公开(公告)号：US12064460B2

公开(公告)日：2024-08-20

申请号：US16964142

申请日：2019-01-25

申请人： TOKUSHIMA UNIVERSITY ,  JICHI MEDICAL UNIVERSITY ,  GENE THERAPY RESEARCH INSTITUTION CO., LTD.

发明人： Kohji Itoh ,  Daisuke Tsuji ,  Shinichi Muramatsu ,  Katsuhito Asai

IPC分类号： A61K35/76 ,  A61P25/28 ,  C12N7/00

CPC分类号： A61K35/76 ,  A61P25/28 ,  C12N7/00 ,  C12N2750/14121 ,  C12N2750/14143 ,  C12N2750/14171

摘要： According to a conventional method for treatment of Sandhoff disease and Tay-Sachs disease comprising administering a modified β-subunit to a patient in the form of a protein, it is necessary that administration be performed frequently. This invention relates to a recombinant adeno-associated virus virion comprising: capsomere comprising a protein capable of forming a virus virion; and a polynucleotide packaged in the capsomere comprising a promoter sequence and nucleotide sequences operably linked to the promoter sequence encoding a first amino acid sequence derived from the amino acid sequence of the β-subunit of wild-type human β-hexosaminidase composed of amino acids 55 to 556 in the sequence as shown in SEQ ID NO: 28 by substitution of amino acids 312 to 318 with glycine, serine, glutamic acid, proline, serine, glycine, and threonine in that order and a second amino acid sequence, which is an amino acid sequence of a signal peptide linked to the N terminus of the first amino acid.

公开(公告)号：US20220364101A1

公开(公告)日：2022-11-17

申请号：US17623302

申请日：2020-07-03

申请人： Tokushima University

发明人： Kohji Itoh ,  Jun Tsukimoto

IPC分类号： C12N15/52 ,  A61P3/00 ,  C12N15/86 ,  C12N9/48

摘要： Provided are a modified-type neuraminidase, a gene encoding the modified-type neuraminidase, a combination of the modified-type neuraminidase and cathepsin A, a combination of the gene encoding the modified-type neuraminidase and a gene encoding cathepsin A, a vector including said genes, and a pharmaceutical composition containing same. The pharmaceutical composition can be used for the therapy of lysosomal storage disease.