Title:
COMPOSITIONS AND METHODS FOR THE TREATMENT OF MYOTONIC DYSTROPHIES
Document Type and Number:
WIPO Patent Application WO/2024/006775
Kind Code:
A3
Abstract:
The present invention relates to adeno-associated virus (AAV) delivery of nucleic acids for treating myotonic dystrophy, e.g., myotonic dystrophy type 1 (DM1 ), in patients in need thereof, e.g., patients diagnosed with DM1 or displaying one or more symptoms of DM1, e.g., myotonia. The invention provides AAV products and methods of using the AAV in the treatment of myotonic dystrophy, e.g., DM1.
Inventors:
TAKEUCHI MIKIO (JP)
Application Number:
PCT/US2023/069191
Publication Date:
February 22, 2024
Filing Date:
June 27, 2023
Export Citation:
Assignee:
ASTELLAS GENE THERAPIES INC (US)
International Classes:
C12N15/864; A61K48/00; C07K14/47; C12N7/00
Domestic Patent References:
| WO2021087007A1 | 2021-05-06 |
Foreign References:
| US20210162073A1 | 2021-06-03 |
Attorney, Agent or Firm:
BELLIVEAU, Michael, J. et al. (US)
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