[go: up one dir, main page]

CO2019005992A2 - Composiciones de arni de serpina1 y sus métodos de uso - Google Patents

Composiciones de arni de serpina1 y sus métodos de uso

Info

Publication number
CO2019005992A2
CO2019005992A2 CONC2019/0005992A CO2019005992A CO2019005992A2 CO 2019005992 A2 CO2019005992 A2 CO 2019005992A2 CO 2019005992 A CO2019005992 A CO 2019005992A CO 2019005992 A2 CO2019005992 A2 CO 2019005992A2
Authority
CO
Colombia
Prior art keywords
methods
arni1
serni
compositions
serpina1
Prior art date
Application number
CONC2019/0005992A
Other languages
English (en)
Inventor
Kallanthottathil G Rajeev
Klaus Charisse
Mark K Schlegal
Maja Janas
Vasant R Jadhav
Donald Foster
Muthiah Manoharan
Alexander V Kel'in
Jayaprakash K Nair
Martin A Maier
Shigeo Matsuda
Muthusamy Jayaraman
Alfica Sehgal
Christopher Brown
Kevin Fitzgerrald
Stuart Milstein
Original Assignee
Alnylam Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CO2019005992A2 publication Critical patent/CO2019005992A2/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Agentes de ARNi, por ejemplo, agentes de ARNi de hebra doble, direccionados al gen de Serpina1 y métodos para usar dichos agentes de ARNi para inhibir la expresión de Serpina1 y métodos de tratamiento de sujetos que padecen una enfermedad asociada con Serpina1, tal como un trastorno hepático
CONC2019/0005992A 2016-11-23 2019-06-07 Composiciones de arni de serpina1 y sus métodos de uso CO2019005992A2 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662425907P 2016-11-23 2016-11-23
US201762548589P 2017-08-22 2017-08-22
US201762549099P 2017-08-23 2017-08-23
US201762561514P 2017-09-21 2017-09-21
PCT/US2017/062701 WO2018098117A1 (en) 2016-11-23 2017-11-21 SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Publications (1)

Publication Number Publication Date
CO2019005992A2 true CO2019005992A2 (es) 2019-06-28

Family

ID=62195353

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2019/0005992A CO2019005992A2 (es) 2016-11-23 2019-06-07 Composiciones de arni de serpina1 y sus métodos de uso

Country Status (22)

Country Link
US (3) US10995336B2 (es)
EP (1) EP3545093A1 (es)
JP (1) JP7348067B2 (es)
KR (1) KR20190082317A (es)
CN (1) CN110114463B (es)
AU (1) AU2017363604A1 (es)
BR (1) BR112019010342A2 (es)
CA (1) CA3044562A1 (es)
CL (1) CL2019001388A1 (es)
CO (1) CO2019005992A2 (es)
CR (1) CR20190294A (es)
DO (1) DOP2019000132A (es)
IL (1) IL266746A (es)
MA (1) MA46923A (es)
MX (1) MX2019005898A (es)
PH (1) PH12019550076A1 (es)
RU (2) RU2761667C2 (es)
SG (1) SG10201913877WA (es)
TW (2) TWI788312B (es)
UY (1) UY37494A (es)
WO (1) WO2018098117A1 (es)
ZA (1) ZA202103080B (es)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RS57418B1 (sr) 2013-05-22 2018-09-28 Alnylam Pharmaceuticals Inc Serpina1 irnk sastavi i postupci njihove primene
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
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
US10450565B2 (en) 2017-01-10 2019-10-22 Arrowhead Pharmaceuticals, Inc. Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
AU2019239971A1 (en) * 2018-03-21 2020-09-03 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
CA3118537A1 (en) * 2018-11-09 2020-05-14 Alnylam Pharmaceuticals, Inc. Modified double stranded oligonucleotides
US20220211743A1 (en) * 2019-05-17 2022-07-07 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
JP7304565B2 (ja) 2019-07-08 2023-07-07 エムディー ヘルスケア インコーポレイテッド 細菌メタゲノム解析を通した脳腫瘍診断方法
US11207416B2 (en) 2020-03-04 2021-12-28 Verve Therapeutics, Inc. Compositions and methods for targeted RNA delivery
US20230416739A1 (en) * 2020-11-13 2023-12-28 The George Washington University, A Congressionally Chartered Not-For-Profit Corporation B-spectrin (sptbn1) deficiency protects mice from high-fat diet-induced liver disease and cancer development
WO2022162155A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
EP4175965B1 (en) 2021-01-30 2024-03-13 E-Therapeutics plc Conjugated oligonucleotide compounds, methods of making and uses thereof
JP2024537251A (ja) * 2021-10-08 2024-10-10 イー－セラピューティクス・ピーエルシー 脱塩基ヌクレオシドを含有する核酸
KR20250006359A (ko) * 2022-03-16 2025-01-10 엠피리코 인크. Sirna 생체 이용률을 개선하기 위한 galnac 조성물
WO2023192830A2 (en) 2022-03-28 2023-10-05 Empirico Inc. Modified oligonucleotides
EP4562148A1 (en) * 2022-07-27 2025-06-04 E-Therapeutics plc Nucleic acid compounds
CN117070583B (zh) * 2023-10-16 2024-05-14 吉林凯莱英制药有限公司 抑制PCSK9基因表达的siRNA的制备方法

