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MX2018006989A - Metodos y composiciones para el tratamiento contra un trastorno asociado a serpinc1. - Google Patents

Metodos y composiciones para el tratamiento contra un trastorno asociado a serpinc1.

Info

Publication number
MX2018006989A
MX2018006989A MX2018006989A MX2018006989A MX2018006989A MX 2018006989 A MX2018006989 A MX 2018006989A MX 2018006989 A MX2018006989 A MX 2018006989A MX 2018006989 A MX2018006989 A MX 2018006989A MX 2018006989 A MX2018006989 A MX 2018006989A
Authority
MX
Mexico
Prior art keywords
serpinc1
compositions
methods
treating
double stranded
Prior art date
Application number
MX2018006989A
Other languages
English (en)
Inventor
Akinc Akin
Robbie Gabriel
SORENSEN Benny
GARG Pushkal
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of MX2018006989A publication Critical patent/MX2018006989A/es

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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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • C12N2310/335—Modified T or U
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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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    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • 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
    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La invención se refiere al ARNi, por ejemplo, el ácido ribonucleico bicatenario (ARN bicatenario), a las composiciones con especificidad de objetivo contra el gen Serpinc1 y a los métodos de uso de tales ARNi, por ejemplo, ARN bicatenario, a composiciones para inhibir la expresión de Serpinc1 y someter a tratamiento a los sujetos que tengan una enfermedad asociada a Serpinc1, por ejemplo, un trastorno hemorrágico, tal como una hemofilia.
MX2018006989A 2015-12-07 2016-12-07 Metodos y composiciones para el tratamiento contra un trastorno asociado a serpinc1. MX2018006989A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562264013P 2015-12-07 2015-12-07
US201662315228P 2016-03-30 2016-03-30
US201662366304P 2016-07-25 2016-07-25
US201662429241P 2016-12-02 2016-12-02
PCT/US2016/065245 WO2017100236A1 (en) 2015-12-07 2016-12-07 Methods and compositions for treating a serpinc1-associated disorder

Publications (1)

Publication Number Publication Date
MX2018006989A true MX2018006989A (es) 2018-09-05

Family

ID=58800158

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018006989A MX2018006989A (es) 2015-12-07 2016-12-07 Metodos y composiciones para el tratamiento contra un trastorno asociado a serpinc1.
MX2024008022A MX2024008022A (es) 2015-12-07 2018-06-07 Composiciones y kits para el tratamiento de la hemofilia a o b.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2024008022A MX2024008022A (es) 2015-12-07 2018-06-07 Composiciones y kits para el tratamiento de la hemofilia a o b.

Country Status (25)

Country Link
US (2) US11091759B2 (es)
EP (2) EP4424828A1 (es)
JP (3) JP6985283B2 (es)
KR (3) KR20230119027A (es)
CN (2) CN108601795A (es)
AU (3) AU2016368289C1 (es)
BR (1) BR112018011450A2 (es)
CA (1) CA3007014A1 (es)
CO (1) CO2018006590A2 (es)
DK (1) DK3386518T3 (es)
ES (1) ES3041380T3 (es)
FI (1) FI3386518T3 (es)
HR (1) HRP20251125T1 (es)
HU (1) HUE072861T2 (es)
IL (2) IL308717A (es)
LT (1) LT3386518T (es)
MX (2) MX2018006989A (es)
PL (1) PL3386518T3 (es)
PT (1) PT3386518T (es)
RS (1) RS67250B1 (es)
RU (2) RU2021124592A (es)
SG (2) SG10201913085TA (es)
SI (1) SI3386518T1 (es)
TW (3) TW202423457A (es)
WO (1) WO2017100236A1 (es)

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