Recombinant AAV Vectors for Treating Muscular Dystrophy
Summary
USPTO published patent application US20260097132A1 assigned to Sarepta Therapeutics, Inc. covering recombinant adeno-associated virus (rAAV) vectors for expressing human micro-dystrophin gene to treat muscular dystrophy, including Duchenne Muscular Dystrophy. The application includes methods for genotyping DMD gene to determine rAAV gene therapy contraindications. Filing date was September 22, 2023.
What changed
USPTO published Sarepta Therapeutics' patent application covering recombinant AAV vectors for expressing micro-dystrophin gene to treat muscular dystrophy, including Duchenne Muscular Dystrophy. The application includes claims to rAAV compositions, methods of treatment, and genotyping methods to identify contraindications for gene therapy.
Affected parties including biotech companies, pharmaceutical researchers, and gene therapy developers should monitor this patent's prosecution for potential freedom-to-operate implications in the DMD gene therapy space. Patent publication alerts allow competitors to assess prior art and potential licensing opportunities.
What to do next
- Monitor for updates on patent prosecution status
Archived snapshot
Apr 11, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY
Application US20260097132A1 Kind: A1 Apr 09, 2026
Assignee
Sarepta Therapeutics, Inc.
Inventors
Louise RODINO-KLAPAC, Ida H. MOELLER, Stefanie E. MASON
Abstract
The present disclosure provides gene therapy vectors, such as recombinant adeno-associated virus (rAAV) for expressing a human micro-dystrophin gene. The present disclosure also provides compositions and methods of using these rAAV to treat muscular dystrophy, such as, e.g., Duchenne Muscular Dystrophy. The present disclosure also provides genotyping a subject's DMD gene to determine if rAAV gene therapy should be contraindicated.
CPC Classifications
A61K 48/005 A61K 48/0075 A61K 48/0083 A61P 21/00 C07K 14/4708 C12N 5/0018 C12N 5/0686 C12N 7/00 C12N 15/86 C12Q 1/6883 A61K 38/00 C12N 2500/84 C12N 2527/00 C12N 2710/10322 C12N 2750/14143 C12N 2750/14152 C12N 2800/40 C12Q 2600/106
Filing Date
2023-09-22
Application No.
19114103
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