公开(公告)号：US11234995B2

公开(公告)日：2022-02-01

申请号：US16068163

申请日：2017-01-05

申请人： OSAKA UNIVERSITY ,  NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITY ,  NATIONAL INSTITUTES OF BIOMEDICAL INNOVATION, HEALTH AND NUTRITION

发明人： Masayuki Nakamori ,  Hideki Mochizuki ,  Satoshi Obika ,  Takanori Yokota ,  Tetuya Nagata ,  Yuya Kasahara

IPC分类号： C12N15/11 ,  A61K31/712 ,  C07K14/47 ,  C12N15/113 ,  C07H21/02 ,  C12N15/09 ,  A61P25/28 ,  A61P25/16

摘要： The present invention can provide a nucleic acid medicine which has a higher effect and a more prolonged effect of inhibiting the expression of α-synudein can be provided. Disclosed is the oligonucleotide or a pharmacologically acceptable salt thereof, the oligonucleotide containing at least one nucleoside structure represented by Formula (I): (where each of Base and A are defined substituent or structure), can bind to an α-synudein gene, has activity for inhibiting expression of the α-synudein gene, and is complementary to the α-synudein gene, and the oligonucleotide has a length of twelve to twenty bases.

公开(公告)号：US11672786B2

公开(公告)日：2023-06-13

申请号：US16325066

申请日：2017-08-12

申请人： The Hospital for Sick Children ,  Osaka University

发明人： Christopher E. Pearson ,  Masayuki Nakamori ,  Kazuhiko Nakatani

IPC分类号： A61K31/4375 ,  A61P25/28 ,  A61K9/00 ,  A61K9/127

CPC分类号： A61K31/4375 ,  A61K9/0019 ,  A61K9/127 ,  A61P25/28

摘要： Methods of treating diseases caused by repeat DNA instability are described herein. The methods described herein can inhibit the further expansion of repeat DNA and, in some instances, reduce the size of the repeat DNA (e.g., reduce the number of repeats).

公开(公告)号：US20210283114A1

公开(公告)日：2021-09-16

申请号：US16325066

申请日：2017-08-12

申请人： The Hospital for Sick Children ,  Osaka University

发明人： Christopher E. Pearson ,  Masayuki Nakamori ,  Kazuhiko Nakatani

IPC分类号： A61K31/4375 ,  A61K9/00 ,  A61K9/127 ,  A61P25/28

摘要： Methods of treating diseases caused by repeat DNA instability are described herein. The methods described herein can inhibit the further expansion of repeat DNA and, in some instances, reduce the size of the repeat DNA (e.g., reduce the number of repeats).

公开(公告)号：US20180200277A1

公开(公告)日：2018-07-19

申请号：US15743622

申请日：2016-07-07

申请人： Osaka University

发明人： Masayuki Nakamori ,  Hideki Mochizuki

IPC分类号： A61K31/7048 ,  A61K31/7052 ,  A61P21/00

CPC分类号： A61K31/7048 ,  A61K31/7052 ,  A61P21/00

摘要： The present invention provides a therapeutic agent for myotonic dystrophy which inhibits aberrant splicing responsible for myotonic dystrophy, resulting in an increase in a normally spliced product and thus improvement in a symptom of myotonic dystrophy, and is highly safe for use in long-term administration. The therapeutic agent for myotonic dystrophy comprises, as an active ingredient, at least one compound selected from the group consisting of erythromycin, clarithromycin and azithromycin, a pharmaceutically acceptable salt or hydrate thereof, or a prodrug thereof.