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PE20190354A1 - Moduladores del factor del complemento b - Google Patents

Moduladores del factor del complemento b

Info

Publication number
PE20190354A1
PE20190354A1 PE2018003111A PE2018003111A PE20190354A1 PE 20190354 A1 PE20190354 A1 PE 20190354A1 PE 2018003111 A PE2018003111 A PE 2018003111A PE 2018003111 A PE2018003111 A PE 2018003111A PE 20190354 A1 PE20190354 A1 PE 20190354A1
Authority
PE
Peru
Prior art keywords
wing segment
segment
linked
complement
nucleobase sequence
Prior art date
Application number
PE2018003111A
Other languages
English (en)
Inventor
Tamar R Grossman
Michael L Mccaleb
Andrew T Watt
Susan M Freier
Original Assignee
Ionis 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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of PE20190354A1 publication Critical patent/PE20190354A1/es

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    • 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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic 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
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21047—Alternative-complement-pathway C3/C5 convertase (3.4.21.47), i.e. properdin factor B
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Peptides Or Proteins (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Epidemiology (AREA)
  • Amplifiers (AREA)
  • Optical Communication System (AREA)

Abstract

Se refiere a un compuesto que comprende un oligonucleotido modificado que consiste de 16 a 30 nucleotidos enlazados y que tiene una secuencia de nucleobases que comprende la secuencia de nucleobases de cualquiera de las SEQ ID NO: 6 A 197; 199 a 227, 229 a 236, 238 a 439, entre otros; en donde el oligonucleotido modificado comprende: (a) un segmento hueco que consiste en desoxinucleosidos enlazados; (b) un segmento de ala 5´ que consiste en nucleosidos enlazados; y (c) un segmento de ala 3´ que consiste en nucleosidos enlazados; en donde el segmento de hueco se encuentra ubicado inmediatamente adyacente a y entre el segmento de ala 5´ y el segmento de ala 3´, y donde cada segmento ala comprende un azucar biciclico modificado, la cual se selecciona del grupo que consiste en: 4´-(CH2)-O-2´ (LNA); 4´- (CH2)2-O-2´ (ENA); y 4´- CH(CH3)-O-2´ (cEt), siendo la preferida 2´-O-metoxietilo. Dicha composicion es util para prevenir, mejorar o tratar una enfermedad asociada con la desregulacion de la via alternativa de complemento mediante la administracion de un inhibidor especifico del factor del complemento B (CFB) a un sujeto.
PE2018003111A 2013-09-13 2014-09-12 Moduladores del factor del complemento b PE20190354A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201361877624P 2013-09-13 2013-09-13

Publications (1)

Publication Number Publication Date
PE20190354A1 true PE20190354A1 (es) 2019-03-07

Family

ID=52666522

Family Applications (2)

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PE2016000376A PE20160500A1 (es) 2013-09-13 2014-09-12 Moduladores del factor del complemento b
PE2018003111A PE20190354A1 (es) 2013-09-13 2014-09-12 Moduladores del factor del complemento b

Family Applications Before (1)

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Country Status (32)

