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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
    • 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
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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)
  • 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 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) 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)

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