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NOVEL AMYLOID FIBRIL FORMATION INHIBITOR

外国特許コード F170008947
整理番号 (S2015-1616-N0)
掲載日 2017年2月2日
出願国 世界知的所有権機関(WIPO)
国際出願番号 2016JP067373
国際公開番号 WO 2016199892
国際出願日 平成28年6月10日(2016.6.10)
国際公開日 平成28年12月15日(2016.12.15)
優先権データ
  • 特願2015-117150 (2015.6.10) JP
発明の名称 (英語) NOVEL AMYLOID FIBRIL FORMATION INHIBITOR
発明の概要(英語) 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.
特許請求の範囲(英語) [claim1]
1. The amyloid tissue formation inhibiter which includes the mixture which consists of RNA which causes RNA interference vis-a-vis mRNA of joining union and [toransusairechin] [shikurodekisutorin] and [poriamidoamindendorima] with as the active ingredient.
[claim2]
2. Aforementioned [shikurodekisutorin], [gurukuronirugurukoshiru] - in the claim 1 which is ***.beta.-shikurodekisutorin (GUG-.beta.-CyD) the amyloid tissue formation inhibiter of statement.
[claim3]
3. The description above RNA, is shRNA or siRNA, in claim 1 or 2 the amyloid tissue formation inhibiter of statement.
[claim4]
4. The description above RNA, in the claim 3 which is shRNA the amyloid tissue formation inhibiter of statement.
[claim5]
5. Either of the claim 2-4 where the aforementioned joining union, [gurukuronirugurikoshiru] - is the joining union (GUG-.beta.-CDE) with ***.beta.-shikurodekisutorin and [poriamidoamindendorima], is GUG-.beta.-CDE (G2 and DS 1.2), GUG-.beta.-CDE (G2 and DS 1.8), GUG-.beta.-CDE (G2 and DS 2.5) or GUG-.beta.-CDE (G2 and DS 4.5) in one the amyloid tissue formation inhibiter of statement.
[claim6]
6. In the aforementioned mixture, the charge ratio of GUG-.beta.-CDE /RNA, either of the claim 2-5 which is the 20-100 in one the amyloid tissue formation inhibiter of statement.
[claim7]
7. Either of the claim 1-6 prevention of the amyloidosis which includes the amyloid tissue formation inhibiter of statement in one and/or the medicine composition for remedying.
[claim8]
8. The aforementioned amyloidosis, family characteristic [amiroidoshisuporiniyuropachi] (FAP), in the claim 7 which is the Alzheimer's disease, the senile systemic characteristic amyloidosis (SSA) or the AA amyloidosis the medicine composition of statement.
  • 出願人(英語)
  • ※2012年7月以前掲載分については米国以外のすべての指定国
  • KUMAMOTO UNIVERSITY
  • 発明者(英語)
  • JONO HIROFUMI
  • ARIMA HIDETOSHI
  • ANDO YUKIO
  • MOTOYAMA KEIICHI
  • HIGASHI TAISHI
国際特許分類(IPC)
指定国 (WO2016199892)
National States: AE AG AL AM AO AT AU AZ BA BB BG BH BN BR BW BY BZ CA CH CL CN CO CR CU CZ DE DK DM DO DZ EC EE EG ES FI GB GD GE GH GM GT HN HR HU ID IL IN IR IS JP KE KG KN KP KR KZ LA LC LK LR LS LU LY MA MD ME MG MK MN MW MX MY MZ NA NG NI NO NZ OM PA PE PG PH PL PT QA RO RS RU RW SA SC SD SE SG SK SL SM ST SV SY TH TJ TM TN TR TT TZ UA UG US UZ VC VN ZA ZM ZW
ARIPO: BW GH GM KE LR LS MW MZ NA RW SD SL SZ TZ UG ZM ZW
EAPO: AM AZ BY KG KZ RU TJ TM
EPO: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
OAPI: BF BJ CF CG CI CM GA GN GQ GW KM ML MR NE SN ST TD TG
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