Family Cites Families (228)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US564562A (en) 1896-07-21 Joseph p
US513030A (en) 1894-01-16 Machine for waxing or coating paper
US1861117A (en) 1927-11-14 1932-05-31 Scovill Manufacturing Co Self locking floating trim stud
US1861608A (en) 1929-12-21 1932-06-07 Emerson Electric Mfg Co Fan and means for directing the air current therethrough
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US3974817A (en) 1974-07-18 1976-08-17 Colt Industries Operating Corporation Breakerless ignition control system
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
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
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
GB8504906D0 (en) 1985-02-26 1985-03-27 Deane G Suction irrigation apparatus
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
JP2828642B2 (ja) 1987-06-24 1998-11-25 ハワード フローレイ インスティテュト オブ イクスペリメンタル フィジオロジー アンド メディシン ヌクレオシド誘導体
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5744101A (en) 1989-06-07 1998-04-28 Affymax Technologies N.V. Photolabile nucleoside protecting groups
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
CA2029273A1 (en) 1989-12-04 1991-06-05 Christine L. Brakel Modified nucleotide compounds
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US6783931B1 (en) 1990-01-11 2004-08-31 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
US7037646B1 (en) 1990-01-11 2006-05-02 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ATE167523T1 (de) 1990-05-11 1998-07-15 Microprobe Corp Teststreifen zum eintauchen für nukleinsäure- hybridisierungsassays und verfahren zur kovalenten immobilisierung von oligonucleotiden
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
ES2083593T3 (es) 1990-08-03 1996-04-16 Sterling Winthrop Inc Compuestos y metodos para inhibir la expresion de genes.
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
WO1992005186A1 (en) 1990-09-20 1992-04-02 Gilead Sciences Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
JP3172178B2 (ja) 1990-11-08 2001-06-04 ハイブライドン インコーポレイテッド 合成オリゴヌクレオチドに対する多重リポータ基の組込み
US5152817A (en) 1991-01-15 1992-10-06 Corning Incorporated Reactor for coating optical fibers
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
EP0916396B1 (en) 1991-11-22 2005-04-13 Affymetrix, Inc. (a Delaware Corporation) Combinatorial strategies for polymer synthesis
US6235887B1 (en) 1991-11-26 2001-05-22 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines
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
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
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
NZ253943A (en) 1992-06-18 1997-01-29 Genpharm Int Transfering polynucleotides into eukaryotic cells using co-lipofection complexes of a cationic lipid and the polynucleotide
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
CA2140343A1 (en) 1992-07-17 1994-02-03 Sean M. Sullivan Method and reagent for treatment of animal diseases
US5783701A (en) 1992-09-08 1998-07-21 Vertex Pharmaceuticals, Incorporated Sulfonamide inhibitors of aspartyl protease
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
DE69424406T2 (de) 1993-02-19 2000-10-26 Nippon Shinyaku Co., Ltd. Arzneistoffzusammensetzung, die ein nukleinsäurecopolymer enthält
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
AU6412794A (en) 1993-03-31 1994-10-24 Sterling Winthrop Inc. Oligonucleotides with amide linkages replacing phosphodiester linkages
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US6191105B1 (en) 1993-05-10 2001-02-20 Protein Delivery, Inc. Hydrophilic and lipophilic balanced microemulsion formulations of free-form and/or conjugation-stabilized therapeutic agents such as insulin
US6015886A (en) 1993-05-24 2000-01-18 Chemgenes Corporation Oligonucleotide phosphate esters
US6294664B1 (en) 1993-07-29 2001-09-25 Isis Pharmaceuticals, Inc. Synthesis of oligonucleotides
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US6054299A (en) 1994-04-29 2000-04-25 Conrad; Charles A. Stem-loop cloning vector and method
