BR112015022876A2 - agente, composição farmacêutica e método para inibição da expressão do componente do complemento c5, uso do referido agente, vetor e célula. - Google Patents

agente, composição farmacêutica e método para inibição da expressão do componente do complemento c5, uso do referido agente, vetor e célula.

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Publication number
BR112015022876A2
BR112015022876A2 BR112015022876A BR112015022876A BR112015022876A2 BR 112015022876 A2 BR112015022876 A2 BR 112015022876A2 BR 112015022876 A BR112015022876 A BR 112015022876A BR 112015022876 A BR112015022876 A BR 112015022876A BR 112015022876 A2 BR112015022876 A2 BR 112015022876A2
Authority
BR
Brazil
Prior art keywords
complement component
expression
dsrna
agent
double
Prior art date
Application number
BR112015022876A
Other languages
English (en)
Other versions
BR112015022876B1 (pt
Inventor
Borodovsky Anna
Bettencourt Brian
Foster Donald
Butler James
G Rajeev Kallanthottathil
Fitzgerald Kevin
Charisse Klaus
Maier Martin
Manoharan Muthiah
Kuchimanchi Satyanarayana
Original Assignee
Alnylam Pharmaceuticals Inc
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of BR112015022876A2 publication Critical patent/BR112015022876A2/pt
Publication of BR112015022876B1 publication Critical patent/BR112015022876B1/pt

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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
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Abstract

complement component c5 irna compositions and methods of use thereof abstract of the invention the invention relates to irna, e.g., double-stranded ribonucleic acid (dsrna), compositions targeting the complement component c5 gene, and methods of using such irna, e.g., dsrna, compositions to inhibit expression of c5 and to treat subjects having a complement component c5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria. 1 the species oligo name reflects the genbank record (e.g., nm_001735.2) and the position in the nucleotide sequence of the genbank record (e.g., 1517-1539) that the antisense strand targets. 2 the number following the decimal point refers to the lot number. 3 um = unmodified tradução do resumo resumo "um agente de ácido ribonucleico de fita dupla (dsrna) e um agente de rnai de fita dupla para inibição da expressão do componente do complemento c5, um agente de rnai de fita dupla capaz de inibir a expressão do componente do complemento c5 em uma célula, uma célula, um vetor codificando pelo menos uma fita de um agente de dsrna, uma composição farmacêutica para inibição da expressão de um gene do componente do complemento c5, um método de inibição da expressão do componente do complemento c5 em uma célula, um método de tratamento de um sujeito tendo uma doença ou distúrbio que beneficiaria de redução na expressão do componente do complemento c5 e um método de prevenção de pelo menos um sintoma em um sujeito tendo uma doença ou distúrbio que beneficiaria de redução na expressão do componente do complemento c5" a invenção se relaciona com irna, p.ex., ácido ribonucleico de fita dupla (dsrna), composições se dirigindo ao gene do componente do complemento c5, e métodos de uso de tal irna, p.ex., dsrna, composições para inibir a expressão de c5 e para tratar sujeitos tendo uma doença associada ao componente do complemento c5, p.ex., hemoglobinúria paroxística noturna. 24/24 1/1
BR112015022876-3A 2013-03-14 2014-03-13 Agente de rna fita dupla, composição farmacêutica e método para inibição da expressão do componente do complemento c5 em uma célula BR112015022876B1 (pt)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201361782531P 2013-03-14 2013-03-14
US61/782,531 2013-03-14
US201361837399P 2013-06-20 2013-06-20
US61/837,399 2013-06-20
US201361904579P 2013-11-15 2013-11-15
US61/904,579 2013-11-15
US201361912777P 2013-12-06 2013-12-06
US61/912,777 2013-12-06
US201461942367P 2014-02-20 2014-02-20
US61/942,367 2014-02-20
PCT/US2014/025882 WO2014160129A2 (en) 2013-03-14 2014-03-13 Complement component c5 irna compositions and methods of use thereof

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