ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Patent Information
- Application Number
- ARP20250100605
- Authority / Receiving Office
- AR · AR
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-08-28
- Filing Date
- 2025-03-05
- Publication Date
- 2026-09-16
Claims
The present invention provides novel gene therapy compositions for use in the treatment of Duchenne muscular dystrophy (DMD).
1. A recombinant nucleic acid comprising: a muscle-specific promoter; a sequence encoding microdystrophin; and at least one dorsal root ganglion (DRG) miRNA binding site.
44. A recombinant nucleic acid comprising a promoter, wherein the promoter comprises or consists of a nucleic acid sequence that is at least 85%, at least 86%, at least 87%, at least 88%, at least 89%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, or at least 99% identical to the nucleic acid sequence of any of SEQ ID No. 2402-2404.
47. A recombinant nucleic acid comprising a codon-optimized microdystrophin encoding sequence that is at least 85%, at least 86%, at least 87%, at least 88%, at least 89%, at least 90%, at least 91%, at least 92%, at least 93%, at least 94%, at least 95%, at least 96%, at least 97%, at least 98%, or at least 99% identical to the nucleic acid sequence of any of SEQ ID No. 2405-2407.
50. An adeno-associated virus (AAV) particle comprising the recombinant nucleic acid of any of claims 1-49 and a capsid protein.
64. A pharmaceutical composition comprising the recombinant nucleic acid of any of claims 1-49 or the adeno-associated virus (AAV) particle of any of claims 50-63, and a pharmaceutically acceptable carrier.