CL2016000188A1 - Composiciones de arní de serpinc1 y métodos de uso de las mismas - Google Patents

Composiciones de arní de serpinc1 y métodos de uso de las mismas

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Publication number
CL2016000188A1
CL2016000188A1 CL2016000188A CL2016000188A CL2016000188A1 CL 2016000188 A1 CL2016000188 A1 CL 2016000188A1 CL 2016000188 A CL2016000188 A CL 2016000188A CL 2016000188 A CL2016000188 A CL 2016000188A CL 2016000188 A1 CL2016000188 A1 CL 2016000188A1
Authority
CL
Chile
Prior art keywords
serpinc1
harness
compositions
methods
cell
Prior art date
Application number
CL2016000188A
Other languages
English (en)
Inventor
Muthiah Manoharan
Martin Maier
Satyanarayana Kuchimanchi
Klaus Charisse
Brian Bettencourt
Akin Akinc
Alfica Sehgal
Ivanka Toudjarska
Donald Foster
Stuart Milstein
Kallanthottathil G Rajev
Original Assignee
Alnylam 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CL2016000188A1 publication Critical patent/CL2016000188A1/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
    • 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
    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3523—Allyl
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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

Abstract

ÁCIDO RIBONUCLEICO DE DOBLE HEBRA (ARNdh) PARA INHIBIR LA EXPRESIÓN DE SERPINC1; CÉLULA QUE LO CONTIENE; VECTOR QUE LO CODIFICA; CÉLULA HUÉSPED;COMPOSICIÓN FARMACÉUTICA; Y USO EN PREVENCIÓN O TRATAMIENTO DE TRASTORNOS QUE SE BENEFICIAN DE REDUCCIÓN DE EXPRESIÓN DE SERPINC1, TAL COMO HEMOFILIA (DIVISIONAL DE SOL. N°2869-14).
CL2016000188A 2012-04-26 2016-01-25 Composiciones de arní de serpinc1 y métodos de uso de las mismas CL2016000188A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201261638952P 2012-04-26 2012-04-26
US201261669249P 2012-07-09 2012-07-09
US201261734573P 2012-12-07 2012-12-07
US13/837,129 US9127274B2 (en) 2012-04-26 2013-03-15 Serpinc1 iRNA compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
CL2016000188A1 true CL2016000188A1 (es) 2016-08-19

Family

ID=49484030

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2016000188A CL2016000188A1 (es) 2012-04-26 2016-01-25 Composiciones de arní de serpinc1 y métodos de uso de las mismas

Country Status (25)

Country Link
US (6) US9127274B2 (es)
EP (2) EP4209592A1 (es)
JP (3) JP6466832B2 (es)
KR (4) KR20220038521A (es)
CN (4) CN110295167A (es)
AR (1) AR090869A1 (es)
AU (4) AU2013251494A1 (es)
BR (1) BR112014026758A2 (es)
CA (1) CA2869922A1 (es)
CL (1) CL2016000188A1 (es)
CO (1) CO7240361A2 (es)
ES (1) ES2934152T3 (es)
FI (1) FI2841443T3 (es)
HK (1) HK1204336A1 (es)
HR (1) HRP20221498T1 (es)
HU (1) HUE060846T2 (es)
IL (4) IL234941B (es)
LT (1) LT2841443T (es)
MX (2) MX365419B (es)
PL (1) PL2841443T3 (es)
PT (1) PT2841443T (es)
RS (1) RS63838B1 (es)
RU (2) RU2019115881A (es)
SI (1) SI2841443T1 (es)
WO (1) WO2013163430A2 (es)

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