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DK3607068T3 - RNA-interferensmedieret inhibering af TMPRSS6 - Google Patents

RNA-interferensmedieret inhibering af TMPRSS6 Download PDF

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Publication number
DK3607068T3
DK3607068T3 DK18715705.2T DK18715705T DK3607068T3 DK 3607068 T3 DK3607068 T3 DK 3607068T3 DK 18715705 T DK18715705 T DK 18715705T DK 3607068 T3 DK3607068 T3 DK 3607068T3
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Prior art keywords
tmprss6
rna interference
mediated inhibition
mediated
inhibition
Prior art date
Application number
DK18715705.2T
Other languages
English (en)
Inventor
Sibylle Dames
Ute Schaeper
Adrien Weingärtner
Judith Hauptmann
Christian Frauendorf
Lucas Bethge
Original Assignee
Silence Therapeutics Gmbh
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
Priority claimed from EP17165007.0A external-priority patent/EP3385380A1/en
Application filed by Silence Therapeutics Gmbh filed Critical Silence Therapeutics Gmbh
Application granted granted Critical
Publication of DK3607068T3 publication Critical patent/DK3607068T3/da

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    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
DK18715705.2T 2017-04-05 2018-04-05 RNA-interferensmedieret inhibering af TMPRSS6 DK3607068T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP17165007.0A EP3385380A1 (en) 2017-04-05 2017-04-05 Products and compositions
EP17201405 2017-11-13
PCT/EP2018/058764 WO2018185240A1 (en) 2017-04-05 2018-04-05 Products and compositions

Publications (1)

Publication Number Publication Date
DK3607068T3 true DK3607068T3 (da) 2023-01-23

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DK18715705.2T DK3607068T3 (da) 2017-04-05 2018-04-05 RNA-interferensmedieret inhibering af TMPRSS6

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Country Link
US (2) US11174483B2 (da)
EP (2) EP4219714A3 (da)
JP (4) JP7247167B2 (da)
KR (1) KR102589803B1 (da)
CN (2) CN110573619B (da)
AU (2) AU2018247924B2 (da)
CA (2) CA3057561A1 (da)
DK (1) DK3607068T3 (da)
ES (1) ES2936109T3 (da)
FI (1) FI3607068T3 (da)
HR (1) HRP20221547T1 (da)
HU (1) HUE061894T2 (da)
IL (2) IL269576B2 (da)
LT (1) LT3607068T (da)
PL (1) PL3607068T3 (da)
RS (1) RS63887B1 (da)
SI (1) SI3607068T1 (da)
WO (1) WO2018185240A1 (da)

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JP7155239B2 (ja) * 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
CA3056179A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
EP3598995A1 (en) * 2018-07-26 2020-01-29 Silence Therapeutics GmbH Products and compositions
CN111973619B (zh) * 2019-05-23 2024-01-30 苏州瑞博生物技术股份有限公司 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
MX2023012834A (es) 2021-04-27 2023-11-08 Silence Therapeutics Gmbh Inhibidor de la matriptasa-2 para el tratamiento de trastornos mieloproliferativos.
TWI868755B (zh) * 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
WO2024060649A1 (zh) * 2022-09-29 2024-03-28 广州必贝特医药股份有限公司 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用
AU2024244929A1 (en) 2023-03-27 2025-09-11 New York Blood Center, Inc. Compounds and compositions for use in stem cell transplantation
WO2025132845A1 (en) 2023-12-21 2025-06-26 Silence Therapeutics Gmbh Galnac phosphoramidites and their uses
WO2025163186A1 (en) * 2024-02-01 2025-08-07 Silence Therapeutics Gmbh Conjugated nucleic acids and nucleic acids comprising locked nucleosides and inverted nucleotides for inhibiting gene expression in a cell

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CA3217805A1 (en) * 2011-03-29 2012-10-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of tmprss6 gene
EP3650544A1 (en) * 2011-11-07 2020-05-13 Ionis Pharmaceuticals, Inc. Modulation of tmprss6 expression
AP2015008862A0 (en) 2013-05-22 2015-11-30 Alnylam Pharmaceuticals Inc Tmprss6 irna compositions and methods of use thereof
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3204397B1 (en) * 2014-10-10 2024-10-02 F. Hoffmann-La Roche AG Galnac phosphoramidites, nucleic acid conjugates thereof and their use
WO2016085852A1 (en) * 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
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EP3228326A1 (en) 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
JP7155239B2 (ja) * 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
CA3056179A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions

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HUE061894T2 (hu) 2023-08-28
ES2936109T3 (es) 2023-03-14
PL3607068T3 (pl) 2023-03-20
AU2019247637A1 (en) 2020-10-22
KR102589803B1 (ko) 2023-10-16
JP7419252B2 (ja) 2024-01-22
LT3607068T (lt) 2023-02-10
CN110573619A (zh) 2019-12-13
SI3607068T1 (sl) 2023-02-28
JP7495544B2 (ja) 2024-06-04
IL277636B2 (en) 2025-03-01
RS63887B1 (sr) 2023-02-28
AU2019247637B2 (en) 2025-06-05
AU2018247924B2 (en) 2023-02-02
IL277636B1 (en) 2024-11-01
EP4219714A2 (en) 2023-08-02
IL269576B2 (en) 2024-06-01
JP2024036347A (ja) 2024-03-15
US11174483B2 (en) 2021-11-16
JP2020512838A (ja) 2020-04-30
HRP20221547T1 (hr) 2023-03-03
CN112424354A (zh) 2021-02-26
IL269576B1 (en) 2024-02-01
AU2018247924A1 (en) 2019-11-14
WO2018185240A1 (en) 2018-10-11
IL277636A (en) 2020-11-30
EP4219714A3 (en) 2023-08-30
CN110573619B (zh) 2024-11-29
JP7247167B2 (ja) 2023-03-28
JP2023068035A (ja) 2023-05-16
CA3095523A1 (en) 2019-10-10
EP3607068B1 (en) 2022-11-02
KR20190137842A (ko) 2019-12-11
IL269576A (en) 2019-11-28
US20220025377A1 (en) 2022-01-27
BR112019019921A2 (pt) 2020-04-22
CA3057561A1 (en) 2018-10-11
FI3607068T3 (fi) 2023-02-19
US20200208158A1 (en) 2020-07-02
EP3607068A1 (en) 2020-02-12
JP2021520207A (ja) 2021-08-19

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