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PE20160500A1 - Moduladores del factor del complemento b - Google Patents

Moduladores del factor del complemento b

Info

Publication number
PE20160500A1
PE20160500A1 PE2016000376A PE2016000376A PE20160500A1 PE 20160500 A1 PE20160500 A1 PE 20160500A1 PE 2016000376 A PE2016000376 A PE 2016000376A PE 2016000376 A PE2016000376 A PE 2016000376A PE 20160500 A1 PE20160500 A1 PE 20160500A1
Authority
PE
Peru
Prior art keywords
modified
seq
antisense compounds
nucleobase
compounds
Prior art date
Application number
PE2016000376A
Other languages
English (en)
Inventor
Susan M Freier
Andrew T Watt
Michael L Mccaleb
Tamar R Grossman
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 PE20160500A1 publication Critical patent/PE20160500A1/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—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
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    • 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)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (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 compuestos antisentido dirigidos a un acido nucleico (SEQ ID NO: 1 o SEQ ID NO: 2) que expresa al Factor del Complemento B (CFB), dichos compuestos comprenden un oligonucleotido modificado que consiste en 8 a 80 nucleosidos enlazados cuya secuencia de nucleobases comprende una parte de al menos 8 nucleobases contiguas 100% complementarias a una parte de una region de igual longitud en la SEQ ID NO: 1 o SEQ ID NO: 2. El oligonucleotido modificado comprende al menos un enlace internucleosido modificado, al menos un azucar modificado o al menos una nucleobase modificada. En un aspecto preferido, el enlace internucleosido modificado es un enlace fosforotioato; el azucar modificado es un azucar biciclico seleccionado entre LNA, ENA y cEt; y la nucleobase modificada es 5–metilcitosina. En otro aspecto preferido, los compuestos antisentido de la invencion comprenden un oligonucleotido modificado cuya secuencia de nucleobases comprende cualquiera de las SEQ ID NOS: 198, 228, 237, 440, 444, 448, 450, 453, 455, 549 y 598. Tambien se refiere a composiciones farmaceuticas que comprenden los compuestos antisentido de la invencion. Dichos compuestos inhiben la expresion de CFB, por lo cual son utiles en la terapia de enfermedades asociadas con una desregulacion de la via alternativa del complemento, tales como la degeneracion macular relacionada con la edad (AMD), nefritis lupica, lupus eritematoso sistemico, glomerulonefritis C3 (C3GN), nefropatia CFHR5 o sindrome uremico hemolitico atipico, entre otros
PE2016000376A 2013-09-13 2014-09-12 Moduladores del factor del complemento b PE20160500A1 (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
PE20160500A1 true PE20160500A1 (es) 2016-05-21

Family

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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 After (1)

Application Number Title Priority Date Filing Date
PE2018003111A PE20190354A1 (es) 2013-09-13 2014-09-12 Moduladores del factor del complemento b

Country Status (32)

