CR20150236A - Métodos para el tratamiento del síndrome de alport - Google Patents

Métodos para el tratamiento del síndrome de alport

Info

Publication number
CR20150236A
CR20150236A CR20150236A CR20150236A CR20150236A CR 20150236 A CR20150236 A CR 20150236A CR 20150236 A CR20150236 A CR 20150236A CR 20150236 A CR20150236 A CR 20150236A CR 20150236 A CR20150236 A CR 20150236A
Authority
CR
Costa Rica
Prior art keywords
alport syndrome
treatment
methods
mir
target modified
Prior art date
Application number
CR20150236A
Other languages
English (en)
Inventor
Jeremy Duffield
Balkrishen Bhat
Deidre Mackenna
Original Assignee
Regulus Therapeutics 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 Regulus Therapeutics Inc filed Critical Regulus Therapeutics Inc
Publication of CR20150236A publication Critical patent/CR20150236A/es

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/4015—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil having oxo groups directly attached to the heterocyclic ring, e.g. piracetam, ethosuximide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/59—Compounds containing 9, 10- seco- cyclopenta[a]hydrophenanthrene ring systems
    • A61K31/593—9,10-Secocholestane derivatives, e.g. cholecalciferol, i.e. vitamin D3
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/3233—Morpholino-type ring

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)

Abstract

Se proporcionan métodos para el tratamiento del síndrome de Alport, utilizando oligonucleótidos modificados a objetivo miR-21. En ciertas realizaciones, un oligonucleótido modificado a objetivo miR-21 mejoran la función renal y/o reducen la fibrosis en sujetos con síndrome de Alport.
CR20150236A 2012-10-09 2015-05-07 Métodos para el tratamiento del síndrome de alport CR20150236A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261711514P 2012-10-09 2012-10-09
US201361779137P 2013-03-13 2013-03-13
PCT/US2013/063884 WO2014058881A1 (en) 2012-10-09 2013-10-08 Methods for treatment of alport syndrome

Publications (1)

Publication Number Publication Date
CR20150236A true CR20150236A (es) 2015-06-30

Family

ID=49382663

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20150236A CR20150236A (es) 2012-10-09 2015-05-07 Métodos para el tratamiento del síndrome de alport

Country Status (34)

Country Link
US (8) US9012423B2 (es)
EP (2) EP3620522B1 (es)
JP (4) JP6272880B2 (es)
KR (4) KR20220005634A (es)
CN (1) CN104718295B (es)
AR (1) AR092940A1 (es)
AU (3) AU2013329427B2 (es)
BR (1) BR112015007709B1 (es)
CA (1) CA2885605A1 (es)
CL (1) CL2015000873A1 (es)
CR (1) CR20150236A (es)
CY (1) CY1122479T1 (es)
DK (1) DK2906698T3 (es)
EA (1) EA201590711A1 (es)
ES (2) ES2960488T3 (es)
HR (1) HRP20191948T1 (es)
HU (1) HUE045858T2 (es)
IL (2) IL237768B (es)
LT (1) LT2906698T (es)
MX (1) MX356340B (es)
MY (1) MY183921A (es)
NZ (1) NZ630596A (es)
PH (1) PH12015500781B1 (es)
PL (1) PL2906698T3 (es)
PT (1) PT2906698T (es)
RS (1) RS60014B1 (es)
SG (1) SG11201502748RA (es)
SI (1) SI2906698T1 (es)
TN (1) TN2015000099A1 (es)
TW (1) TWI641388B (es)
UA (1) UA116639C2 (es)
UY (1) UY35069A (es)
WO (1) WO2014058881A1 (es)
ZA (1) ZA201502087B (es)

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CN104718295A (zh) 2015-06-17
BR112015007709B1 (pt) 2022-05-17
RS60014B1 (sr) 2020-04-30
ZA201502087B (en) 2016-06-29
TN2015000099A1 (en) 2016-06-29
MX356340B (es) 2018-05-23
KR102349184B1 (ko) 2022-01-07
AU2021269404A1 (en) 2021-12-16
AU2013329427B2 (en) 2019-05-02
EP3620522A1 (en) 2020-03-11
EP2906698B1 (en) 2019-07-31
TW201420120A (zh) 2014-06-01
IL237768B (en) 2018-10-31
HRP20191948T1 (hr) 2020-04-03
CY1122479T1 (el) 2021-01-27
EP2906698A1 (en) 2015-08-19
AU2019210494A1 (en) 2019-08-15
KR20200118245A (ko) 2020-10-14
ES2753174T3 (es) 2020-04-07
UY35069A (es) 2014-04-30
JP2015536301A (ja) 2015-12-21
EP3620522B1 (en) 2023-07-26
JP2018080190A (ja) 2018-05-24
ES2960488T3 (es) 2024-03-05
US20220098582A1 (en) 2022-03-31
PT2906698T (pt) 2019-11-05
IL262200A (en) 2018-11-29
KR102165189B1 (ko) 2020-10-14
DK2906698T3 (da) 2019-11-04
MY183921A (en) 2021-03-17
HK1212378A1 (en) 2016-06-10
PH12015500781A1 (en) 2015-06-15
SI2906698T1 (sl) 2019-11-29
KR20150064072A (ko) 2015-06-10
BR112015007709A2 (pt) 2017-08-08
CN104718295B (zh) 2020-03-13
CL2015000873A1 (es) 2015-08-14
HUE045858T2 (hu) 2020-01-28
WO2014058881A1 (en) 2014-04-17
US20160319283A1 (en) 2016-11-03
CA2885605A1 (en) 2014-04-17
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US20200283765A1 (en) 2020-09-10
US9688986B2 (en) 2017-06-27
US20240167032A1 (en) 2024-05-23
JP6272880B2 (ja) 2018-01-31
US20140100263A1 (en) 2014-04-10
US20170369879A1 (en) 2017-12-28
JP2020073479A (ja) 2020-05-14
MX2015004460A (es) 2015-11-23
JP2022110140A (ja) 2022-07-28
PH12015500781B1 (en) 2015-06-15
AU2013329427A1 (en) 2015-04-02
AR092940A1 (es) 2015-05-06
EA201590711A1 (ru) 2015-07-30
UA116639C2 (uk) 2018-04-25
US9359609B2 (en) 2016-06-07
US9970011B2 (en) 2018-05-15
PL2906698T3 (pl) 2020-05-18
KR20220005634A (ko) 2022-01-13
SG11201502748RA (en) 2015-05-28
NZ630596A (en) 2017-05-26
LT2906698T (lt) 2019-11-11
KR20230136686A (ko) 2023-09-26
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US20150299704A1 (en) 2015-10-22
US20180298385A1 (en) 2018-10-18

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