CR20160554A - 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
CR20160554A
CR20160554A CR20160554A CR20160554A CR20160554A CR 20160554 A CR20160554 A CR 20160554A CR 20160554 A CR20160554 A CR 20160554A CR 20160554 A CR20160554 A CR 20160554A CR 20160554 A CR20160554 A CR 20160554A
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CR
Costa Rica
Prior art keywords
compositions
methods
modulate
expression
complement factor
Prior art date
Application number
CR20160554A
Other languages
English (en)
Inventor
Andrew T Watt
Punit P Seth
Susan M Freier
Eric E Swayze
Tamar R Grossman
Michael L Mccaleb
Thazha P Prakash
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 CR20160554A publication Critical patent/CR20160554A/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
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • 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
    • A61K47/555—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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
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    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
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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
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21047—Alternative-complement-pathway C3/C5 convertase (3.4.21.47), i.e. properdin factor B
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

Abstract

Las modalidades de la presente proporcionan métodos, compuestos y composiciones para tratar, prevenir o mejorar una enfermedad asociada a una desregulación de la vía alternativa del complemento al administrarle un inhibidor específico del Factor B del Complemento (CFB) a un sujeto.
CR20160554A 2014-05-01 2015-05-01 Composiciones y métodos para modular la expresión del factor b del complemento CR20160554A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461987471P 2014-05-01 2014-05-01
US201462076273P 2014-11-06 2014-11-06
PCT/US2015/028916 WO2015168635A2 (en) 2014-05-01 2015-05-01 Compositions and methods for modulating complement factor b expression

Publications (1)

Publication Number Publication Date
CR20160554A true CR20160554A (es) 2017-03-07

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Family Applications (2)

Application Number Title Priority Date Filing Date
CR20200228A CR20200228A (es) 2014-05-01 2015-05-01 COMPOSICIONES Y MÈTODOS PARA MODULAR LA EXPRESIÒN DEL FACTOR B DEL COMPLEMENTO (Divisional 2016-0554)
CR20160554A CR20160554A (es) 2014-05-01 2015-05-01 Composiciones y métodos para modular la expresión del factor b del complemento

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CR20200228A CR20200228A (es) 2014-05-01 2015-05-01 COMPOSICIONES Y MÈTODOS PARA MODULAR LA EXPRESIÒN DEL FACTOR B DEL COMPLEMENTO (Divisional 2016-0554)

Country Status (28)

Country Link
US (4) US10280423B2 (es)
EP (3) EP3608406B1 (es)
JP (2) JP6637442B2 (es)
KR (1) KR102369736B1 (es)
CN (2) CN110724687B (es)
AU (1) AU2015252858C1 (es)
BR (1) BR112016022593B1 (es)
CA (1) CA2943894A1 (es)
CL (2) CL2016002764A1 (es)
CR (2) CR20200228A (es)
DK (1) DK3137596T3 (es)
DO (1) DOP2016000291A (es)
EA (1) EA036496B1 (es)
ES (2) ES2941742T3 (es)
HR (1) HRP20191664T1 (es)
HU (1) HUE046272T2 (es)
IL (2) IL247883B (es)
LT (1) LT3137596T (es)
MX (1) MX2016014294A (es)
PE (2) PE20190626A1 (es)
PH (1) PH12016502062A1 (es)
PL (2) PL3608406T3 (es)
PT (1) PT3137596T (es)
RS (1) RS59182B1 (es)
RU (1) RU2701645C2 (es)
SG (2) SG10201809953QA (es)
SI (1) SI3137596T1 (es)
WO (1) WO2015168635A2 (es)

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JP6667453B2 (ja) 2014-05-01 2020-03-18 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 成長ホルモン受容体発現を調節するための組成物及び方法
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KR20170068469A (ko) 2014-10-10 2017-06-19 에프. 호프만-라 로슈 아게 ＧａｌＮＡｃ 포스포라미다이트, 그의 핵산 컨쥬게이트 및 그의 용도
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JOP20190215A1 (ar) * 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
WO2020109343A1 (en) 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Combination therapy for treatment of macular degeneration
AU2020340987A1 (en) * 2019-09-03 2022-04-14 Arcturus Therapeutics, Inc. Asialoglycoprotein receptor mediated delivery of therapeutically active conjugates
CN116096381A (zh) * 2020-04-30 2023-05-09 阿尔尼拉姆医药品有限公司 补体因子B（CFB）iRNA组合物及其使用方法
AU2022336157A1 (en) 2021-09-02 2024-03-14 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
TW202333749A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 補體因子ｂ（ｃｆｂ）ｉｒｎａ組成物及其使用方法

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