JP6666250B2 - 補体ｂ因子の調節薬 - Google Patents

補体ｂ因子の調節薬 Download PDF

Info

Publication number
JP6666250B2
JP6666250B2 JP2016542824A JP2016542824A JP6666250B2 JP 6666250 B2 JP6666250 B2 JP 6666250B2 JP 2016542824 A JP2016542824 A JP 2016542824A JP 2016542824 A JP2016542824 A JP 2016542824A JP 6666250 B2 JP6666250 B2 JP 6666250B2
Authority
JP
Japan
Prior art keywords
certain embodiments
antisense
modified
compound
nucleic acid
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
JP2016542824A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016533765A (ja
JP2016533765A5 (enExample
Inventor
グロスマン，タマル・アール
マッケレーブ，マイケル・エル
ワット，アンドリュー・ティー
フレアー，スーザン・エム
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.)
Ionis Pharmaceuticals Inc
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 JP2016533765A publication Critical patent/JP2016533765A/ja
Publication of JP2016533765A5 publication Critical patent/JP2016533765A5/ja
Application granted granted Critical
Publication of JP6666250B2 publication Critical patent/JP6666250B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • C12N2310/323—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

Landscapes

  • 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)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (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)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Epidemiology (AREA)
  • Amplifiers (AREA)
  • Optical Communication System (AREA)
JP2016542824A 2013-09-13 2014-09-12 補体ｂ因子の調節薬 Active JP6666250B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361877624P 2013-09-13 2013-09-13
US61/877,624 2013-09-13
PCT/US2014/055458 WO2015038939A2 (en) 2013-09-13 2014-09-12 Modulators of complement factor b

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019201990A Division JP7158363B2 (ja) 2013-09-13 2019-11-07 補体ｂ因子の調節薬

Publications (3)

Publication Number Publication Date
JP2016533765A JP2016533765A (ja) 2016-11-04
JP2016533765A5 JP2016533765A5 (enExample) 2017-10-19
JP6666250B2 true JP6666250B2 (ja) 2020-03-13

Family

ID=52666522

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2016542824A Active JP6666250B2 (ja) 2013-09-13 2014-09-12 補体ｂ因子の調節薬
JP2019201990A Active JP7158363B2 (ja) 2013-09-13 2019-11-07 補体ｂ因子の調節薬
JP2022011551A Active JP7297112B2 (ja) 2013-09-13 2022-01-28 補体ｂ因子の調節薬

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2019201990A Active JP7158363B2 (ja) 2013-09-13 2019-11-07 補体ｂ因子の調節薬
JP2022011551A Active JP7297112B2 (ja) 2013-09-13 2022-01-28 補体ｂ因子の調節薬

Country Status (32)

Country Link
US (5) US20160222389A1 (enExample)
EP (2) EP3603677A1 (enExample)
JP (3) JP6666250B2 (enExample)
KR (1) KR102381790B1 (enExample)
CN (2) CN105744959B (enExample)
AU (2) AU2014318580B2 (enExample)
BR (1) BR112016004671B1 (enExample)
CA (1) CA2921842A1 (enExample)
CL (2) CL2016000606A1 (enExample)
CR (1) CR20160170A (enExample)
DK (1) DK3043827T3 (enExample)
DO (1) DOP2016000063A (enExample)
EA (1) EA035433B1 (enExample)
ES (1) ES2745758T3 (enExample)
HR (1) HRP20191600T1 (enExample)
HU (1) HUE045109T2 (enExample)
IL (1) IL244095A0 (enExample)
LT (1) LT3043827T (enExample)
MA (1) MA38959A1 (enExample)
MX (2) MX371518B (enExample)
MY (1) MY181251A (enExample)
PE (2) PE20160500A1 (enExample)
PH (1) PH12016500443A1 (enExample)
PL (1) PL3043827T3 (enExample)
PT (1) PT3043827T (enExample)
RS (1) RS59252B1 (enExample)
RU (1) RU2662967C2 (enExample)
SG (2) SG10201806787VA (enExample)
SI (1) SI3043827T1 (enExample)
UA (1) UA119046C2 (enExample)
WO (1) WO2015038939A2 (enExample)
ZA (1) ZA201601236B (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020036600A (ja) * 2013-09-13 2020-03-12 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 補体ｂ因子の調節薬

Families Citing this family (30)

* 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
AU2007310989B2 (en) 2006-10-18 2014-05-29 Isis Pharmaceuticals, Inc. Antisense compounds
EP2475675B1 (en) 2009-09-11 2016-11-16 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
KR20240170595A (ko) * 2013-12-12 2024-12-03 알닐람 파마슈티칼스 인코포레이티드 보체 성분 ｉＲＮＡ 조성물 및 이의 이용 방법
KR102369736B1 (ko) * 2014-05-01 2022-03-02 아이오니스 파마수티컬즈, 인코포레이티드 보체 인자 b 발현을 조절하기 위한 조성물 및 방법
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
KR20190036520A (ko) 2016-06-27 2019-04-04 아칠리온 파르마세우티칼스 인코포레이티드 의학적 장애를 치료하기 위한 퀴나졸린 및 인돌 화합물
CA3043768A1 (en) 2016-11-29 2018-06-07 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
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
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
IL292360A (en) 2019-10-22 2022-06-01 Alnylam Pharmaceuticals Inc IRNA components c3 supplementary component and methods of using them
CA3181198A1 (en) * 2020-04-30 2021-11-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
EP4194451A4 (en) 2020-08-07 2024-07-24 Shanghai Meiyue Biotech Development Co., Ltd. SUPPLEMENT FACTOR B INHIBITOR AND PHARMACEUTICAL COMPOSITION THEREOF, CORRESPONDING PREPARATION METHOD AND ASSOCIATED USE
EP4194449A4 (en) 2020-08-07 2024-03-20 Shanghai Meiyue Biotech Development Co., Ltd. Heterocyclic compound, preparation method therefor and use thereof
CA3225937A1 (en) 2021-07-17 2023-02-16 Dmitry Samarsky Products and compositions
WO2023031359A1 (en) 2021-09-02 2023-03-09 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
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
US20250075209A1 (en) * 2021-12-27 2025-03-06 Apellis Pharmaceuticals, Inc. Rnas for complement inhibition
TWI843416B (zh) 2022-01-26 2024-05-21 大陸商上海美悦生物科技發展有限公司 補體因子b抑制劑的鹽型、晶型及其製備方法和用途
JP2025530222A (ja) 2022-09-10 2025-09-11 江▲蘇▼豪森▲薬▼▲業▼集▲団▼有限公司 ２－置換ピペリジン誘導体、その製造方法及び医薬的用途
JP2025537139A (ja) * 2022-11-02 2025-11-14 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 補体因子ｂの発現を調節するための方法
AU2024261191A1 (en) 2023-04-27 2025-11-06 Jiangsu Hansoh Pharmaceutical Group Co., Ltd. Salt of piperidylindole compound and preparation method therefor
WO2024245424A1 (zh) * 2023-06-02 2024-12-05 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
WO2025002299A1 (en) * 2023-06-28 2025-01-02 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of complement factor b (cfb)
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
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
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
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral 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
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
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
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
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
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
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
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
IL145778A0 (en) 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
DE60033927T2 (de) 1999-05-04 2007-11-29 Santaris Pharma A/S L-ribo-lna analoge
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
ATE322493T1 (de) 1999-12-30 2006-04-15 Leuven K U Res & Dev Cyclohexennukleinsäuren
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
JP4634799B2 (ja) * 2002-09-13 2011-02-16 ライフ テクノロジーズ コーポレーション 耐熱性逆転写酵素およびその使用法
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
AU2003290596B2 (en) 2002-11-05 2011-05-12 Isis Pharmaceuticals, Inc. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP2314691A3 (en) 2002-11-14 2012-01-18 Dharmacon, Inc. Fuctional 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
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
JP5379347B2 (ja) 2003-09-18 2013-12-25 アイシス ファーマシューティカルズ， インコーポレーテッド ４’−チオヌクレオシドおよびオリゴマー化合物
US7959919B2 (en) 2003-11-19 2011-06-14 Novelmed Therapeutics, Inc. Method of inhibiting factor B-mediated complement activation
BRPI0506629A (pt) * 2004-02-10 2007-05-02 Univ Colorado inibição do fator b, a via alternativa do sistema complemento e 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
EP1765416A4 (en) 2004-06-03 2010-03-24 Isis Pharmaceuticals Inc DOUBLE-STRONG COMPOSITIONS WITH DIFFERENTLY MODIFIED STRANDS FOR USE IN GENE MODULATION
AU2005286738A1 (en) 2004-09-17 2006-03-30 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
PL2314594T3 (pl) 2006-01-27 2014-12-31 Isis Pharmaceuticals Inc Zmodyfikowane w pozycji 6 analogi bicykliczne kwasów nukleinowych
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP2081949B1 (en) * 2006-09-22 2014-12-10 GE Healthcare Dharmacon, Inc. Tripartite 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
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
KR20090122465A (ko) * 2007-03-01 2009-11-30 어드벤스드 비젼 테라피스, 인코포레이티드 염증성 질환의 치료
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US8278426B2 (en) 2007-06-08 2012-10-02 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
US8703730B2 (en) * 2008-07-10 2014-04-22 Regenesance B.V. Complement antagonists and uses thereof
WO2010036696A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
CN103154014B (zh) 2010-04-28 2015-03-25 Isis制药公司 修饰核苷、其类似物以及由它们制备的寡聚化合物
EP3031920B1 (en) * 2010-07-19 2019-08-21 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (dmpk) expression
AU2012242642A1 (en) * 2011-04-13 2013-05-02 Ionis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
HRP20191600T1 (hr) 2013-09-13 2019-11-29 Ionis Pharmaceuticals Inc Modulatori faktora b komplementa
KR102369736B1 (ko) * 2014-05-01 2022-03-02 아이오니스 파마수티컬즈, 인코포레이티드 보체 인자 b 발현을 조절하기 위한 조성물 및 방법