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5543152A (en) 1994-06-20 1996-08-06 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5556752A (en) 1994-10-24 1996-09-17 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
JP3269301B2 (ja) 1994-12-28 2002-03-25 豊田合成株式会社 ガラスラン用ゴム配合物
WO1996027606A1 (en) 1995-03-06 1996-09-12 Isis Pharmaceuticals, Inc. Improved process for the synthesis of 2'-o-substituted pyrimidines and oligomeric compounds therefrom
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
WO1996037194A1 (en) 1995-05-26 1996-11-28 Somatix Therapy Corporation Delivery vehicles comprising stable lipid/nucleic acid complexes
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
AU723163B2 (en) 1995-06-07 2000-08-17 Tekmira Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5545531A (en) 1995-06-07 1996-08-13 Affymax Technologies N.V. Methods for making a device for concurrently processing multiple biological chip assays
US5858397A (en) 1995-10-11 1999-01-12 University Of British Columbia Liposomal formulations of mitoxantrone
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US5858401A (en) 1996-01-22 1999-01-12 Sidmak Laboratories, Inc. Pharmaceutical composition for cyclosporines
US5994316A (en) 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
US6576752B1 (en) 1997-02-14 2003-06-10 Isis Pharmaceuticals, Inc. Aminooxy functionalized oligomers
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
US6034135A (en) 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
AU731909B2 (en) 1997-07-01 2001-04-05 Isis Pharmaceuticals, Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
CA2303299C (en) 1997-09-12 2016-02-23 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6528640B1 (en) 1997-11-05 2003-03-04 Ribozyme Pharmaceuticals, Incorporated Synthetic ribonucleic acids with RNAse activity
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6436989B1 (en) 1997-12-24 2002-08-20 Vertex Pharmaceuticals, Incorporated Prodrugs of aspartyl protease inhibitors
TR200002402T2 (tr) 1997-12-24 2001-01-22 Vertex Pharmaceuticals Incorporated Aspartil proteaz inhibitörü ön-ilaçlar.
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
ATE237312T1 (de) 1998-07-20 2003-05-15 Protiva Biotherapeutics Inc In liposomen verkapselte nukleinsäurekomplexe
BR9914773A (pt) 1998-10-09 2002-02-05 Ingene Inc Conjunto de elementos genéricos, método para a produção de dna de cordão único, transcrição de mrna, construção de ácido nucléico, transcrição de ssdna, vetor, sistema vetor, célula hospedeira, conjunto para a produção de uma sequência de ácido nucléico de cordão único, método para a produção in vivo ou in vitro de uma sequência de ácido nucléico de cordão único, transcrição de cdna de cordão único, ácido nucléico inibidor, molécula heteroduplex, e composição farmacêutica
KR20010099682A (ko) 1998-10-09 2001-11-09 추후보충 단일가닥 ｄｎａ의 효소 합성
US6319946B1 (en) 1999-02-12 2001-11-20 Vertex Pharmaceuticals Incorporated Inhibitors of aspartyl protease
JP2002537343A (ja) 1999-02-23 2002-11-05 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 多重粒子製剤
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
KR100782896B1 (ko) 1999-05-04 2007-12-06 엑시콘 에이/에스 Ｌ-리보-ｌｎａ 유사체
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US6147200A (en) 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
IT1318539B1 (it) 2000-05-26 2003-08-27 Italfarmaco Spa Composizioni farmaceutiche a rilascio prolungato per lasomministrazione parenterale di sostanze idrofile biologicamente
EP1334109B1 (en) 2000-10-04 2006-05-10 Santaris Pharma A/S Improved synthesis of purine locked nucleic acid analogues
US7063860B2 (en) 2001-08-13 2006-06-20 University Of Pittsburgh Application of lipid vehicles and use for drug delivery
US20050137153A1 (en) * 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
CA2489174C (en) 2002-07-10 2013-02-05 Thomas Tuschl Rna-interference by single-stranded rna molecules
EP1562971B1 (en) 2002-11-05 2014-02-12 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP2957568B1 (en) 2002-11-05 2016-12-21 Ionis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
EP2213738B1 (en) 2002-11-14 2012-10-10 Dharmacon, Inc. siRNA molecules targeting Bcl-2
