ES2941742T3 - Composiciones y procedimientos para modular la expresión del factor B del complemento - Google Patents

Composiciones y procedimientos para modular la expresión del factor B del complemento Download PDF

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ES2941742T3
ES2941742T3 ES19184459T ES19184459T ES2941742T3 ES 2941742 T3 ES2941742 T3 ES 2941742T3 ES 19184459 T ES19184459 T ES 19184459T ES 19184459 T ES19184459 T ES 19184459T ES 2941742 T3 ES2941742 T3 ES 2941742T3
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compound
modified
certain embodiments
carbohydrate
antisense
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Thazha Prakash
Punit Seth
Eric Swayze
Tamar Grossman
Michael Mccaleb
Andrew Watt
Susan Freier
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • 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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    • 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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    • 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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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • 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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ES19184459T 2014-05-01 2015-05-01 Composiciones y procedimientos para modular la expresión del factor B del complemento Active ES2941742T3 (es)

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US201461987471P 2014-05-01 2014-05-01
US201462076273P 2014-11-06 2014-11-06

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ES2941742T3 true ES2941742T3 (es) 2023-05-25

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ES15786214T Active ES2745825T3 (es) 2014-05-01 2015-05-01 Composiciones y métodos para modular la expresión del factor B del complemento

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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) CR20160554A (enExample)
DK (1) DK3137596T3 (enExample)
DO (1) DOP2016000291A (enExample)
EA (1) EA036496B1 (enExample)
ES (2) ES2941742T3 (enExample)
HR (1) HRP20191664T1 (enExample)
HU (1) HUE046272T2 (enExample)
IL (2) IL247883B (enExample)
LT (1) LT3137596T (enExample)
MX (1) MX380866B (enExample)
PE (2) PE20170010A1 (enExample)
PH (1) PH12016502062B1 (enExample)
PL (2) PL3137596T3 (enExample)
PT (1) PT3137596T (enExample)
RS (1) RS59182B1 (enExample)
RU (1) RU2701645C2 (enExample)
SG (2) SG11201608502TA (enExample)
SI (1) SI3137596T1 (enExample)
UA (1) UA121656C2 (enExample)
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PT3137596T (pt) 2014-05-01 2019-09-27 Ionis Pharmaceuticals Inc Composições e métodos para modulação da expressão do factor b do complemento
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US12447173B2 (en) 2019-12-26 2025-10-21 Eisai R&D Management Co., Ltd. Pharmaceutical composition containing double-stranded ribonucleic acid inhibiting expression of complement C5
KR20230017789A (ko) * 2020-04-30 2023-02-06 알닐람 파마슈티칼스 인코포레이티드 보체 인자 B (CFB) iRNA 조성물 및 이의 사용 방법
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CA3234636A1 (en) * 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
EP4611768A1 (en) * 2022-11-02 2025-09-10 Ionis Pharmaceuticals, Inc. Methods for modulating complement factor b expression
EP4638743A2 (en) * 2022-12-19 2025-10-29 Sanegene Bio USA Inc. Small interfering rna targeting cfb and uses thereof
AU2024240837A1 (en) * 2023-03-21 2025-09-11 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement factor b (cfb) pharmaceutical compositions thereof, and methods of use
WO2024245424A1 (zh) * 2023-06-02 2024-12-05 维亚臻生物技术(苏州)有限公司 一种用于免疫类疾病的双链核苷酸化合物及其应用
CN118620964A (zh) * 2023-06-28 2024-09-10 百奥赛图(北京)医药科技股份有限公司 一种cfb基因修饰的非人动物
TW202519657A (zh) * 2023-07-28 2025-05-16 大陸商蘇州炫景生物科技有限公司 Cfb抑制劑組合物及其應用
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