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020036600A (ja) * 2013-09-13 2020-03-12 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 補体ｂ因子の調節薬
JP2022058784A (ja) * 2013-09-13 2022-04-12 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 補体ｂ因子の調節薬
JP7158363B2 (ja) 2013-09-13 2022-10-21 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 補体ｂ因子の調節薬
JP7297112B2 (ja) 2013-09-13 2023-06-23 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 補体ｂ因子の調節薬

Also Published As

Publication number Publication date
PT3043827T (pt) 2019-09-26
IL244095A0 (en) 2016-04-21
US20160222389A1 (en) 2016-08-04
US20200149047A1 (en) 2020-05-14
CL2016000606A1 (es) 2017-02-17
HUE045109T2 (hu) 2019-12-30
RU2016113763A (ru) 2017-10-16
PL3043827T3 (pl) 2020-03-31
AU2020201763A1 (en) 2020-04-02
LT3043827T (lt) 2019-08-12
DK3043827T3 (da) 2019-08-26
KR102381790B1 (ko) 2022-03-31
US11926830B2 (en) 2024-03-12
HRP20191600T1 (hr) 2019-11-29
EA035433B1 (ru) 2020-06-15
ES2745758T3 (es) 2020-03-03
CN112359042A (zh) 2021-02-12
MY181251A (en) 2020-12-21
EP3603677A1 (en) 2020-02-05
JP2022058784A (ja) 2022-04-12
KR20160054595A (ko) 2016-05-16
HK1225638A1 (en) 2017-09-15
WO2015038939A2 (en) 2015-03-19
CR20160170A (es) 2016-07-15
CL2018002334A1 (es) 2018-12-14
US20240247268A1 (en) 2024-07-25
US20220243209A1 (en) 2022-08-04
MX2020001258A (es) 2020-03-20
DOP2016000063A (es) 2016-06-01
AU2014318580A1 (en) 2016-03-03
EP3043827A2 (en) 2016-07-20
MA38959A1 (fr) 2017-08-31
PE20190354A1 (es) 2019-03-07
EA201690582A1 (ru) 2016-07-29
EP3043827B1 (en) 2019-07-03
PH12016500443A1 (en) 2016-05-16
ZA201601236B (en) 2017-05-31
US20190048351A1 (en) 2019-02-14
MX371518B (es) 2020-01-31
MX2016003263A (es) 2016-12-08
JP7297112B2 (ja) 2023-06-23
JP7158363B2 (ja) 2022-10-21
CN105744959A (zh) 2016-07-06
CN105744959B (zh) 2020-12-01
UA119046C2 (uk) 2019-04-25
CA2921842A1 (en) 2015-03-19
SG10201806787VA (en) 2018-09-27
RS59252B1 (sr) 2019-10-31
RU2662967C2 (ru) 2018-07-31
WO2015038939A3 (en) 2015-07-02
EP3043827A4 (en) 2017-05-24
SG11201601445XA (en) 2016-03-30
AU2014318580B2 (en) 2019-12-12
JP2016533765A (ja) 2016-11-04
JP2020036600A (ja) 2020-03-12
SI3043827T1 (sl) 2019-08-30
BR112016004671B1 (pt) 2020-12-29
PE20160500A1 (es) 2016-05-21

Similar Documents

Publication Publication Date Title
JP7297112B2 (ja) 補体ｂ因子の調節薬
EP3608406B1 (en) Compositions and methods for modulating complement factor b expression
CN105814204B (zh) 促血管生成素样3表达的调节
CA2978100C (en) Compounds and methods for modulating tmprss6 expression
AU2014312196C1 (en) Modulation of prekallikrein (PKK) expression
HK40022442A (en) Modulators of complement factor b
HK1225638B (en) Modulators of complement factor b
HK1234433B (en) Compositions and methods for modulating complement factor b expression
HK1234433A1 (en) Compositions and methods for modulating complement factor b expression

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170905

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20170905

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20180827

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20181115

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190227

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20190227

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20190708

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191107

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20191114

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20200122

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20200220

R150 Certificate of patent or registration of utility model

Ref document number: 6666250

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250