公开(公告)号：US20210087560A1

公开(公告)日：2021-03-25

申请号：US16967483

申请日：2019-02-06

申请人： NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY ,  NIPPON MEDICAL SCHOOL FOUNDATION ,  DAIICHI SANKYO COMPANY, LIMITED

发明人： Ken Inoue ,  Heng Li ,  Takashi Okada ,  Yu Ohki ,  Makoto Koizumi

IPC分类号： C12N15/113 ,  C12N15/86

摘要： An object of the present invention is to provide a vector capable of oligodendrocyte-specifically suppressing expression of the PLP1 gene for treating PMD caused by abnormality of the PLP1 gene, and a promoter and miRNA therefor, and a pharmaceutical composition comprising the vector. The oligodendrocyte-specific promoter of the present invention comprises a nucleic acid having a sequence identity of at least 90% to a nucleotide sequence set forth in SEQ ID NO: 1. The miRNA of the present invention specific to the PLP1 gene comprises a pair of nucleotide sequences consisting of a specific antisense sequence and sense sequence.

公开(公告)号：US20220127606A1

公开(公告)日：2022-04-28

申请号：US17310239

申请日：2020-01-29

申请人： NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY ,  NIPPON MEDICAL SCHOOL FOUNDATION

发明人： Takashi Okada ,  Hiromi KINOH ,  Yoshitsugu AOKI ,  Shin'ichi TAKEDA

IPC分类号： C12N15/113 ,  A61P21/04 ,  A61K47/64 ,  A61K9/10 ,  A61K9/14

摘要： Provided are a complex that comprises a nucleic acid-containing nanoparticle and a hollow particle of a non-enveloped virus, a method for producing the complex, and a pharmaceutical composition comprising the complex.

公开(公告)号：US11028131B2

公开(公告)日：2021-06-08

申请号：US16481159

申请日：2018-01-29

申请人： NIPPON MEDICAL SCHOOL FOUNDATION ,  NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY ,  TAKARA BIO INC.

发明人： Takashi Okada ,  Hironori Okada ,  Hiromi Kinoh ,  Tatsuji Enoki ,  Toshikazu Nishie ,  Junichi Mineno

IPC分类号： C12N7/00 ,  C12N15/00 ,  C07K14/005 ,  C12N15/85

摘要： The present invention provides: a mutant of adeno-associated virus (AAV) capsid protein, which contains at least one amino acid substitution in PLA2 domain when compared with the amino acid sequence for wild-type AAV capsid protein; a nucleic acid encoding the mutant; a cell containing the nucleic acid; a method for producing a recombinant AAV particle, comprising a step of culturing the cell to produce the recombinant AAV particle; a recombinant AAV particle containing the mutant; a composition containing the recombinant AAV particle; and a method for transferring a gene into a target cell, comprising a step of bringing the recombinant AAV particle into contact with the target cell.

公开(公告)号：US11191733B2

公开(公告)日：2021-12-07

申请号：US15445157

申请日：2017-02-28

申请人： National Center of Neurology and Psychiatry

发明人： Takashi Okada ,  Shin'ichi Takeda ,  Hiromi Kinoh

IPC分类号： A61K35/76 ,  A61K48/00 ,  A61K38/17 ,  A61K9/51 ,  A61K31/7088 ,  A61K31/713 ,  A61K9/14 ,  A61K47/42 ,  C12N5/00 ,  C12N7/00 ,  C12N15/113 ,  A61K47/26

摘要： An object of the present invention is to develop and provide a method for conveniently introducing a nucleic acid, a peptide, and/or a low-molecular-weight compound into an empty capsid with viral early infection activities kept. The present invention provides a method for producing a drug delivery particle, comprising the steps of: mixing an empty capsid or an empty particle with a drug including a nucleic acid, a peptide, and/or a low-molecular-weight compound in a solution comprising 0.1 to 20% of a surfactant; and keeping the obtained mixed solution at −5 to 50° C. to introduce the drug into the empty capsid or the empty particle.

