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DK3137596T3 - Sammensætninger og fremgangsmåder til modulation af komplement faktor b-ekspression - Google Patents

Sammensætninger og fremgangsmåder til modulation af komplement faktor b-ekspression Download PDF

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Publication number
DK3137596T3
DK3137596T3 DK15786214.5T DK15786214T DK3137596T3 DK 3137596 T3 DK3137596 T3 DK 3137596T3 DK 15786214 T DK15786214 T DK 15786214T DK 3137596 T3 DK3137596 T3 DK 3137596T3
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Prior art keywords
modulation
procedures
expression
compositions
complement factor
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DK15786214.5T
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English (en)
Inventor
Thazha P Prakash
Punit P Seth
Eric E Swayze
Tamar R Grossman
Michael L Mccaleb
Andrew T Watt
Susan M Freier
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Ionis Pharmaceuticals Inc
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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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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    • C12N15/1137—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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DK15786214.5T 2014-05-01 2015-05-01 Sammensætninger og fremgangsmåder til modulation af komplement faktor b-ekspression DK3137596T3 (da)

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
DK3137596T3 true DK3137596T3 (da) 2019-09-02

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US (4) US10280423B2 (da)
EP (3) EP3608406B1 (da)
JP (2) JP6637442B2 (da)
KR (1) KR102369736B1 (da)
CN (2) CN110724687B (da)
AU (1) AU2015252858C1 (da)
BR (1) BR112016022593B1 (da)
CA (1) CA2943894A1 (da)
CL (2) CL2016002764A1 (da)
CR (2) CR20160554A (da)
DK (1) DK3137596T3 (da)
DO (1) DOP2016000291A (da)
EA (1) EA036496B1 (da)
ES (2) ES2745825T3 (da)
HR (1) HRP20191664T1 (da)
HU (1) HUE046272T2 (da)
IL (2) IL247883B (da)
LT (1) LT3137596T (da)
MX (1) MX2016014294A (da)
PE (2) PE20170010A1 (da)
PH (1) PH12016502062A1 (da)
PL (2) PL3137596T3 (da)
PT (1) PT3137596T (da)
RS (1) RS59182B1 (da)
RU (1) RU2701645C2 (da)
SG (2) SG10201809953QA (da)
SI (1) SI3137596T1 (da)
UA (1) UA121656C2 (da)
WO (1) WO2015168635A2 (da)

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KR20240147701A (ko) 2013-05-01 2024-10-08 아이오니스 파마수티컬즈, 인코포레이티드 Hbv 및 ttr 발현을 조절하는 조성물 및 방법
EA035433B1 (ru) * 2013-09-13 2020-06-15 Ионис Фармасьютикалз, Инк. Модуляторы фактора в комплемента
KR102149571B1 (ko) 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
PE20170010A1 (es) 2014-05-01 2017-03-04 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del factor b del complemento
JP6626101B2 (ja) 2014-10-10 2019-12-25 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ ＧａｌＮＡｃホスホラミダイト、その核酸結合体およびそれらの使用
BR112018003291A2 (pt) 2015-11-06 2018-09-25 Ionis Pharmaceuticals, Inc. modulando a expressão da apolipoproteina (a)
WO2018013999A1 (en) 2016-07-15 2018-01-18 Am Chemicals Llc Non-nucleosidic solid supports and phosphoramidite building blocks for oligonucleotide synthesis
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
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
MX2024002767A (es) 2021-09-02 2024-08-20 Silence Therapeutics Gmbh Ácidos nucleicos para inhibir la expresión del factor b del complemento (cfb) en una célula.
AU2022378567A1 (en) 2021-10-29 2024-04-11 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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