PL3607068T3 - Hamowanie TMPRSS6 mediowane interferencją RNA - Google Patents

Hamowanie TMPRSS6 mediowane interferencją RNA

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Publication number
PL3607068T3
PL3607068T3 PL18715705.2T PL18715705T PL3607068T3 PL 3607068 T3 PL3607068 T3 PL 3607068T3 PL 18715705 T PL18715705 T PL 18715705T PL 3607068 T3 PL3607068 T3 PL 3607068T3
Authority
PL
Poland
Prior art keywords
tmprss6
rna interference
mediated inhibition
interference mediated
inhibition
Prior art date
Application number
PL18715705.2T
Other languages
English (en)
Inventor
Sibylle DAMES
Ute SCHAEPER
Judith HAUPTMANN
Christian Frauendorf
Lucas Bethge
Adrien WEINGÄRTNER
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
Publication of PL3607068T3 publication Critical patent/PL3607068T3/pl

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61K47/56Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/14Type of nucleic acid interfering N.A.
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
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    • C12N2310/3212'-O-R Modification
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    • C12N2310/34Spatial arrangement of the modifications
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    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
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  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
PL18715705.2T 2017-04-05 2018-04-05 Hamowanie TMPRSS6 mediowane interferencją RNA PL3607068T3 (pl)

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
PL3607068T3 true PL3607068T3 (pl) 2023-03-20

Family

ID=61899310

Family Applications (1)

Application Number Title Priority Date Filing Date
PL18715705.2T PL3607068T3 (pl) 2017-04-05 2018-04-05 Hamowanie TMPRSS6 mediowane interferencją RNA

Country Status (18)

Country Link
US (2) US11174483B2 (pl)
EP (2) EP3607068B1 (pl)
JP (4) JP7247167B2 (pl)
KR (1) KR102589803B1 (pl)
CN (2) CN110573619A (pl)
AU (2) AU2018247924B2 (pl)
CA (2) CA3057561A1 (pl)
DK (1) DK3607068T3 (pl)
ES (1) ES2936109T3 (pl)
FI (1) FI3607068T3 (pl)
HR (1) HRP20221547T1 (pl)
HU (1) HUE061894T2 (pl)
IL (2) IL269576B2 (pl)
LT (1) LT3607068T (pl)
PL (1) PL3607068T3 (pl)
RS (1) RS63887B1 (pl)
SI (1) SI3607068T1 (pl)
WO (1) WO2018185240A1 (pl)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11414660B2 (en) 2017-04-05 2022-08-16 Silence Therapeutics Gmbh Products and compositions
AU2018247925B2 (en) * 2017-04-05 2024-06-06 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
CA3216859A1 (en) * 2021-04-27 2022-11-03 Silence Therapeutics Gmbh Sirna targeting tmprss6 for the treatment of myeloproliferative disorders
TW202400193A (zh) 2022-06-24 2024-01-01 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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
EP2601204B1 (en) * 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
SG10201602369PA (en) * 2011-03-29 2016-05-30 Alnylam Pharmaceuticals Inc Double-Stranded RNA (dsRNA) For Inhibiting Expression Of TMPRSS6
ES2761343T3 (es) * 2011-11-07 2020-05-19 Ionis Pharmaceuticals Inc Modulación de la expresión de TMPRSS6
MY181888A (en) * 2013-05-22 2021-01-12 Alnylam Pharmaceuticals Inc Tmprss6 irna compositions and methods of use thereof
US10233448B2 (en) * 2014-08-20 2019-03-19 Alnylam Pharmaceuticals, Inc. Modified double-stranded RNA agents
WO2016085852A1 (en) * 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
WO2016149020A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
EP3228326A1 (en) 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate

Also Published As

Publication number Publication date
EP4219714A2 (en) 2023-08-02
HRP20221547T1 (hr) 2023-03-03
US20220025377A1 (en) 2022-01-27
EP3607068A1 (en) 2020-02-12
JP2021520207A (ja) 2021-08-19
EP4219714A3 (en) 2023-08-30
SI3607068T1 (sl) 2023-02-28
HUE061894T2 (hu) 2023-08-28
IL269576B1 (en) 2024-02-01
JP7419252B2 (ja) 2024-01-22
RS63887B1 (sr) 2023-02-28
CA3095523A1 (en) 2019-10-10
CN110573619A (zh) 2019-12-13
BR112019019921A2 (pt) 2020-04-22
IL269576A (en) 2019-11-28
JP7495544B2 (ja) 2024-06-04
US20200208158A1 (en) 2020-07-02
AU2018247924B2 (en) 2023-02-02
AU2019247637A1 (en) 2020-10-22
US11174483B2 (en) 2021-11-16
DK3607068T3 (da) 2023-01-23
KR20190137842A (ko) 2019-12-11
JP7247167B2 (ja) 2023-03-28
IL269576B2 (en) 2024-06-01
ES2936109T3 (es) 2023-03-14
WO2018185240A1 (en) 2018-10-11
JP2023068035A (ja) 2023-05-16
CN112424354A (zh) 2021-02-26
EP3607068B1 (en) 2022-11-02
AU2018247924A1 (en) 2019-11-14
CA3057561A1 (en) 2018-10-11
IL277636A (en) 2020-11-30
JP2020512838A (ja) 2020-04-30
FI3607068T3 (fi) 2023-02-19
LT3607068T (lt) 2023-02-10
KR102589803B1 (ko) 2023-10-16
JP2024036347A (ja) 2024-03-15

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