公开(公告)号：EP4005638A1

公开(公告)日：2022-06-01

申请号：EP20846028.7

申请日：2020-07-28

申请人： Takeda Pharmaceutical Company Limited

发明人： TANAKA, Yuta ,  TANAKA, Yuta ,  KIKUCHI, Fumiaki ,  YAMAMOTO, Takeshi ,  NAKAMURA, Minoru ,  TAKAMI, Kazuaki ,  MURAKAMI, Masataka ,  DAINI, Masaki ,  WADA, Yasufumi ,  KAKEGAWA, Keiko ,  KASAHARA, Takahito ,  OHASHI, Tomohiro ,  WANG, Junsi ,  IKEDA, Zenichi ,  PÜNNER, Florian ,  SETO, Masaki ,  MIKAMI, Satoshi ,  SASAKI, Minoru

IPC分类号： A61P25/16 ,  A61P25/28 ,  A61P39/02 ,  A61P43/00 ,  C07D471/04 ,  C07D487/04 ,  C07D498/04 ,  C07D519/00 ,  A61K31/4162 ,  A61K31/437 ,  A61K31/4439 ,  A61K31/444 ,  A61K31/4545 ,  A61K31/496 ,  A61K31/497 ,  A61K31/501 ,  A61K31/5025 ,  A61K31/506 ,  A61K31/519 ,  A61K31/5377 ,  A61K31/551

摘要： The present invention provides a compound having a glucosylceramide synthase inhibitory action, which is expected to be useful as an agent for the prophylaxis or treatment of lysosomal storage diseases (e.g., Gaucher's disease, Fabry's disease, GM1-gangliosidosis, GM2 activator deficiency, Tay-Sachs disease, Sandhoffs disease), neurodegenerative diseases (e.g., Parkinson's disease, Lewy body dementia, multiple-system atrophy) and the like.
The present invention relates to a compound represented by the formula (I):

wherein each symbol is as described in the description, or a salt thereof.

公开(公告)号：EP4034536A1

公开(公告)日：2022-08-03

申请号：EP20800345.9

申请日：2020-09-24

申请人： Takeda Pharmaceutical Company Limited

发明人： ITO, Masahiro ,  SUGIYAMA, Hideyuki ,  YAMAMOTO, Takeshi ,  KAKEGAWA, Keiko ,  LI, Jinxing ,  WANG, Junsi ,  KASAHARA, Takahito ,  YOSHIKAWA, Masato

IPC分类号： C07D413/04 ,  A61P25/28 ,  A61K31/4245 ,  C07D413/14

公开(公告)号：EP4032551A1

公开(公告)日：2022-07-27

申请号：EP20864622.4

申请日：2020-09-16

申请人： National University Corporation Tokyo Medical and Dental University ,  Takeda Pharmaceutical Company Limited

发明人： YOKOTA, Takanori ,  NAGATA, Tetsuya ,  FURUKAWA, Hideki ,  YOGO, Takatoshi ,  NAKAGAWA, Yasuo ,  SASAKI, Shigekazu ,  TOKUNOH, Ryosuke ,  SEKI, Tomohiro ,  HIDAKA, Kosuke ,  KIKUCHI, Fumiaki ,  KUBO, Osamu ,  KASAHARA, Takahito ,  KOJIMA, Takuto ,  WANG, Junsi ,  TOKUNAGA, Norihito

IPC分类号： A61K48/00 ,  A61P25/00 ,  A61P43/00 ,  C12N5/079 ,  C12N5/10 ,  A61K47/54 ,  C12N15/113 ,  A61K31/713

摘要： The object of the present invention is to provide a nucleic acid agent that is efficiently delivered to the nervous system, for example, the central nervous system to which drug delivery can be prevented by BBB, and produces an antisense effect on the target transcriptional product at the delivery site, and a composition comprising the same. In an embodiment, the present invention provides a double-stranded nucleic acid complex formed by annealing a first nucleic acid strand that hybridizes to a part of a target transcriptional product and has an antisense effect on the target transcriptional product, and a second nucleic acid strand that comprises a base sequence complementary to the first nucleic acid strand and is bound to a C 22-35 alkyl group optionally substituted with a hydroxy group or an analog thereof.