Title:
TISSUE SPECIFIC PEPTIDE CONJUGATES AND METHODS
Document Type and Number:
Japanese Patent JP2018171066
Kind Code:
A
Abstract:
PROBLEM TO BE SOLVED: To provide cell-penetrating peptides useful for targeting a therapeutic compound to a selected mammalian tissue, methods for their identification, methods of forming conjugate compounds containing such peptides, and conjugates formed thereby.SOLUTION: The present invention relates to a peptide-antisense conjugate for treating a given disease condition, comprising a phosphorodiamidate morpholino antisense oligomer directed against a gene whose expression is associated with the given disease condition in a specific target tissue, and a cell-penetrating peptide that comprises 8 to 20 amino acid residues. The present invention also relates to a peptide-antisense conjugate, in which the cell-penetrating peptide comprises arginine and/or β-alanine and a linear amino acid.SELECTED DRAWING: Figure 1B
Inventors:
HONG M MOULTON
PATRICK L IVERSEN
PATRICK L IVERSEN
Application Number:
JP2018121949A
Publication Date:
November 08, 2018
Filing Date:
June 27, 2018
Export Citation:
Assignee:
SAREPTA THERAPEUTICS INC
International Classes:
C12N15/113; A61K31/7088; A61K47/64; A61K48/00; A61P1/16; A61P9/00; A61P19/02; A61P21/04; A61P29/00; A61P31/12; A61P31/16; A61P37/02; C07K7/06; C07K7/08; G01N33/15; G01N33/50
Attorney, Agent or Firm:
Hatsushi Shimizu
Masao Haruna
Hirotaka Yamaguchi
Toshi Gobe
Ryuichi Inoue
Toshimitsu Sato
Koichi Niimi
Tomohiko Kobayashi
Masato Ozeki
Kazuya Kawamoto
Masao Haruna
Hirotaka Yamaguchi
Toshi Gobe
Ryuichi Inoue
Toshimitsu Sato
Koichi Niimi
Tomohiko Kobayashi
Masato Ozeki
Kazuya Kawamoto