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
WO2005070948A1 (en) * 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
CA2554212A1 (en) 2004-02-10 2005-08-25 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using multifunctional short interfering nucleic acid (multifunctional sina)
JPWO2005116204A1 (ja) 2004-05-11 2008-06-19 株式会社アルファジェン Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
CA2584207A1 (en) 2004-10-19 2006-04-27 Replicor, Inc. Antiviral oligonucleotides
ATE541928T1 (de) 2005-03-31 2012-02-15 Calando Pharmaceuticals Inc Inhibitoren der untereinheit 2 der ribonukleotidreduktase und ihre verwendungen
PL2314594T3 (pl) 2006-01-27 2014-12-31 Isis Pharmaceuticals Inc Zmodyfikowane w pozycji 6 analogi bicykliczne kwasów nukleinowych
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
WO2007091269A2 (en) 2006-02-08 2007-08-16 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
JP5761911B2 (ja) 2006-04-07 2015-08-12 イデラ ファーマシューティカルズ インコーポレイテッドＩｄｅｒａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｔｌｒ７およびｔｌｒ８に対する安定化免疫調節ｒｎａ（ｓｉｍｒａ）化合物
WO2007134181A2 (en) 2006-05-11 2007-11-22 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP2068886B1 (en) 2006-10-03 2013-09-18 Tekmira Pharmaceuticals Corporation Lipid containing formulations
US20100105134A1 (en) 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
DK2162538T3 (en) 2007-05-22 2016-06-06 Arcturus Therapeutics Inc Oligomers for THERAPY
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
ATE538127T1 (de) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
CN102921003B (zh) 2007-07-09 2014-11-26 艾德拉药物股份有限公司 稳定化免疫调控性rna(simra)化合物
CN102006890A (zh) 2007-12-04 2011-04-06 阿尔尼拉姆医药品有限公司 靶向脂质
EP2244728B1 (en) * 2008-01-21 2016-08-31 Dermadis Use of serine protease inhibitors in the treatment of skin diseases
WO2009134487A2 (en) 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of dsrna targeting the pcsk9 gene
US20110269814A1 (en) 2008-03-26 2011-11-03 Alnylam Pharamaceuticals, Inc. 2'-f modified rna interference agents
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US20110223665A1 (en) 2008-07-25 2011-09-15 Alnylam Pharmaceuticals, Inc. ENHANCEMENT OF siRNA SILENCING ACTIVITY USING UNIVERSAL BASES OR MISMATCHES IN THE SENSE STRAND
AU2009293636A1 (en) 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsRNA possessing modifications
CN102239259A (zh) 2008-12-03 2011-11-09 玛瑞纳生物技术有限公司 UsiRNA复合物
US20100184841A1 (en) 2008-12-18 2010-07-22 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
ES2804764T3 (es) 2009-06-01 2021-02-09 Halo Bio Rnai Therapeutics Inc Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos
EP3431076B1 (en) 2009-06-10 2021-10-06 Arbutus Biopharma Corporation Improved lipid formulation
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
WO2011005861A1 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
CN104673795A (zh) 2009-08-27 2015-06-03 艾德拉药物股份有限公司 用于抑制基因表达的组合物及其用途
US10913767B2 (en) 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
EP2563922A1 (en) 2010-04-26 2013-03-06 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2012178033A2 (en) * 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
HK1198766A1 (en) 2011-09-07 2015-06-05 玛瑞纳生物技术有限公司 Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
WO2013142514A1 (en) 2012-03-19 2013-09-26 Isis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
ES2730015T3 (es) 2013-05-01 2019-11-07 Ionis Pharmaceuticals Inc Composiciones y métodos para modular expresión de HBV y de TTR
RS57418B1 (sr) * 2013-05-22 2018-09-28 Alnylam Pharmaceuticals Inc Serpina1 irnk sastavi i postupci njihove primene
PL3444350T3 (pl) 2013-07-03 2022-03-21 Dicerna Pharmaceuticals, Inc. Sposoby i kompozycje do specyficznego hamowania alfa-1 antytrypsyny przez dwuniciowy rna
KR20150114637A (ko) * 2014-04-01 2015-10-13 부산대학교 산학협력단 ＳｅｒｐｉｎＡ1 유전자의 발현을 저해하는 저해제를 유효성분으로 포함하는 위암 치료용 약학적 조성물
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
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
US10450565B2 (en) 2017-01-10 2019-10-22 Arrowhead Pharmaceuticals, Inc. Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use

