses thereof
WO2025096809A1 (en) 2023-10-31 2025-05-08 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
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
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
EP4671372A3 (en) 2024-01-29 2026-03-11 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof
WO2025193807A1 (en) 2024-03-12 2025-09-18 Sarepta Therapeutics, Inc. Humanized umod mouse model generation, characterization and methods of use
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025224036A1 (en) 2024-04-22 2025-10-30 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2025260042A1 (en) 2024-06-14 2025-12-18 Amgen Inc. Rnai constructs and methods for inhibiting cnr1 expression
WO2025260068A1 (en) 2024-06-14 2025-12-18 Tune Therapeutics, Inc. Lipid nanoparticle formulation for delivery of nucleic acids to cells
WO2026006436A1 (en) 2024-06-25 2026-01-02 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of tar dna binding protein 43 kda (tdp43)
WO2026006275A2 (en) 2024-06-26 2026-01-02 Amgen Inc. Rnai constructs and methods for inhibiting expression of inhbe
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
US20260015617A1 (en) 2024-07-12 2026-01-15 Novartis Ag DOUBLE STRANDED RNAi AGENTS, COMPOSITIONS AND METHODS OF USE
WO2026017140A2 (en) 2024-07-18 2026-01-22 Sanegene Bio Usa Inc. Small interfering rna targeting fxi and uses thereof
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use
WO2026041067A2 (en) 2024-08-20 2026-02-26 Sanegene Bio Usa Inc. Small interfering rna targeting myostatin (mstn) and uses thereof
WO2026044153A1 (en) 2024-08-21 2026-02-26 Bpgbio, Inc. Use of biomarkers in the diagnosis of pancreatic cancer
WO2026050243A1 (en) 2024-08-26 2026-03-05 Korro Bio, Inc. Galnac conjugated oligonucleotides for rna editing
WO2026046411A1 (en) 2024-09-02 2026-03-05 Sanegene Bio Usa Inc. Small interfering rna targeting plasminogen (plg) and uses thereof
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor

Family Cites Families (134)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
BE795866A (fr) 1972-02-29 1973-08-23 Basf Ag Procede de preparation de chlorure de choline solide a ecoulement libre
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
CH624011A5 (https=) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
DE3013839A1 (de) 1979-04-13 1980-10-30 Freunt Ind Co Ltd Verfahren zur herstellung einer aktivierten pharmazeutischen zusammensetzung
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
JPS5921613A (ja) 1982-07-28 1984-02-03 Takeda Chem Ind Ltd 直腸投与製剤
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4486435A (en) 1983-05-16 1984-12-04 Basf Wyandotte Corporation Spray-dried vitamin powders using hydrophobic silica
JPH0818982B2 (ja) 1983-11-09 1996-02-28 昭和電工株式会社 乳量の増加方法
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
WO1986005519A1 (en) 1985-03-15 1986-09-25 James Summerton Polynucleotide assay reagent and method
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
FR2594693B1 (fr) 1986-02-24 1990-01-05 Farah Nabil Nouveaux procedes de preparation a partir d'emulsions seches de formes orales solides a liberation modifiee et retardee de leur principes actifs
EP0243686B1 (en) * 1986-03-27 1992-07-15 Sumitomo Pharmaceuticals Company, Limited Beta-lactam compounds, and their production
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
JPH081881B2 (ja) 1987-03-31 1996-01-10 日立エーアイシー株式会社 電気二重層コンデンサ
