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Duchenne Muscular Dystrophy Treatment, University of Pennsylvania

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Summary

The USPTO published patent application US20260109998A1 by The Trustees of the University of Pennsylvania, covering triple-splice mutants of dystrophin or utrophin, viral vectors encoding them, and compositions for treating Duchenne Muscular Dystrophy in human patients. Inventor Hansell Stedman filed the application on September 15, 2025, with CPC classifications C12N 15/86 and C07K 14/4708 indicating biotech/pharmaceutical subject matter. The application is published under Application No. 19328483.

“Described herein are triple-splice mutants of dystrophin or utrophin and methods of use thereof for treating Duchenne Muscular Dystrophy.”

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About this source

GovPing monitors USPTO Patent Applications - Biotech (C12N) for new healthcare & life sciences regulatory changes. Every update since tracking began is archived, classified, and available as free RSS or email alerts — 199 changes logged to date.

What changed

The USPTO has published a new patent application from The Trustees of the University of Pennsylvania covering compositions and methods for treating Duchenne Muscular Dystrophy. The application discloses triple-splice mutants of dystrophin or utrophin, viral vectors comprising nucleic acids encoding these mutants under regulatory element control, and formulated compositions for patient delivery.

Competitors in the DMD therapeutics space should monitor the claims scope and prosecution history of this application to assess potential freedom-to-operate implications. No compliance obligations arise for third parties at this application stage; however, if and when the patent grants, it may affect competitive dynamics in gene therapy approaches to muscular dystrophy treatment.

Archived snapshot

Apr 23, 2026

GovPing 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.

← USPTO Patent Applications

COMPOSITIONS AND METHODS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY

Application US20260109998A1 Kind: A1 Apr 23, 2026

Assignee

The Trustees of the University of Pennsylvania

Inventors

Hansell Stedman

Abstract

Described herein are triple-splice mutants of dystrophin or utrophin and methods of use thereof for treating Duchenne Muscular Dystrophy. Also provided are viral vectors which comprise a nucleic acid encoding a triple-splice mutant dystrophin or utrophin under the control of regulatory elements direct expression thereof. Compositions are also provided which contain such viral vectors formulated for delivery to a human patient.

CPC Classifications

C12N 15/86 C07K 14/4708 C12N 2750/14121 C12N 2750/14122

Filing Date

2025-09-15

Application No.

19328483

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Classification

Agency
USPTO
Published
April 23rd, 2026
Instrument
Notice
Branch
Executive
Legal weight
Binding
Stage
Final
Change scope
Minor
Document ID
US20260109998A1

Who this affects

Applies to
Pharmaceutical companies Biotechnology companies
Industry sector
3254 Pharmaceutical Manufacturing 3254.1 Biotechnology
Activity scope
Patent application Biotechnology research
Geographic scope
United States US

Taxonomy

Primary area
Intellectual Property
Operational domain
Legal
Topics
Pharmaceuticals Healthcare

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