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CL2018001996A1 - Composiciones y métodos para modular la expresión del factor b del complemento (divisional de solicitud 2764-16) - Google Patents

Composiciones y métodos para modular la expresión del factor b del complemento (divisional de solicitud 2764-16)

Info

Publication number
CL2018001996A1
CL2018001996A1 CL2018001996A CL2018001996A CL2018001996A1 CL 2018001996 A1 CL2018001996 A1 CL 2018001996A1 CL 2018001996 A CL2018001996 A CL 2018001996A CL 2018001996 A CL2018001996 A CL 2018001996A CL 2018001996 A1 CL2018001996 A1 CL 2018001996A1
Authority
CL
Chile
Prior art keywords
compositions
modulate
expression
methods
complement factor
Prior art date
Application number
CL2018001996A
Other languages
English (en)
Inventor
Eric E Swayze
Susan M Freier
Thazha P Prakash
Punit P Seth
Tamar R Grossman
Michael L Mccaleb
Andrew T Watt
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 CL2018001996A1 publication Critical patent/CL2018001996A1/es

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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
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    • A—HUMAN NECESSITIES
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    • 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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    • 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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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy

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

Publication Number Publication Date
CL2018001996A1 true CL2018001996A1 (es) 2019-03-08

Family

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Family Applications (2)

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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)

Family Applications Before (1)

Application Number Title Priority Date Filing Date
CL2016002764A CL2016002764A1 (es) 2014-05-01 2016-11-02 Composiciones y métodos para modular la expresión del factor b del complemento

Country Status (29)

Country Link
US (4) US10280423B2 (es)
EP (3) EP3608406B1 (es)
JP (2) JP6637442B2 (es)
KR (1) KR102369736B1 (es)
CN (2) CN106232804B (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) ES2745825T3 (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) PL3137596T3 (es)
PT (1) PT3137596T (es)
RS (1) RS59182B1 (es)
RU (1) RU2701645C2 (es)
SG (2) SG11201608502TA (es)
SI (1) SI3137596T1 (es)
UA (1) UA121656C2 (es)
WO (1) WO2015168635A2 (es)

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CN105378085B (zh) 2013-05-01 2019-02-15 Ionis制药公司 用于调节hbv和ttr表达的组合物和方法
SI3043827T1 (sl) * 2013-09-13 2019-08-30 Ionis Pharmaceuticals, Inc. Modulatorji komplementnega faktorja B
PE20190626A1 (es) 2014-05-01 2019-04-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del factor b del complemento
CA2942570A1 (en) 2014-05-01 2015-11-05 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
WO2016055601A1 (en) 2014-10-10 2016-04-14 F. Hoffmann-La Roche Ag Galnac phosphoramidites, nucleic acid conjugates thereof and their use
EP3370708A4 (en) * 2015-11-06 2019-06-26 Ionis Pharmaceuticals, Inc. MODULATION OF APOLIPOPROTEIN EXPRESSION (A)
US10781175B2 (en) 2016-07-15 2020-09-22 Am Chemicals Llc Solid supports and phosphoramidite building blocks for oligonucleotide conjugates
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
WO2021046260A1 (en) 2019-09-03 2021-03-11 Arcturus Therapeutics, Inc. Asialoglycoprotein receptor mediated delivery of therapeutically active conjugates
BR112022021136A2 (pt) * 2020-04-30 2022-11-29 Alnylam Pharmaceuticals Inc Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas
WO2023031359A1 (en) 2021-09-02 2023-03-09 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
WO2023076451A1 (en) * 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2024097861A1 (en) * 2022-11-02 2024-05-10 Ionis Pharmaceuticals, Inc. Methods for modulating complement factor b expression
WO2024137590A2 (en) * 2022-12-19 2024-06-27 Sanegene Bio Usa Inc. Small interfering rna targeting cfb and uses thereof

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