SE8701479L (sv) 1987-04-09 1988-10-10 Carbomatrix Ab Metod foer inneslutning av biologiskt verksamma preparat samt anvaendning daerav
JPH081884B2 (ja) 1987-04-10 1996-01-10 松下電子工業株式会社 レジストパタ−ンの形成方法
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 (https=) 1987-09-16 1989-10-31 Nestle Sa
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
AU637800B2 (en) 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. 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
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
US5223618A (en) 1990-08-13 1993-06-29 Isis Pharmaceuticals, Inc. 4'-desmethyl nucleoside analog compounds
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
JPH0874B2 (ja) 1990-07-27 1996-01-10 アイシス・ファーマシューティカルス・インコーポレーテッド 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
ES2061416T3 (es) 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
US5110455A (en) 1990-12-13 1992-05-05 Cyprus Minerals Company Method for achieving enhanced copper flotation concentrate grade by oxidation and flotation
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
AU4541093A (en) 1992-06-18 1994-01-24 Genpharm International, Inc. Methods for producing transgenic non-human animals harboring a yeast artificial chromosome
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304620D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
JPH07101882A (ja) 1993-09-30 1995-04-18 Sanei Gen F F I Inc 水溶性ヘミセルロースを含有する製剤
JPH07101881A (ja) 1993-09-30 1995-04-18 Sanei Gen F F I Inc 水溶性ヘミセルロースを含有する製剤
JP3342550B2 (ja) 1993-10-01 2002-11-11 三栄源エフ・エフ・アイ株式会社 水溶性ヘミセルロースを含有する製剤
JPH07101884A (ja) 1993-10-01 1995-04-18 Sanei Gen F F I Inc 水溶性ヘミセルロースを含有する製剤
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5539083A (en) 1994-02-23 1996-07-23 Isis Pharmaceuticals, Inc. Peptide nucleic acid combinatorial libraries and improved methods of synthesis
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
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
WO1996011266A2 (en) 1994-10-05 1996-04-18 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
CA2220950A1 (en) 1995-05-26 1996-11-28 Somatix Therapy Corporation Delivery vehicles comprising stable lipid/nucleic acid complexes
US5976567A (en) 1995-06-07 1999-11-02 Inex Pharmaceuticals Corp. Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
CN1120707C (zh) * 1995-11-22 2003-09-10 约翰斯·霍普金斯大学 增强生物分子的细胞摄取的配体
EP0957107B1 (en) * 1996-05-16 2004-07-21 Nissin Food Products Co., Ltd. Novel compounds 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
CA2361201A1 (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
US7491805B2 (en) * 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
CN100406065C (zh) 2000-12-01 2008-07-30 细胞工厂治疗公司 糖基化/半乳糖基化肽、双官能接头和核苷酸单体/多聚体的缀合物以及相关的组合物和使用方法
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
ATE427948T1 (de) 2001-04-24 2009-04-15 Purdue Research Foundation Folat-mimetika und deren folatrezeptorbindende konjugate
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
EP2415486B1 (en) 2001-05-18 2017-02-22 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
WO2003008628A2 (en) 2001-07-20 2003-01-30 Ribozyme Pharmacuticals, Inc. Enzymatic nucleic acid peptide conjugates
CA2498772A1 (en) 2002-09-11 2004-03-25 Santaris Pharma A/S Modified pna molecules
WO2004090108A2 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals Irna conjugates
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
WO2005014655A2 (en) * 2003-08-08 2005-02-17 Fresenius Kabi Deutschland Gmbh Conjugates of hydroxyalkyl starch and a protein
