CL2016002764A1 - Composiciones y métodos para modular la expresión del factor b del complemento - Google Patents

Composiciones y métodos para modular la expresión del factor b del complemento

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Publication number
CL2016002764A1
CL2016002764A1 CL2016002764A CL2016002764A CL2016002764A1 CL 2016002764 A1 CL2016002764 A1 CL 2016002764A1 CL 2016002764 A CL2016002764 A CL 2016002764A CL 2016002764 A CL2016002764 A CL 2016002764A CL 2016002764 A1 CL2016002764 A1 CL 2016002764A1
Authority
CL
Chile
Prior art keywords
modulate
expression
compositions
methods
complement factor
Prior art date
Application number
CL2016002764A
Other languages
English (en)
Spanish (es)
Inventor
Susan M Freier
Punit P Seth
Thazha P Prakash
Tamar R Grossman
Michael L Mccaleb
Andrew T Watt
Eric E Swayze
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 CL2016002764A1 publication Critical patent/CL2016002764A1/es

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    • 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
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/555Medicinal 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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
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    • C12N15/1137Non-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
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    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21047Alternative-complement-pathway C3/C5 convertase (3.4.21.47), i.e. properdin factor B
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    • 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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    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy

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CL2016002764A 2014-05-01 2016-11-02 Composiciones y métodos para modular la expresión del factor b del complemento CL2016002764A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461987471P 2014-05-01 2014-05-01
US201462076273P 2014-11-06 2014-11-06

Publications (1)

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CL2016002764A1 true CL2016002764A1 (es) 2017-12-29

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CL2016002764A CL2016002764A1 (es) 2014-05-01 2016-11-02 Composiciones y métodos para modular la expresión del factor b del complemento
CL2018001996A CL2018001996A1 (es) 2014-05-01 2018-07-23 Composiciones y métodos para modular la expresión del factor b del complemento (divisional de solicitud 2764-16)

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CL2018001996A CL2018001996A1 (es) 2014-05-01 2018-07-23 Composiciones y métodos para modular la expresión del factor b del complemento (divisional de solicitud 2764-16)

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US (4) US10280423B2 (enExample)
EP (3) EP3608406B1 (enExample)
JP (2) JP6637442B2 (enExample)
KR (1) KR102369736B1 (enExample)
CN (2) CN106232804B (enExample)
AU (1) AU2015252858C1 (enExample)
BR (1) BR112016022593B1 (enExample)
CA (1) CA2943894A1 (enExample)
CL (2) CL2016002764A1 (enExample)
CR (2) CR20200228A (enExample)
DK (1) DK3137596T3 (enExample)
DO (1) DOP2016000291A (enExample)
EA (1) EA036496B1 (enExample)
ES (2) ES2745825T3 (enExample)
HR (1) HRP20191664T1 (enExample)
HU (1) HUE046272T2 (enExample)
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LT (1) LT3137596T (enExample)
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JP2023523790A (ja) * 2020-04-30 2023-06-07 アルナイラム ファーマシューティカルズ, インコーポレイテッド 補体因子B(CFB)iRNA組成物およびその使用方法
JP2024525868A (ja) * 2021-07-17 2024-07-12 サーナオミクス インコーポレイテッド 産物及び組成物
EP4396346A1 (en) 2021-09-02 2024-07-10 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
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