Country Link
US (5) US20160222389A1 (es)
EP (2) EP3043827B1 (es)
JP (3) JP6666250B2 (es)
KR (1) KR102381790B1 (es)
CN (2) CN112359042A (es)
AU (2) AU2014318580B2 (es)
BR (1) BR112016004671B1 (es)
CA (1) CA2921842A1 (es)
CL (2) CL2016000606A1 (es)
CR (1) CR20160170A (es)
DK (1) DK3043827T3 (es)
DO (1) DOP2016000063A (es)
EA (1) EA035433B1 (es)
ES (1) ES2745758T3 (es)
HR (1) HRP20191600T1 (es)
HU (1) HUE045109T2 (es)
IL (1) IL244095A0 (es)
LT (1) LT3043827T (es)
MA (1) MA38959A1 (es)
MX (2) MX371518B (es)
MY (1) MY181251A (es)
PE (2) PE20160500A1 (es)
PH (1) PH12016500443A1 (es)
PL (1) PL3043827T3 (es)
PT (1) PT3043827T (es)
RS (1) RS59252B1 (es)
RU (1) RU2662967C2 (es)
SG (2) SG11201601445XA (es)
SI (1) SI3043827T1 (es)
UA (1) UA119046C2 (es)
WO (1) WO2015038939A2 (es)
ZA (1) ZA201601236B (es)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3210633B1 (en) 2006-01-26 2019-06-19 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
EP2092065B2 (en) 2006-10-18 2019-07-24 Ionis Pharmaceuticals, Inc. Antisense compounds
KR101774526B1 (ko) 2009-09-11 2017-09-04 아이오니스 파마수티컬즈, 인코포레이티드 헌팅틴 발현의 조절
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
BR112015027369B1 (pt) 2013-05-01 2021-06-08 Ionis Pharmaceuticals, Inc compostos compreendendo um oligonucleotídeo modificado e um grupo de conjugado, composição compreendendo os referidos compostos e usos dos mesmos
MX371518B (es) * 2013-09-13 2020-01-31 Ionis Pharmaceuticals Inc Moduladores del factor del complemento b.
IL314045A (en) * 2013-12-12 2024-09-01 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
EP4219718A3 (en) * 2014-05-01 2024-01-10 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating complement factor b expression
WO2017135397A1 (ja) * 2016-02-05 2017-08-10 協和発酵キリン株式会社 補体ｂ因子の発現を抑制するアンチセンスオリゴヌクレオチド
JP7057290B2 (ja) 2016-06-27 2022-04-19 アキリオン ファーマシューティカルズ，インコーポレーテッド 医学的障害を治療するためのキナゾリン及びインドール化合物
EP3548005A4 (en) 2016-11-29 2020-06-17 Puretech Health LLC Exosomes for delivery of therapeutic agents
TW201827596A (zh) * 2016-12-23 2018-08-01 日商協和醱酵麒麟有限公司 抑制補體b因子之表現之核酸
WO2019027015A1 (ja) * 2017-08-02 2019-02-07 協和発酵キリン株式会社 核酸複合体
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2020109343A1 (en) 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Combination therapy for treatment of macular degeneration
BR112022007540A2 (pt) 2019-10-22 2022-07-12 Alnylam Pharmaceuticals Inc Composições de irna de componente complementar c3 e métodos de uso das mesmas
MX2022013606A (es) * 2020-04-30 2023-01-16 Alnylam Pharmaceuticals Inc Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas.
AU2021323300B2 (en) 2020-08-07 2025-12-18 Shanghai Meiyue Biotech Development Co., Ltd. Complement factor B inhibitor, and pharmaceutical composition thereof, preparation method therefor and use thereof
WO2022028507A1 (zh) 2020-08-07 2022-02-10 上海美悦生物科技发展有限公司 一种杂环类化合物、其制备方法及用途
JP2024525868A (ja) 2021-07-17 2024-07-12 サーナオミクス インコーポレイテッド 産物及び組成物
IL311146A (en) 2021-09-02 2024-04-01 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
JP2024541989A (ja) 2021-10-29 2024-11-13 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
US20250075209A1 (en) * 2021-12-27 2025-03-06 Apellis Pharmaceuticals, Inc. Rnas for complement inhibition
WO2023143293A1 (zh) 2022-01-26 2023-08-03 上海美悦生物科技发展有限公司 补体因子b抑制剂的盐型、晶型及其制备方法和应用
WO2024051849A1 (en) 2022-09-10 2024-03-14 Jiangsu Hansoh Pharmaceutical Group Co., Ltd. 2-substituted piperidine derivatives, preparation methods and medicinal uses thereof
CN120379677A (zh) * 2022-11-02 2025-07-25 Ionis制药公司 用于调节补体因子b表达的方法
TW202506657A (zh) 2023-04-27 2025-02-16 大陸商上海翰森生物醫藥科技有限公司 一種哌啶基吲哚化合物的鹽及其製備方法
CN121311588A (zh) * 2023-06-02 2026-01-09 维亚臻生物技术（苏州）有限公司 一种用于免疫类疾病的双链核苷酸化合物及其应用
CN118620964A (zh) * 2023-06-28 2024-09-10 百奥赛图(北京)医药科技股份有限公司 一种cfb基因修饰的非人动物
AU2024307421A1 (en) * 2023-06-28 2026-02-12 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of complement factor b (cfb)
WO2025122669A1 (en) * 2023-12-05 2025-06-12 Regeneron Pharmaceuticals, Inc. Non-human animals having a humanized complement factor b gene

