PL3608406T3 - Kompozycje i sposoby modulowania ekspresji czynnika B dopełniacza - Google Patents

Kompozycje i sposoby modulowania ekspresji czynnika B dopełniacza

Info

Publication number
PL3608406T3
PL3608406T3 PL19184459.6T PL19184459T PL3608406T3 PL 3608406 T3 PL3608406 T3 PL 3608406T3 PL 19184459 T PL19184459 T PL 19184459T PL 3608406 T3 PL3608406 T3 PL 3608406T3
Authority
PL
Poland
Prior art keywords
expression
compositions
methods
complement factor
modulating complement
Prior art date
Application number
PL19184459.6T
Other languages
English (en)
Inventor
Thazha P Prakash
Punit P. Seth
Eric E. Swayze
Tamar R. GROSSMAN
Michael L. Mccaleb
Andrew T. Watt
Susan M. 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 PL3608406T3 publication Critical patent/PL3608406T3/pl

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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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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
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    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
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    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
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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
    • C12N15/1137Non-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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    • 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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    • C12N2310/31Chemical structure of the backbone
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
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    • C12N2310/00Structure or type of the nucleic acid
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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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
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PL19184459.6T 2014-05-01 2015-05-01 Kompozycje i sposoby modulowania ekspresji czynnika B dopełniacza PL3608406T3 (pl)

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
PL3608406T3 true PL3608406T3 (pl) 2023-05-22

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PL19184459.6T PL3608406T3 (pl) 2014-05-01 2015-05-01 Kompozycje i sposoby modulowania ekspresji czynnika B dopełniacza
PL15786214T PL3137596T3 (pl) 2014-05-01 2015-05-01 Kompozycja i sposoby modulowania ekspresji czynnika b dopełniacza

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US (5) US10280423B2 (pl)
EP (3) EP3137596B1 (pl)
JP (2) JP6637442B2 (pl)
KR (1) KR102369736B1 (pl)
CN (2) CN106232804B (pl)
AU (1) AU2015252858C1 (pl)
BR (1) BR112016022593B1 (pl)
CA (2) CA2943894C (pl)
CL (2) CL2016002764A1 (pl)
CR (2) CR20160554A (pl)
DK (1) DK3137596T3 (pl)
DO (1) DOP2016000291A (pl)
EA (1) EA036496B1 (pl)
ES (2) ES2745825T3 (pl)
HR (1) HRP20191664T1 (pl)
HU (1) HUE046272T2 (pl)
IL (2) IL247883B (pl)
LT (1) LT3137596T (pl)
MX (1) MX380866B (pl)
PE (2) PE20190626A1 (pl)
PH (1) PH12016502062B1 (pl)
PL (2) PL3608406T3 (pl)
PT (1) PT3137596T (pl)
RS (1) RS59182B1 (pl)
RU (1) RU2701645C2 (pl)
SG (2) SG11201608502TA (pl)
SI (1) SI3137596T1 (pl)
UA (1) UA121656C2 (pl)
WO (1) WO2015168635A2 (pl)

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TW202134435A (zh) 2019-10-22 2021-09-16 美商阿尼拉製藥公司 補體成分c3 irna組成物及其使用方法
JP6961860B1 (ja) 2019-12-26 2021-11-05 エーザイ・アール・アンド・ディー・マネジメント株式会社 補体c5の発現を抑制する二本鎖リボ核酸を含む医薬組成物
MX2022013606A (es) * 2020-04-30 2023-01-16 Alnylam Pharmaceuticals Inc Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas.
JP2024525868A (ja) 2021-07-17 2024-07-12 サーナオミクス インコーポレイテッド 産物及び組成物
KR20240051221A (ko) 2021-09-02 2024-04-19 사일런스 테라퓨틱스 게엠베하 세포에서 보체 인자 b (cfb)의 발현을 억제하기 위한 핵산
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
PE20241340A1 (es) 2021-11-24 2024-07-03 Adarx Pharmaceuticals Inc Composiciones moduladoras de complemento del factor b y metodos de uso de estas
AU2023372971A1 (en) * 2022-11-02 2025-05-15 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
WO2024197017A2 (en) * 2023-03-21 2024-09-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement factor b (cfb) pharmaceutical compositions thereof, and methods of use
TW202513799A (zh) * 2023-06-02 2025-04-01 大陸商維亞臻生物技術(蘇州)有限公司 用於免疫類疾病的雙鏈核苷酸化合物及其用途
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
WO2025026228A1 (zh) * 2023-07-28 2025-02-06 苏州炫景生物科技有限公司 Cfb抑制剂组合物及其应用
WO2025151250A2 (en) * 2024-01-09 2025-07-17 Adarx Pharmaceuticals, Inc. Complement factor b-modulating compositions and methods of use thereof
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