AR092940A1 - Metodos para el tratamiento del sindrome de alport - Google Patents

Metodos para el tratamiento del sindrome de alport

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Publication number
AR092940A1
AR092940A1 ARP130103643A ARP130103643A AR092940A1 AR 092940 A1 AR092940 A1 AR 092940A1 AR P130103643 A ARP130103643 A AR P130103643A AR P130103643 A ARP130103643 A AR P130103643A AR 092940 A1 AR092940 A1 AR 092940A1
Authority
AR
Argentina
Prior art keywords
alport syndrome
mir
modified oligonucleotide
treatment
certain embodiments
Prior art date
Application number
ARP130103643A
Other languages
English (en)
Inventor
Mkenna Deidre
duffield Jeremy
Bhat Balkrishen
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 AR092940A1 publication Critical patent/AR092940A1/es

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40Heterocyclic 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
    • A61K31/4015Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/59Compounds containing 9, 10- seco- cyclopenta[a]hydrophenanthrene ring systems
    • A61K31/5939,10-Secocholestane derivatives, e.g. cholecalciferol, i.e. vitamin D3
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/113Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/313Phosphorodithioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring

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  • 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)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (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

Métodos para el tratamiento del Síndrome de Alport, usando oligonucleótidos modificados dirigidos a miR-21. En ciertas realizaciones, un oligonucleótido modificado dirigido a miR-21 mejora la función renal y/o reduce la fibrosis en sujetos que tienen Síndrome de Alport. En ciertas realizaciones, la administración de un oligonucleótido modificado dirigido a miR-21 retrasa el inicio de la enfermedad renal de etapa terminal en un sujeto que tiene Síndrome de Alport. En ciertas realizaciones, un oligonucleótido modificado dirigido a miR-21 retrasa la necesidad de diálisis o trasplante de riñón en un sujeto que tiene Síndrome de Alport. Reivindicación 30: Uso de un oligonucleótido modificado que consiste en 12 a 25 nucleósidos ligados, en donde la secuencia de bases nitrogenadas del oligonucleótido modificado es complementaria a miR-21, para el tratamiento del Síndrome de Alport.
ARP130103643A 2012-10-09 2013-10-08 Metodos para el tratamiento del sindrome de alport AR092940A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261711514P 2012-10-09 2012-10-09
US201361779137P 2013-03-13 2013-03-13

Publications (1)

Publication Number Publication Date
AR092940A1 true AR092940A1 (es) 2015-05-06

Family

ID=49382663

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP130103643A AR092940A1 (es) 2012-10-09 2013-10-08 Metodos para el tratamiento del sindrome de alport

Country Status (35)

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US (8) US9012423B2 (es)
EP (2) EP2906698B1 (es)
JP (4) JP6272880B2 (es)
KR (4) KR102165189B1 (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)
HK (1) HK1212378A1 (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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LT2906698T (lt) 2019-11-11
US20200283765A1 (en) 2020-09-10
BR112015007709A2 (pt) 2017-08-08
SI2906698T1 (sl) 2019-11-29
TWI641388B (zh) 2018-11-21
AU2021269404A1 (en) 2021-12-16
PH12015500781A1 (en) 2015-06-15
MY183921A (en) 2021-03-17
EP3620522B1 (en) 2023-07-26
US9012423B2 (en) 2015-04-21
MX2015004460A (es) 2015-11-23
CY1122479T1 (el) 2021-01-27
JP2020073479A (ja) 2020-05-14
CA2885605A1 (en) 2014-04-17
PT2906698T (pt) 2019-11-05
TN2015000099A1 (en) 2016-06-29
NZ630596A (en) 2017-05-26
WO2014058881A1 (en) 2014-04-17
EP2906698A1 (en) 2015-08-19
PL2906698T3 (pl) 2020-05-18
HUE045858T2 (hu) 2020-01-28
JP2022110140A (ja) 2022-07-28
CN104718295A (zh) 2015-06-17
ES2753174T3 (es) 2020-04-07
SG11201502748RA (en) 2015-05-28
US20160319283A1 (en) 2016-11-03
AU2019210494A1 (en) 2019-08-15
CR20150236A (es) 2015-06-30
CN104718295B (zh) 2020-03-13
CL2015000873A1 (es) 2015-08-14
AU2013329427B2 (en) 2019-05-02
KR20220005634A (ko) 2022-01-13
JP2015536301A (ja) 2015-12-21
US20220098582A1 (en) 2022-03-31
EA201590711A1 (ru) 2015-07-30
TW201420120A (zh) 2014-06-01
KR102165189B1 (ko) 2020-10-14
EP2906698B1 (en) 2019-07-31
JP2018080190A (ja) 2018-05-24
ZA201502087B (en) 2016-06-29
KR102349184B1 (ko) 2022-01-07
DK2906698T3 (da) 2019-11-04
AU2013329427A1 (en) 2015-04-02
IL237768B (en) 2018-10-31
HK1212378A1 (en) 2016-06-10
US20140100263A1 (en) 2014-04-10
PH12015500781B1 (en) 2015-06-15
US20180298385A1 (en) 2018-10-18
HRP20191948T1 (hr) 2020-04-03
EP3620522A1 (en) 2020-03-11
JP6272880B2 (ja) 2018-01-31
US20150299704A1 (en) 2015-10-22
KR20150064072A (ko) 2015-06-10
US9970011B2 (en) 2018-05-15
UA116639C2 (uk) 2018-04-25
US9688986B2 (en) 2017-06-27
US9359609B2 (en) 2016-06-07
US20170369879A1 (en) 2017-12-28
RS60014B1 (sr) 2020-04-30
IL262200A (en) 2018-11-29
KR20200118245A (ko) 2020-10-14
US20240167032A1 (en) 2024-05-23
KR20230136686A (ko) 2023-09-26
BR112015007709B1 (pt) 2022-05-17
UY35069A (es) 2014-04-30
ES2960488T3 (es) 2024-03-05
MX356340B (es) 2018-05-23

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