公开(公告)号：US20170172936A1

公开(公告)日：2017-06-22

申请号：US15445157

申请日：2017-02-28

申请人： National Center of Neurology and Psychiatry

发明人： Takashi Okada ,  Shin'ichi Takeda ,  Hiromi Kinoh

IPC分类号： A61K9/51 ,  A61K48/00 ,  C12N5/00 ,  C12N15/113 ,  C12N7/00

CPC分类号： A61K9/5184 ,  A61K9/14 ,  A61K31/7088 ,  A61K31/713 ,  A61K47/26 ,  A61K47/42 ,  A61K48/0008 ,  A61K48/0091 ,  C12N5/00 ,  C12N7/00 ,  C12N15/113 ,  C12N2310/14 ,  C12N2320/32 ,  C12N2510/02 ,  C12N2710/10042 ,  C12N2750/00042 ,  C12N2750/00051 ,  C12N2750/14142

摘要： An object of the present invention is to develop and provide a method for conveniently introducing a nucleic acid, a peptide, and/or a low-molecular-weight compound into an empty capsid with viral early infection activities kept. The present invention provides a method for producing a drug delivery particle, comprising the steps of: mixing an empty capsid or an empty particle with a drug including a nucleic acid, a peptide, and/or a low-molecular-weight compound in a solution comprising 0.1 to 20% of a surfactant; and keeping the obtained mixed solution at −5 to 50° C. to introduce the drug into the empty capsid or the empty particle.

公开(公告)号：US11845952B2

公开(公告)日：2023-12-19

申请号：US16968397

申请日：2019-01-16

申请人： NIPPON MEDICAL SCHOOL FOUNDATION ,  TEIKA PHARMACEUTICAL CO., LTD.

发明人： Takashi Okada ,  Tsutomu Igarashi ,  Asaka Shiozawa

IPC分类号： C12N15/861 ,  A61P27/02 ,  A61K35/761 ,  A61K38/18 ,  A61K39/235

CPC分类号： C12N15/861 ,  A61K35/761 ,  A61K38/1833 ,  A61K39/235 ,  A61P27/02

摘要： One disadvantage of adeno-associated virus (AAV) is low gene expression efficiency due to delayed expression of the inserted gene. Provided is a vector plasmid for producing a vector genome to be inserted into an adeno-associated virus (AAV). The vector plasmid is provided with a vector genome cassette as a template of the vector genome, and the vector genome cassette is provided with: (1) an expression cassette including a nucleic acid molecule encoding a target gene and (b) a nucleic acid molecule encoding an expression regulatory region that allows expression of the target gene; and (2) two nucleic acid molecules positioned on both sides of the expression cassette and encoding inverted terminal repeats (ITRs). Also provided is a pharmaceutical composition containing the AVV vector, for treating or preventing an eye disease or disease associated with same. Further provided is a method for treating or preventing the eye disease or disease associated with same, comprising administering a therapeutically effective amount of the pharmaceutical composition for treating or preventing the eye disease or disease associated with same to a subject suffering from the eye disease or disease associated with same.

公开(公告)号：US20200329684A1

公开(公告)日：2020-10-22

申请号：US16846718

申请日：2020-04-13

申请人： Nippon Medical School Foundation ,  Teika Pharmaceutical Co., Ltd.

发明人： Takashi Okada ,  Tsutomu Igarashi ,  Asaka Shiozawa

IPC分类号： A01K67/027

摘要： Retinal detachment sometimes occurs during the aging process of retinal degeneration 6 model mouse (rd6 model mouse). The present invention provides a method for obtaining a retinal degeneration (rd) model mouse having a low risk of causing retinal detachment, including: acquiring tissue data about a retinal tissue in an rd model mouse by using a retinal examination means; determining whether or not the tissue data meets an evaluation criterion; and selecting the rd model mouse, the tissue data of which meets the evaluation criterion.