Title:
COMPOSITIONS AND METHODS OF TREATING HUNTINGTON'S DISEASE
Document Type and Number:
Japanese Patent JP2023093461
Kind Code:
A
Abstract:
To provide adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).SOLUTION: An AAV viral genome comprises, in order: a) a 5' inverted terminal repeat (ITR) sequence region, having a specific basic sequence, b) an enhancer sequence region, having a specific basic sequence, c) a promoter sequence region, having a specific basic sequence, d) a first intron sequence region, having a specific basic sequence, e) a modulatory polynucleotide sequence region, having a specific basic sequence, f) a polyA signal sequence region, having a specific basic sequence, and g) a 3' ITR sequence region, having a specific basic sequence.SELECTED DRAWING: None
Inventors:
DINAH WEN-YEE SAH
CHEN FENG
ZHOU PENGCHENG
WANG XIN
SHU YANQUAN
HOU JINZHAO
CHEN FENG
ZHOU PENGCHENG
WANG XIN
SHU YANQUAN
HOU JINZHAO
Application Number:
JP2023049305A
Publication Date:
July 04, 2023
Filing Date:
March 27, 2023
Export Citation:
Assignee:
VOYAGER THERAPEUTICS INC
International Classes:
C12N15/35; A61K35/76; A61K48/00; A61P25/14; C12N7/01; C12N15/113
Attorney, Agent or Firm:
Makoto Onda
Hironobu Onda
Atsushi Honda
Miki Nakamura
Hironobu Onda
Atsushi Honda
Miki Nakamura