MA38959A1 - Modulateurs du facteur b du complément - Google Patents

Modulateurs du facteur b du complément

Info

Publication number
MA38959A1
MA38959A1 MA38959A MA38959A MA38959A1 MA 38959 A1 MA38959 A1 MA 38959A1 MA 38959 A MA38959 A MA 38959A MA 38959 A MA38959 A MA 38959A MA 38959 A1 MA38959 A1 MA 38959A1
Authority
MA
Morocco
Prior art keywords
modulators
complement factor
cfb
deregulation
ameliorating
Prior art date
Application number
MA38959A
Other languages
English (en)
Inventor
Susan M Freier
Tamar R Grossman
Michael L Mccaleb
Andrew T Watt
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 MA38959A1 publication Critical patent/MA38959A1/fr

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)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (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)
  • Investigating Or Analysing Biological Materials (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Epidemiology (AREA)
  • Amplifiers (AREA)
  • Optical Communication System (AREA)

Abstract

L'invention concerne des procédés, des composés et des compositions permettant de traiter, prévenir ou améliorer une maladie associée à la dérégulation de la voie d'activation alterne du complément par l'administration d'un inhibiteur spécifique du facteur b du complément (cfb) à un sujet.
MA38959A 2013-09-13 2014-09-12 Modulateurs du facteur b du complément MA38959A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361877624P 2013-09-13 2013-09-13
PCT/US2014/055458 WO2015038939A2 (fr) 2013-09-13 2014-09-12 Modulateurs du facteur b du complément

Publications (1)

Publication Number Publication Date
MA38959A1 true MA38959A1 (fr) 2017-08-31

Family

ID=52666522

Family Applications (1)

Application Number Title Priority Date Filing Date
MA38959A MA38959A1 (fr) 2013-09-13 2014-09-12 Modulateurs du facteur b du complément

Country Status (33)

Country Link
US (5) US20160222389A1 (fr)
EP (2) EP3603677A1 (fr)
JP (3) JP6666250B2 (fr)
KR (1) KR102381790B1 (fr)
CN (2) CN105744959B (fr)
AU (2) AU2014318580B2 (fr)
BR (1) BR112016004671B1 (fr)
CA (1) CA2921842A1 (fr)
CL (2) CL2016000606A1 (fr)
CR (1) CR20160170A (fr)
DK (1) DK3043827T3 (fr)
DO (1) DOP2016000063A (fr)
EA (1) EA035433B1 (fr)
ES (1) ES2745758T3 (fr)
HK (1) HK1225638A1 (fr)
HR (1) HRP20191600T1 (fr)
HU (1) HUE045109T2 (fr)
IL (1) IL244095A0 (fr)
LT (1) LT3043827T (fr)
MA (1) MA38959A1 (fr)
MX (2) MX371518B (fr)
MY (1) MY181251A (fr)
PE (2) PE20160500A1 (fr)
PH (1) PH12016500443A1 (fr)
PL (1) PL3043827T3 (fr)
PT (1) PT3043827T (fr)
RS (1) RS59252B1 (fr)
RU (1) RU2662967C2 (fr)
SG (2) SG10201806787VA (fr)
SI (1) SI3043827T1 (fr)
UA (1) UA119046C2 (fr)
WO (1) WO2015038939A2 (fr)
ZA (1) ZA201601236B (fr)

Families Citing this family (22)

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DK2092065T4 (da) 2006-10-18 2019-10-21 Ionis Pharmaceuticals Inc Antisense-forbindelser
EP3067421B1 (fr) 2011-02-08 2018-10-10 Ionis Pharmaceuticals, Inc. Composés oligomères comprenant des nucléotides bicycliques et leurs utilisations
RU2686080C2 (ru) 2013-05-01 2019-04-24 Ионис Фармасьютикалз, Инк. Композиции и способы
PL3043827T3 (pl) * 2013-09-13 2020-03-31 Ionis Pharmaceuticals, Inc. Modulatory czynnika b dopełniacza
SG11201604692UA (en) * 2013-12-12 2016-07-28 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
RS59182B1 (sr) * 2014-05-01 2019-10-31 Ionis Pharmaceuticals Inc Kompozicije i postupci za modulaciju ekspresije faktora b komplementa
WO2017135397A1 (fr) * 2016-02-05 2017-08-10 協和発酵キリン株式会社 Oligonucléotide antisens destiné à supprimer l'expression du facteur du complément b
AU2017290593A1 (en) 2016-06-27 2019-01-03 Achillion Pharmaceuticals, Inc. Quinazoline and indole compounds to treat medical disorders
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WO2019027015A1 (fr) * 2017-08-02 2019-02-07 協和発酵キリン株式会社 Complexe d'acide nucléique
EP3704252A1 (fr) 2017-11-01 2020-09-09 Alnylam Pharmaceuticals, Inc. Compositions d'arni de composant du complément c3 et leurs procédés d'utilisation
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EP4142738A1 (fr) * 2020-04-30 2023-03-08 Alnylam Pharmaceuticals, Inc. Compositions d'arni du facteur b du complément (cfb) et leurs procédés d'utilisation
WO2022028507A1 (fr) 2020-08-07 2022-02-10 上海美悦生物科技发展有限公司 Composé hétérocyclique, son procédé de préparation et son utilisation
KR20230049115A (ko) 2020-08-07 2023-04-12 상하이 메이유에 바이오테크 디벨롭먼트 컴퍼니 리미티드 보체 인자 b 억제제 및 이의 약학적 조성물, 제조 방법 및 용도
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EP4396346A1 (fr) 2021-09-02 2024-07-10 Silence Therapeutics GmbH Acides nucléiques pour inhiber l'expression du facteur b du complément (cfb) dans une cellule
IL312399A (en) 2021-10-29 2024-06-01 Alnylam Pharmaceuticals Inc Complement factor B (CFB) iRNA compositions and methods of using them
WO2023129496A2 (fr) * 2021-12-27 2023-07-06 Apellis Pharmaceuticals, Inc. Arn pour l'inhibition du complément
KR20240108505A (ko) 2022-01-26 2024-07-09 상하이 메이유에 바이오테크 디벨롭먼트 컴퍼니 리미티드 보체 인자 b 억제제의 염 형태, 결정형, 이의 제조 방법 및 응용
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Also Published As

Publication number Publication date
SG11201601445XA (en) 2016-03-30
WO2015038939A3 (fr) 2015-07-02
ES2745758T3 (es) 2020-03-03
JP7158363B2 (ja) 2022-10-21
US20160222389A1 (en) 2016-08-04
CA2921842A1 (fr) 2015-03-19
CN105744959B (zh) 2020-12-01
US20190048351A1 (en) 2019-02-14
CL2016000606A1 (es) 2017-02-17
SG10201806787VA (en) 2018-09-27
PE20160500A1 (es) 2016-05-21
HUE045109T2 (hu) 2019-12-30
CR20160170A (es) 2016-07-15
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