NOVEL AMYLOID FIBRIL FORMATION INHIBITOR

The purpose of the present invention is to provide a therapeutic agent that is more effective in refractory amyloidosis. More specifically, it is to provide a novel substance that is highly safe and has a TTR protein amyloid fibril formation-inhibiting effect with which it is possible to inhibit the...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: HIGASHI, Taishi, ANDO, Yukio, MOTOYAMA, Keiichi, ARIMA, Hidetoshi, JONO, Hirofumi
Format: Patent
Sprache:eng ; fre ; ger
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Beschreibung
Zusammenfassung:The purpose of the present invention is to provide a therapeutic agent that is more effective in refractory amyloidosis. More specifically, it is to provide a novel substance that is highly safe and has a TTR protein amyloid fibril formation-inhibiting effect with which it is possible to inhibit the formation of amyloid fibrils better than conventional therapeutic agents. Provided by the invention is an amyloid fibril formation inhibitor containing as an active ingredient a complex comprsing a conjugate (GUG-B-CDE) of glucuronylglucosyl-B-cyclodextrin (GUG-B-CyD) and polyamide amine dendrimer having an alkylene diamine as the core and RNA that causes RNA interference in the mRNA of transthyretin (TTR). Also provided by the present invention is a pharmaceutical composition for the prevention and/or treatment of amyloidosis including the amyloid fibril formation inhibitor.