Login| Sign Up| Help| Contact|

Patent Searching and Data


Title:
NOVEL AMYLOID FIBRIL FORMATION INHIBITOR
Document Type and Number:
WIPO Patent Application WO/2016/199892
Kind Code:
A1
Abstract:
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-β-CDE) of glucuronylglucosyl-β-cyclodextrin (GUG-β-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.

Inventors:
JONO HIROFUMI (JP)
ARIMA HIDETOSHI (JP)
ANDO YUKIO (JP)
MOTOYAMA KEIICHI (JP)
HIGASHI TAISHI (JP)
Application Number:
PCT/JP2016/067373
Publication Date:
December 15, 2016
Filing Date:
June 10, 2016
Export Citation:
Click for automatic bibliography generation   Help
Assignee:
NAT UNIV CORP KUMAMOTO UNIV (JP)
International Classes:
A61K31/724; A61K31/132; A61K31/7016; A61K31/7105; A61P25/00; A61P25/14; A61P25/28; A61P43/00
Domestic Patent References:
WO2011002026A12011-01-06
Foreign References:
JP2010090054A2010-04-22
Other References:
ANNO, TAKAYUKI ET AL.: "Potential use of glucuronylglucosyl-beta-cyclodextrin/dendrimer conjugate (G2) as a siRNA carrier for the treatment of familial amyloidotic polyneuropathy", J DRUG TARGET, vol. 22, no. 10, 2014, pages 883 - 890, XP009507776, ISSN: 1061-186X
HAYASHI, YUYA ET AL.: "Potential Use of Lactosylated Dendrimer (G3)/a-Cyclodextrin Conjugates as Hepatocyte-Specific siRNA Carriers for the Treatment of Familial Amyloidotic Polyneuropathy", MOL PHARM, vol. 9, no. 6, 2012, pages 1645 - 1653, XP055333612, ISSN: 1543-8384
HIDETOSHI ARIMA ET AL.: "Cyclodextrin/Dendrimer conjugates as siRNA delivery carriers", DRUG DELIV SYST, vol. 25, no. 6, 2010, pages 598 - 606, XP055333615, ISSN: 0913-5006
JONO, HIROFUMI ET AL.: "Cyclodextrin, a novel therapeutic tool for suppressing amyloidogenic transthyretin misfolding in transthyretin-related amyloidosis", BIOCHEM J, vol. 437, no. 1, 2011, pages 35 - 42, XP055333617, ISSN: 0264-6021
JONO, HIROFUMI ET AL.: "Potential use of glucuronylglucosyl-beta-cyclodextrin as a novel therapeutic tool for familial amyloidotic polyneuropathy", AMYLOID, vol. 19, no. Sl, 2012, pages 50 - 52, XP009507761, ISSN: 1350-6129
HAYASHI, YUYA ET AL.: "Systemic delivery of transthyretin siRNA mediated by lactosylated dendrimer/beta-cyclodextrin conjugates into hepatocyte for familial amyloidotic polyneuropathy therapy", AMYLOID, vol. 19, no. Sl, 2012, pages 47 - 49, XP009507762, ISSN: 1350-6129
Attorney, Agent or Firm:
OSAWA KENICHI (JP)
Ken-ichi Osawa (JP)
Download PDF: