SG10201809953QA - Compositions and methods for modulating complement factor b expression - Google Patents

Compositions and methods for modulating complement factor b expression

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Publication number
SG10201809953QA
SG10201809953QA SG10201809953QA SG10201809953QA SG10201809953QA SG 10201809953Q A SG10201809953Q A SG 10201809953QA SG 10201809953Q A SG10201809953Q A SG 10201809953QA SG 10201809953Q A SG10201809953Q A SG 10201809953QA SG 10201809953Q A SG10201809953Q A SG 10201809953QA
Authority
SG
Singapore
Prior art keywords
compositions
methods
expression
complement factor
modulating complement
Prior art date
Application number
SG10201809953QA
Other languages
English (en)
Inventor
Thazha Prakash
Punit Seth
Eric Swayze
Tamar Grossman
Michael Mccaleb
Andrew Watt
Susan Freier
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 SG10201809953QA publication Critical patent/SG10201809953QA/en

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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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
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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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    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • 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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    • C12N2310/30—Chemical structure
    • 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

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SG10201809953QA 2014-05-01 2015-05-01 Compositions and methods for modulating complement factor b expression SG10201809953QA (en)

Applications Claiming Priority (2)

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

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SG10201809953QA true SG10201809953QA (en) 2018-12-28

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US (4) US10280423B2 (ru)
EP (3) EP3608406B1 (ru)
JP (2) JP6637442B2 (ru)
KR (1) KR102369736B1 (ru)
CN (2) CN106232804B (ru)
AU (1) AU2015252858C1 (ru)
BR (1) BR112016022593B1 (ru)
CA (1) CA2943894A1 (ru)
CL (2) CL2016002764A1 (ru)
CR (2) CR20160554A (ru)
DK (1) DK3137596T3 (ru)
DO (1) DOP2016000291A (ru)
EA (1) EA036496B1 (ru)
ES (2) ES2745825T3 (ru)
HR (1) HRP20191664T1 (ru)
HU (1) HUE046272T2 (ru)
IL (2) IL247883B (ru)
LT (1) LT3137596T (ru)
MX (1) MX2016014294A (ru)
PE (2) PE20170010A1 (ru)
PH (1) PH12016502062A1 (ru)
PL (2) PL3137596T3 (ru)
PT (1) PT3137596T (ru)
RS (1) RS59182B1 (ru)
RU (1) RU2701645C2 (ru)
SG (2) SG10201809953QA (ru)
SI (1) SI3137596T1 (ru)
UA (1) UA121656C2 (ru)
WO (1) WO2015168635A2 (ru)

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RU2686080C2 (ru) 2013-05-01 2019-04-24 Ионис Фармасьютикалз, Инк. Композиции и способы
PL3043827T3 (pl) * 2013-09-13 2020-03-31 Ionis Pharmaceuticals, Inc. Modulatory czynnika b dopełniacza
RS59182B1 (sr) 2014-05-01 2019-10-31 Ionis Pharmaceuticals Inc Kompozicije i postupci za modulaciju ekspresije faktora b komplementa
KR102149571B1 (ko) 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
SG11201702877TA (en) 2014-10-10 2017-05-30 Hoffmann La Roche Galnac phosphoramidites, nucleic acid conjugates thereof and their use
CA2999341A1 (en) 2015-11-06 2017-05-11 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
EP3484908A4 (en) 2016-07-15 2020-04-08 AM Chemicals Llc NON-NUCLEOSIDIC SOLID CARRIERS AND PHOSPHORAMIDITE BUILDING BLOCKS FOR OLIGONUCLEOTID 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
KR20220058578A (ko) * 2019-09-03 2022-05-09 아크투루스 쎄라퓨틱스, 인크. 치료 활성 접합체의 아시알로당단백질 수용체 매개 전달
EP4142738A1 (en) * 2020-04-30 2023-03-08 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
EP4396346A1 (en) 2021-09-02 2024-07-10 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
IL312399A (en) 2021-10-29 2024-06-01 Alnylam Pharmaceuticals Inc Complement factor B (CFB) iRNA compositions and methods of using them
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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