EP1664316B1 (en) * 2003-09-15 2012-08-29 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
JP5398982B2 (ja) * 2004-05-24 2014-01-29 ミダテック リミテッド Ｒｎａリガンドを含むナノ粒子
EP1791567B1 (en) * 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
US20060148740A1 (en) * 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
WO2009073809A2 (en) * 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
DE102009039097B3 (de) 2009-08-27 2010-11-25 Siemens Aktiengesellschaft Verfahren zum Übertragen von Daten in einem Sensornetzwerk, Sensorknoten und Zentral-Rechner
WO2012005769A1 (en) 2010-07-09 2012-01-12 Telecommunication Systems, Inc. Location privacy selector
EP3318015B1 (en) 2015-06-30 2019-10-30 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
WO2020078755A1 (en) 2018-10-18 2020-04-23 Signify Holding B.V. Determining light settings and/or daylight blocker settings based on data signal quality
EP3921593B1 (en) 2019-02-04 2023-03-15 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
WO2020235106A1 (ja) 2019-05-23 2020-11-26 三菱電機株式会社 数値制御装置
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
JP6542853B2 (ja) 2019-07-10
US9867882B2 (en) 2018-01-16
AU2008333811B2 (en) 2014-05-01
CA2708173C (en) 2016-02-02
EP3141265A1 (en) 2017-03-15
JP2015025007A (ja) 2015-02-05
WO2009082607A8 (en) 2010-01-28
JP6215284B2 (ja) 2017-10-18
WO2009073809A3 (en) 2009-12-30
CA2708153C (en) 2017-09-26
US20090239814A1 (en) 2009-09-24
AU2008340355A1 (en) 2009-07-02
JP2019023241A (ja) 2019-02-14
EP4321177A3 (en) 2024-05-08
US8106022B2 (en) 2012-01-31
US12059468B2 (en) 2024-08-13
US11110174B2 (en) 2021-09-07
JP5843914B2 (ja) 2016-01-13
JP2016034974A (ja) 2016-03-17
US9814777B2 (en) 2017-11-14
EP2231195A2 (en) 2010-09-29
US20150119445A1 (en) 2015-04-30
US20130178512A1 (en) 2013-07-11
CA2708173A1 (en) 2009-07-02
JP2014139232A (ja) 2014-07-31
US20190184018A1 (en) 2019-06-20
JP2011505426A (ja) 2011-02-24
JP2021080299A (ja) 2021-05-27
US20090247608A1 (en) 2009-10-01
US9370582B2 (en) 2016-06-21
US11666653B2 (en) 2023-06-06
JP2018029599A (ja) 2018-03-01
EP4223299A3 (en) 2023-08-30
CA2930393A1 (en) 2009-06-11
JP6850827B2 (ja) 2021-03-31
US20200297853A1 (en) 2020-09-24
US20150119444A1 (en) 2015-04-30
EP2229186A2 (en) 2010-09-22
CA2910760C (en) 2019-07-09
US9370581B2 (en) 2016-06-21
US20150011615A1 (en) 2015-01-08
US20160375137A9 (en) 2016-12-29
EP4074344A1 (en) 2022-10-19
EP3705125B1 (en) 2023-07-05
JP2023010881A (ja) 2023-01-20
EP3156077B1 (en) 2022-03-09
EP3156077A1 (en) 2017-04-19
US20220008541A1 (en) 2022-01-13
WO2009073809A2 (en) 2009-06-11
CA2930393C (en) 2022-11-29
JP2011505425A (ja) 2011-02-24
JP2022190116A (ja) 2022-12-22
EP4321177A2 (en) 2024-02-14
US20220143189A1 (en) 2022-05-12
CA2910760A1 (en) 2009-07-02
AU2008333811A1 (en) 2009-06-11
EP4223299A2 (en) 2023-08-09
JP7590395B2 (ja) 2024-11-26
US8828956B2 (en) 2014-09-09
CN102006890A (zh) 2011-04-06
JP2019147846A (ja) 2019-09-05
EP2231195B1 (en) 2017-03-29
CA3043911A1 (en) 2009-07-02
US9352048B2 (en) 2016-05-31
CA2708153A1 (en) 2009-06-11
JP2017002082A (ja) 2017-01-05
WO2009082607A2 (en) 2009-07-02
US20120136042A1 (en) 2012-05-31
US20140179761A1 (en) 2014-06-26
JP2021020928A (ja) 2021-02-18
WO2009082607A3 (en) 2009-12-17
US20160051691A1 (en) 2016-02-25
US8450467B2 (en) 2013-05-28
EP3705125A1 (en) 2020-09-09
CA3146103A1 (en) 2009-06-11
US10806791B2 (en) 2020-10-20
US20180326070A1 (en) 2018-11-15
JP5635412B2 (ja) 2014-12-03
JP5519523B2 (ja) 2014-06-11
US20190099493A1 (en) 2019-04-04

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)