Family Cites Families (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
JPH05504552A (ja) 1989-10-24 1993-07-15 ギリアド　サイエンシズ，インコーポレイテッド ２’位が改変されたオリゴヌクレオチド
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
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
EP0745689A3 (en) 1990-05-11 1996-12-11 Microprobe Corporation A dipstick for a nucleic acid hybridization assay
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
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
EP0673559A1 (en) 1992-12-14 1995-09-27 Honeywell Inc. Motor system with individually controlled redundant windings
DK0691968T3 (da) 1993-03-30 1998-02-23 Sanofi Sa Acykliske nukleosid-analoge og oligonukleotidsekvenser indeholdende disse
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
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
IL135000A0 (en) 1997-09-12 2001-05-20 Exiqon As Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
AU1705100A (en) * 1998-10-09 2000-05-01 Musc Foundation For Research Development Blocking factor b to treat complement-mediated immune disease
WO2000063364A2 (en) 1999-04-21 2000-10-26 American Home Products Corporation Methods and compositions for inhibiting the function of polynucleotide sequences
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
DE60027203T2 (de) 1999-12-30 2007-01-04 K.U. Leuven Research & Development Cyclohexennukleinsäuren
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
EP2325303B1 (en) * 2002-09-13 2013-11-06 Life Technologies Corporation Thermostable reverse transcriptases and uses thereof
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
AU2003291755A1 (en) 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
PT2284266E (pt) * 2002-11-14 2013-12-17 Thermo Fisher Scient Biosciences Inc Siarn contra tp53
US6673661B1 (en) 2002-12-20 2004-01-06 Taiwan Semiconductor Manufacturing Co., Ltd. Self-aligned method for forming dual gate thin film transistor (TFT) device
US20070123466A1 (en) * 2003-05-13 2007-05-31 New York Society For The Ruptured And Crippled Maintaining The Hospital For Special Surgery Method of treating recurrent miscarriages
WO2005021570A1 (ja) 2003-08-28 2005-03-10 Gene Design, Inc. Ｎ−０結合性架橋構造型新規人工核酸
WO2005027962A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
US7959919B2 (en) * 2003-11-19 2011-06-14 Novelmed Therapeutics, Inc. Method of inhibiting factor B-mediated complement activation
ES2432112T3 (es) 2004-02-10 2013-11-29 The Regents Of The University Of Colorado, A Body Corporate Inhibición del factor B, de la vía alternativa del complemento y métodos relacionados
CN101052717A (zh) * 2004-05-11 2007-10-10 α基因株式会社 诱发rna干扰的多核苷酸以及使用该多核苷酸的基因表达抑制方法
CA2568735A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
EP1799859B1 (en) 2004-09-17 2014-07-02 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
WO2006047842A2 (en) 2004-11-08 2006-05-11 K.U. Leuven Research And Development Modified nucleosides for rna interference
GB0521716D0 (en) * 2005-10-25 2005-11-30 Genomica Sau Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases
EP2332951A3 (en) 2006-01-27 2014-08-13 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
US8252755B2 (en) * 2006-09-22 2012-08-28 Dharmacon, Inc. Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
CN101589143A (zh) * 2006-11-27 2009-11-25 Isis药物公司 用于治疗高胆固醇血症的方法
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
US20100120665A1 (en) * 2007-03-01 2010-05-13 Advanced Vision Therapies, Inc. Treatment of diseases characterized by inflammation
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
AU2009270020B2 (en) * 2008-07-10 2014-09-25 Regenesance B.V. Complement antagonists and uses thereof
EP2361256B1 (en) 2008-09-24 2013-04-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
EP3173419A1 (en) 2010-04-28 2017-05-31 Ionis Pharmaceuticals, Inc. Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
PL3031920T3 (pl) * 2010-07-19 2020-01-31 Ionis Pharmaceuticals, Inc. Modulowanie ekspresji kinazy białkowej dystrofii miotonicznej (dmpk)
CN105886506A (zh) * 2011-04-13 2016-08-24 Isis制药公司 Ptp1b 表达的反义调节
MX371518B (es) 2013-09-13 2020-01-31 Ionis Pharmaceuticals Inc Moduladores del factor del complemento b.
EP4219718A3 (en) * 2014-05-01 2024-01-10 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating complement factor b expression

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