公开(公告)号：US20230193265A1

公开(公告)日：2023-06-22

申请号：US17925225

申请日：2021-05-12

Applicant: OSAKA UNIVERSITY

Inventor： Kazuhiko NAKATANI ,  Jun MATSUMOTO ,  Tatsumasa OKAMOTO ,  Chikara DOHNO ,  Asako SEIKE ,  Masayuki NAKAMORI

IPC: C12N15/113 ,  A61K31/496 ,  C07D401/04 ,  A61P21/00

CPC classification number: C12N15/113 ,  A61K31/496 ,  C07D401/04 ,  A61P21/00

Abstract: Provided is a novel agent capable of binding to a CUG repeat sequence. The agent comprises a compound A having a binding response of 10 resonance units (RU) or more at 25 nM to a (CUG)9 RNA immobilized at 401 RU as determined by surface plasmon resonance.

公开(公告)号：US20200172903A1

公开(公告)日：2020-06-04

申请号：US16616743

申请日：2018-07-03

Applicant: OSAKA UNIVERSITY ,  DAIICHI SANKYO COMPANY, LIMITED

Inventor： Masayuki NAKAMORI ,  Hideki MOCHIZUKI ,  Satoshi OBIKA ,  Makoto KOIZUMI ,  Akifumi NAKAMURA ,  Kiyosumi TAKAISHI ,  Yumiko ASAHI

IPC: C12N15/113 ,  A61P25/16 ,  A61P25/28

Abstract: The objective of the present invention is to provide nucleic acid therapeutics which exhibits more excellent effect and which shows a substantivity for a prolonged period to suppress an expression of α-synuclein. The oligonucleotide or a pharmacologically acceptable salt thereof according to the present invention is characterized in comprising at least one 2′-O,4′-C-ethylene nucleoside, wherein the oligonucleotide can hybridize with α-synuclein gene, has an activity to suppress an expression of the α-synuclein gene, and is complementary to the α-synuclein gene, 5′ end of the oligonucleotide is a nucleotide complementary to the specific nucleotide, the oligonucleotide is complementary to at least a part of SEQ ID NO: 1, and the oligonucleotide has a length of 13 or more and 15 or less nucleotides.

公开(公告)号：US20190008886A1

公开(公告)日：2019-01-10

申请号：US16068163

申请日：2017-01-05

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

Inventor： Masayuki NAKAMORI ,  Hideki MOCHIZUKI ,  Satoshi OBIKA ,  Takanori YOKOTA ,  Tetuya NAGATA ,  Yuya KASAHARA

IPC: A61K31/712 ,  C12N15/113 ,  C07K14/47 ,  A61P25/28 ,  A61P25/16

Abstract: 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.