DK3357497T3 - Modulation af apolipoprotein ciii (apociii) ekspression - Google Patents

Modulation af apolipoprotein ciii (apociii) ekspression Download PDF

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Publication number
DK3357497T3
DK3357497T3 DK18155337.1T DK18155337T DK3357497T3 DK 3357497 T3 DK3357497 T3 DK 3357497T3 DK 18155337 T DK18155337 T DK 18155337T DK 3357497 T3 DK3357497 T3 DK 3357497T3
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Prior art keywords
ekspression
apociii
modulation
apolipoprotein ciii
ciii
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DK18155337.1T
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English (en)
Inventor
Adam Mullick
Rosanne M Crooke
Mark J Graham
Kenneth W Dobie
Thomas A Bell
Richard Lee
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Ionis Pharmaceuticals Inc
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Publication of DK3357497T3 publication Critical patent/DK3357497T3/da

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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
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
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    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • AHUMAN NECESSITIES
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    • A61P3/06Antihyperlipidemics
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    • A61P9/06Antiarrhythmics
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

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DK18155337.1T 2011-04-27 2012-04-27 Modulation af apolipoprotein ciii (apociii) ekspression DK3357497T3 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161479817P 2011-04-27 2011-04-27
US201261595009P 2012-02-03 2012-02-03
EP12776385.2A EP2701713B1 (en) 2011-04-27 2012-04-27 Modulation of apolipoprotein ciii (apociii) expression

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DK3357497T3 true DK3357497T3 (en) 2020-11-23

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DK12776385.2T DK2701713T3 (da) 2011-04-27 2012-04-27 Modulation af apolipoprotein ciii (apociii) ekspression
DK18155337.1T DK3357497T3 (en) 2011-04-27 2012-04-27 Modulation af apolipoprotein ciii (apociii) ekspression

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US (4) US9157082B2 (da)
EP (2) EP2701713B1 (da)
JP (3) JP6203707B2 (da)
KR (3) KR102021688B1 (da)
CN (3) CN103547271A (da)
AU (3) AU2012249324B2 (da)
BR (1) BR112013027479A8 (da)
CA (1) CA2834232A1 (da)
DK (2) DK2701713T3 (da)
ES (2) ES2833468T3 (da)
IL (1) IL229010A0 (da)
MY (1) MY162715A (da)
NZ (1) NZ710192A (da)
RU (2) RU2603076C9 (da)
SG (2) SG194671A1 (da)
UA (1) UA115309C2 (da)
WO (1) WO2012149495A1 (da)

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