Also Published As

Publication number Publication date
US10995336B2 (en) 2021-05-04
EP3545093A1 (en) 2019-10-02
CA3044562A1 (en) 2018-05-31
KR20190082317A (ko) 2019-07-09
US20210269796A1 (en) 2021-09-02
WO2018098117A1 (en) 2018-05-31
US11725207B2 (en) 2023-08-15
CN110114463A (zh) 2019-08-09
PH12019550076A1 (en) 2020-01-20
RU2761667C2 (ru) 2021-12-13
CR20190294A (es) 2019-08-29
IL266746A (en) 2019-07-31
RU2019119242A (ru) 2020-12-25
UY37494A (es) 2018-06-29
TWI788312B (zh) 2023-01-01
RU2019119242A3 (es) 2021-02-24
TW201829443A (zh) 2018-08-16
ZA202103080B (en) 2023-03-29
WO2018098117A8 (en) 2021-05-14
BR112019010342A2 (pt) 2019-10-22
TW202313978A (zh) 2023-04-01
JP7348067B2 (ja) 2023-09-20
AU2017363604A1 (en) 2019-06-06
MA46923A (fr) 2019-10-02
MX2019005898A (es) 2020-01-27
US20190316127A1 (en) 2019-10-17
CN110114463B (zh) 2024-08-30
RU2021126115A (ru) 2021-09-16
SG10201913877WA (en) 2020-03-30
JP2020500929A (ja) 2020-01-16
DOP2019000132A (es) 2019-08-15
CL2019001388A1 (es) 2019-10-04
US20250043279A1 (en) 2025-02-06

Similar Documents

Publication Publication Date Title
CO2019005992A2 (es) Composiciones de arni de serpina1 y sus métodos de uso
UY35582A (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO
CL2021002998A1 (es) Composiciones de arni contra tmprss6 y métodos para su uso (divisional de solicitud n° 03440-2015)
AR121312A2 (es) AGENTES DE ARNi, COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR)
CL2021000084A1 (es) Inhibidores de pd-1/pd-l1
CO2020015314A2 (es) Composiciones de arni contra el angiotensinógeno (agt) y métodos para su uso
CO2019007258A2 (es) Arn guía modificados
CO2019011543A2 (es) Inhibidores de pd-1/pd-l1
DOP2014000107A (es) AGENTES DE iARN, COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR)
UY36357A (es) Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa))
MX2018005528A (es) Inhibidores de ret.
MX2018003353A (es) Terapias con celulas cart con una eficacia mejorada.
MX382901B (es) Composiciones de angiotensinogeno (agt) arni y métodos de uso de las mismas.
MX384205B (es) Metodos para usar agonistas de fxr.
AR102777A1 (es) COMPOSICIONES DE ARNi CONTRA TMPRSS6 Y MÉTODOS PARA SU USO
UY37800A (es) AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO
AR110225A1 (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO
MX2017014128A (es) 2-tiopirimidinonas.
CL2017002646A1 (es) Composiciones de arni de serpina1 y sus métodos de uso (divisional de solicitud n° 3441-2015).
CR20150644A (es) Composiciones de arni de serapina 1 y sus métodos de uso
AR096390A1 (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO
AR103676A1 (es) COMPOSICIONES DE ARNi QUE CONTIENEN UN DOMINIO FOSFOLIPASA TIPO PATATINA 3 (PNPLA3) Y SUS MÉTODOS DE USO
CL2017000872A1 (es) Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa)
AR104542A1 (es) COMPOSICIONES DE ARNi CONTRA FACTOR XII (FACTOR DE HAGEMAN) (F12), CALICREÍNA B, PLASMÁTICA (FACTOR DE FLETCHER) 1 (KLKB1), Y QUININÓGENO 1 (KNG1) Y MÉTODOS DE USO DE LOS MISMOS