Title:
COMPOSITIONS AND METHODS FOR TREATING MUSCLE ATROPHY AND MYOTONIC DYSTROPHY
Document Type and Number:
Japanese Patent JP2023011614
Kind Code:
A
Abstract:
To provide polynucleic acid molecules for modulating a gene associated with muscle atrophy (or an atrogene).SOLUTION: Disclosed is a polynucleic acid molecule conjugate comprising an antibody or its binding fragment bound to a polynucleic acid molecule that hybridizes to a target sequence of an atrogene where the polynucleic acid molecule comprises at least one 2' modified nucleotide, at least one modified internucleotide linkage or at least one inverted abasic moiety, where the polynucleic acid molecule conjugate mediates RNA interference to the atrogene thereby treating muscle atrophy or myotonic dystrophy of a subject.SELECTED DRAWING: Figure 19A
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Inventors:
Gel, Andrew John
Doparapudi, Venkata Ramana
Chew, David Si-ho
Cochrane, Michael Calamian
Hood, Michael
Dalimont, Beatrice Diana
Bourke, rob
Shi, Yunyu
Malerius, Glynn Erdogan
Malekova, Barbora
Doparapudi, Venkata Ramana
Chew, David Si-ho
Cochrane, Michael Calamian
Hood, Michael
Dalimont, Beatrice Diana
Bourke, rob
Shi, Yunyu
Malerius, Glynn Erdogan
Malekova, Barbora
Application Number:
JP2022162646A
Publication Date:
January 24, 2023
Filing Date:
October 07, 2022
Export Citation:
Assignee:
Avidity Biosciences, Inc.
International Classes:
C12N15/113; A61K31/7115; A61K31/712; A61K31/7125; A61K39/395; A61K47/68; A61P21/00; C07K16/28
Attorney, Agent or Firm:
Yoshihiro Kiyohara