Country Link
US (5) US20160222389A1 (es)
EP (2) EP3603677A1 (es)
JP (3) JP6666250B2 (es)
KR (1) KR102381790B1 (es)
CN (2) CN105744959B (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
WO2007089584A2 (en) 2006-01-26 2007-08-09 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
EP2410054B2 (en) 2006-10-18 2019-12-11 Ionis Pharmaceuticals, Inc. Antisense compounds
CN111705058A (zh) 2009-09-11 2020-09-25 Ionis制药公司 亨廷顿表达的调节
EP3067421B1 (en) 2011-02-08 2018-10-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
RS60796B1 (sr) 2013-05-01 2020-10-30 Ionis Pharmaceuticals Inc Kompozicije i postupci za modulaciju ekspresije apolipoproteina (a)
SG11201601445XA (en) * 2013-09-13 2016-03-30 Ionis Pharmaceuticals Inc Modulators of complement factor b
IL314045A (en) * 2013-12-12 2024-09-01 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
SI3137596T1 (sl) * 2014-05-01 2019-08-30 Ionis Pharmaceuticals, Inc. Sestavki in postopki za moduliranje izražanje faktorja B komplementa
TW201730341A (zh) * 2016-02-05 2017-09-01 Kyowa Hakko Kirin Co Ltd 抑制補體b因子之表現之反義寡核苷酸
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
WO2018117253A1 (ja) * 2016-12-23 2018-06-28 協和発酵キリン株式会社 補体ｂ因子の発現を抑制する核酸
TW201909925A (zh) * 2017-08-02 2019-03-16 日商協和醱酵麒麟有限公司 核酸複合體
CA3078971A1 (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
JP7676377B2 (ja) 2019-10-22 2025-05-14 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 ｉＲＮＡ組成物およびその使用方法
KR20230017789A (ko) * 2020-04-30 2023-02-06 알닐람 파마슈티칼스 인코포레이티드 보체 인자 B (CFB) iRNA 조성물 및 이의 사용 방법
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
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
KR20240035563A (ko) * 2021-07-17 2024-03-15 서나오믹스, 인크. 생성물 및 조성물
CN118234861A (zh) 2021-09-02 2024-06-21 赛伦斯治疗有限责任公司 用于抑制细胞中的补体因子b(cfb)表达的核酸
MX2024004750A (es) * 2021-10-29 2024-05-13 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) contra el factor b del complemento (cfb) y sus metodos de uso.
WO2023129496A2 (en) * 2021-12-27 2023-07-06 Apellis Pharmaceuticals, Inc. Rnas for complement inhibition
EP4471021A4 (en) 2022-01-26 2025-04-30 Shanghai Meiyue Biotech Development Co., Ltd. Salt type and crystal form of complement factor b inhibitor, and preparation method therefor and application thereof
AU2023339335A1 (en) 2022-09-10 2025-04-03 Hansoh Bio Llc 2-substituted piperidine derivatives, preparation methods and medicinal uses thereof
EP4611768A1 (en) * 2022-11-02 2025-09-10 Ionis Pharmaceuticals, Inc. Methods for modulating complement factor b expression
WO2024222852A1 (zh) 2023-04-27 2024-10-31 上海翰森生物医药科技有限公司 一种哌啶基吲哚化合物的盐及其制备方法
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
WO2025002401A1 (zh) * 2023-06-28 2025-01-02 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
TW202500169A (zh) * 2023-06-28 2025-01-01 大陸商上海舶望製藥有限公司 用於抑制補體因子b(cfb)表達的組合物和方法
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
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
EP0497875B1 (en) 1989-10-24 2000-03-22 Isis Pharmaceuticals, Inc. 2' modified oligonucleotides
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
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
EP0455905B1 (en) 1990-05-11 1998-06-17 Microprobe Corporation Dipsticks for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
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
JPH08508492A (ja) 1993-03-30 1996-09-10 スターリング ウィンスロップ インコーポレイティド 非環式ヌクレオシド類似体及びそれらを含むオリゴヌクレオチド配列
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
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
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
WO2000021559A2 (en) * 1998-10-09 2000-04-20 Musc Foundation For Research Development Blocking factor b to treat complement-mediated immune disease
AU781598B2 (en) 1999-04-21 2005-06-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibiting the function of polynucleotide sequences
DK1178999T3 (da) 1999-05-04 2007-08-06 Santaris Pharma As L-RIBO-LNA-analoger
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
ES2261270T3 (es) 1999-12-30 2006-11-16 K.U. LEUVEN RESEARCH & DEVELOPMENT Acidos nucleicos que contienen ciclohexeno.
JP2005504020A (ja) 2001-07-03 2005-02-10 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 ヌクレアーゼ耐性キメラオリゴヌクレオチド
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
EP1554377B1 (en) * 2002-09-13 2012-11-07 Life Technologies Corporation Thermostable reverse transcriptases and uses thereof
WO2004044139A2 (en) 2002-11-05 2004-05-27 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1560931B1 (en) * 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
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
WO2004103288A2 (en) * 2003-05-13 2004-12-02 New York Society For The Ruptured And Crippled Maintaining The Hospital For Special Surgery Method of preventing recurrent miscarriages
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
CA2538252C (en) 2003-09-18 2014-02-25 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
PT1713503E (pt) * 2004-02-10 2013-11-14 Nat Jewish Health Inibição do factor b, a via alternativa do complemento e métodos relacionados
EP1752536A4 (en) * 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
CA2568735A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
US7919472B2 (en) 2004-09-17 2011-04-05 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
EP1812569A2 (en) 2004-11-08 2007-08-01 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
EP1984381B1 (en) 2006-01-27 2010-09-29 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
EP2066684B1 (en) 2006-05-11 2012-07-18 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
JP5352462B2 (ja) * 2006-09-22 2013-11-27 ダーマコン， インコーポレイテッド 二本鎖オリゴヌクレオチド複合体、ｒｎａ干渉による遺伝子サイレンシング方法、および医薬品組成物
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
JP5332064B2 (ja) * 2007-03-01 2013-11-06 ウェルスタット イムノセラピューティクス， エルエルシー 炎症により特徴付けられる疾患の治療
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
DK2176280T4 (en) 2007-07-05 2015-07-20 Isis Pharmaceuticals Inc 6-Disubstituerede bicykliske nukleinsyreanaloge
WO2010005310A2 (en) * 2008-07-10 2010-01-14 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Complement antagonists and uses thereof
EP2361256B1 (en) 2008-09-24 2013-04-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
KR101900770B1 (ko) * 2010-07-19 2018-09-20 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
EP2697244B1 (en) * 2011-04-13 2017-05-17 Ionis Pharmaceuticals, Inc. Antisense modulation of ptp1b expression
SG11201601445XA (en) 2013-09-13 2016-03-30 Ionis Pharmaceuticals Inc Modulators of complement factor b
SI3137596T1 (sl) * 2014-05-01 2019-08-30 Ionis Pharmaceuticals, Inc. Sestavki in postopki za moduliranje izražanje faktorja B komplementa

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