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PE20160158A1 - Oligomeros antisentido y conjugados con diana en pcsk9 - Google Patents

Oligomeros antisentido y conjugados con diana en pcsk9

Info

Publication number
PE20160158A1
PE20160158A1 PE2015002667A PE2015002667A PE20160158A1 PE 20160158 A1 PE20160158 A1 PE 20160158A1 PE 2015002667 A PE2015002667 A PE 2015002667A PE 2015002667 A PE2015002667 A PE 2015002667A PE 20160158 A1 PE20160158 A1 PE 20160158A1
Authority
PE
Peru
Prior art keywords
nucleotides
pcsk9
seq
contiguous sequence
complementary
Prior art date
Application number
PE2015002667A
Other languages
English (en)
Inventor
Marie Lindholm
Nanna Albæk
Jacob Ravn
Andreas Petri
Niels Fisker Nielsen
Maj Hedtjarn
Original Assignee
Roche Innovation Ct Copenhagen As
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
Priority claimed from PCT/EP2013/073858 external-priority patent/WO2014076195A1/en
Application filed by Roche Innovation Ct Copenhagen As filed Critical Roche Innovation Ct Copenhagen As
Publication of PE20160158A1 publication Critical patent/PE20160158A1/es

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    • 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
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    • 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
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    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/554—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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Abstract

SE REFIERE A COMPUESTOS OLIGOMERICOS Y CONJUGADOS DE LOS MISMOS CON DIANA EN EL ARNm DE LA PROPROTEINA–CONVERTASA SUBTILISINA/KEXINA TIPO 9 (PCSK9) EN UNA CELULA, QUE CONDUCEN A UNA EXPRESION REDUCIDA DE PCSK9. LOS OLIGOMEROS SON OLIGONUCLEOTIDOS ANTISENTIDO CON UNA LONGITUD DE ENTRE 12 Y 22 NUCLEOTIDOS, QUE COMPRENDEN: (A) UNA SECUENCIA CONTIGUA DE ENTRE 10 Y 16 NUCLEOTIDOS COMPLEMENTARIOS A UN TRAMO CORRESPONDIENTE DE SEC ID NO 33, 34 o 45; O (B) UNA SECUENCIA CONTIGUA DE 16 NUCLEOTIDOS COMPLEMENTARIOS A UN TRAMO CORRESPONDIENTE DE SEC ID NO 31. LOS CONJUGADOS DE OLIGOMEROS COMPRENDEN: (A) UN OLIGONUCLEOTIDO ANTISENTIDO DE ENTRE 12 Y 22 NUCLEOTIDOS DE LONGITUD, QUE COMPRENDE UNA SECUENCIA CONTIGUA DE ENTRE 10 Y 16 NUCLEOTIDOS COMPLEMENTARIOS A UN TRAMO CORRESPONDIENTE DE SEC ID NO 30, 31, 32, 33, 34 o 45; Y (B) POR LO MENOS UNA FRACCION CONJUGADA NO (POLI)NUCLEOTIDICA UNIDA COVALENTEMENTE AL OLIGONUCLEOTIDO, OPCIONALMENTE MEDIANTE UNA REGION CONECTORA. EN UN ASPECTO PREFERIDO, LA FRACCION CONJUGADA COMPRENDE UN CARBOHIDRATO, Y MAS PREFERENTEMENTE UNA N–ACETILGALACTOSAMINA (GalNAc) MONOVALENTE O MULTIVALENTE. DICHOS COMPUESTOS SON UTILES PARA EL TRATAMIENTO DE HIPERCOLESTEROLEMIA Y TRASTORNOS RELACIONADOS, TALES COMO ATEROSCLEROSIS, HIPERLIPIDEMIA, DISLIPIDEMIAS, ENFERMEDAD ARTERIAL CORONARIA Y ENFERMEDAD CARDIACA CORONARIA, ENTRE OTRAS
PE2015002667A 2013-06-27 2014-06-27 Oligomeros antisentido y conjugados con diana en pcsk9 PE20160158A1 (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
EP13174092 2013-06-27
EP13192930 2013-11-14
EP13192938 2013-11-14
PCT/EP2013/073858 WO2014076195A1 (en) 2012-11-15 2013-11-14 Oligonucleotide conjugates
EP14153253 2014-01-30
EP14168331 2014-05-14

Publications (1)

Publication Number Publication Date
PE20160158A1 true PE20160158A1 (es) 2016-03-18

Family

ID=52141126

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PE2015002667A PE20160158A1 (es) 2013-06-27 2014-06-27 Oligomeros antisentido y conjugados con diana en pcsk9

Country Status (30)

Country Link
US (7) US9879265B2 (es)
EP (2) EP3013959B1 (es)
JP (2) JP6255092B2 (es)
KR (2) KR101931089B1 (es)
CN (2) CN112263682B (es)
AU (1) AU2014300981B2 (es)
BR (1) BR112015032432B1 (es)
CA (1) CA2915316C (es)
CL (1) CL2015003728A1 (es)
CY (1) CY1122738T1 (es)
DK (1) DK3013959T3 (es)
EA (1) EA201592215A1 (es)
ES (1) ES2770667T3 (es)
HR (1) HRP20200163T1 (es)
HU (1) HUE048738T2 (es)
IL (1) IL242875B (es)
LT (1) LT3013959T (es)
MX (2) MX367083B (es)
MY (1) MY183049A (es)
NZ (1) NZ715081A (es)
PE (1) PE20160158A1 (es)
PH (1) PH12015502761A1 (es)
PL (1) PL3013959T3 (es)
PT (1) PT3013959T (es)
RS (1) RS59986B1 (es)
SG (2) SG10201908122XA (es)
SI (1) SI3013959T1 (es)
SM (1) SMT202000104T1 (es)
UA (1) UA119643C2 (es)
WO (1) WO2014207232A1 (es)

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