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UY40039A - COMPOSICIONES DE ARNi DEL COMPONENTE DEL COMPLEMENTO C5 Y MÉTODOS PARA SU USO - Google Patents

COMPOSICIONES DE ARNi DEL COMPONENTE DEL COMPLEMENTO C5 Y MÉTODOS PARA SU USO

Info

Publication number
UY40039A
UY40039A UY0001040039A UY40039A UY40039A UY 40039 A UY40039 A UY 40039A UY 0001040039 A UY0001040039 A UY 0001040039A UY 40039 A UY40039 A UY 40039A UY 40039 A UY40039 A UY 40039A
Authority
UY
Uruguay
Prior art keywords
complement component
methods
rnai
compositions
rnai compositions
Prior art date
Application number
UY0001040039A
Other languages
English (en)
Inventor
Martin Maier
Klaus Charisse
Kevin Fitzgerald
James Butler
Brian Bettencourt
Anna Borodovsky
Satyanarayana Kuchimanchi
Muthiah Manoharan
Donald Foster
G Kallanthothil Rajeev
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 UY40039A publication Critical patent/UY40039A/es

Links

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
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    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
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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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • 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
    • C12N2310/3527—Other alkyl chain
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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Abstract

La invención se relaciona con ARNi, por ejemplo, composiciones de ácido ribonucleico de hebra doble (ARNhd), dirigido al gen del componente del complemento C5, y métodos para usar dicho ARNi, por ejemplo, ARNhd, composiciones para inhibir la expresión de C5 y para tratar sujetos que tienen una enfermedad asociada con el componente del complemento C5, por ejemplo, hemoglobinuria paroxística nocturna. ES DIVISIONAL DE 35442 de fecha 14/03/2014
UY0001040039A 2013-03-14 2014-03-14 COMPOSICIONES DE ARNi DEL COMPONENTE DEL COMPLEMENTO C5 Y MÉTODOS PARA SU USO UY40039A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201361782531P 2013-03-14 2013-03-14
US201361837399P 2013-06-20 2013-06-20
US201361904579P 2013-11-15 2013-11-15
US201361912777P 2013-12-06 2013-12-06
US201461942367P 2014-02-20 2014-02-20
UY0001035442A UY35442A (es) 2013-03-14 2014-03-14 COMPOSICIONES DE ARNi DEL COMPONENTE DEL COMPLEMENTO C5 Y MÉTODOS PARA SU USO

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Publication Number Publication Date
UY40039A true UY40039A (es) 2023-01-31

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UY0001040039A UY40039A (es) 2013-03-14 2014-03-14 COMPOSICIONES DE ARNi DEL COMPONENTE DEL COMPLEMENTO C5 Y MÉTODOS PARA SU USO
UY0001035442A UY35442A (es) 2013-03-14 2014-03-14 COMPOSICIONES DE ARNi DEL COMPONENTE DEL COMPLEMENTO C5 Y MÉTODOS PARA SU USO

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US (7) US9249415B2 (es)
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JP (4) JP6524060B2 (es)
KR (4) KR102342916B1 (es)
CN (2) CN114015692B (es)
AU (3) AU2014244116B9 (es)
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CR (1) CR20150493A (es)
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DO (1) DOP2015000234A (es)
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HR (1) HRP20171813T1 (es)
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PL (1) PL2970974T3 (es)
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RS (1) RS56663B1 (es)
SG (3) SG10201700239UA (es)
SI (1) SI2970974T1 (es)
SM (1) SMT201700552T1 (es)
TW (4) TWI745684B (es)
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WO (1) WO2014160129A2 (es)

Families Citing this family (62)

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