sgRNA for Utrophin Activation to Treat Duchenne Muscular Dystrophy
Summary
Kunming University of Science and Technology published patent application US20260108628A1 covering an sgRNA capable of activating utrophin transcription and translation in human and mouse systems, as a therapeutic approach for Duchenne muscular dystrophy. The application, filed January 18, 2024, contains DNA sequences represented by SEQ ID NOS: 1-20 and 21-39. The invention provides a gene therapy option targeting endogenous utrophin upregulation as an alternative to dystrophin replacement.
“By means of sgRNAs targeting endogenous utrophin, the sgRNAs can efficiently activate the transcription and translation expression of human and mouse utrophin, thus providing a therapeutic option for the treatment of Duchenne muscular dystrophy.”
About this source
USPTO classification A61K covers pharmaceutical preparations: formulations, dosage forms, combination therapies, delivery systems, and the vehicles that carry active ingredients. Every newly published application in A61K lands in this feed, around 245 a month. Applications publish 18 months after filing, so this feed reveals what pharma formulators at major sponsors and generics were working on in the prior year and a half. Watch this if you compete in drug formulation, file freedom-to-operate analyses, scout competitive drug products before clinical readouts, or follow novel delivery platforms like nanoparticles, lipid carriers, and long-acting injectables.
What changed
USPTO published patent application US20260108628A1 for Kunming University of Science and Technology, covering sgRNA compositions that activate utrophin gene expression as a treatment for Duchenne muscular dystrophy. The application discloses 39 distinct DNA sequences targeting both human and mouse utrophin loci, enabling cross-species therapeutic development. Patent rights have not yet been granted; this publication establishes a priority date of January 18, 2024 and creates prior art for subsequent applications in the field.
For pharmaceutical and biotechnology entities engaged in genetic therapy development for muscular dystrophies, this application signals competitive activity in utrophin activation approaches and may affect freedom-to-operate considerations for related gene therapy programs.
Archived snapshot
Apr 24, 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.
SGRNA FOR UTROPHIN ACTIVATION TO TREAT DUCHENNE MUSCULAR DYSTROPHY AND USE THEREOF
Application US20260108628A1 Kind: A1 Apr 23, 2026
Assignee
KUNMING UNIVERSITY OF SCIENCE AND TECHNOLOGY
Inventors
Yongchang CHEN, Ruo WU, Peng LI, Wenting GUO
Abstract
An sgRNA for treating Duchenne muscular dystrophy by the activation of utrophin, and a use are provided. The sgRNA is an sgRNA capable of activating the transcription and translation expression of human or mouse utrophin; and the DNA sequence of the sgRNA is as represented by SEQ ID NOS: 1-20 or SEQ ID NOS: 21-39. By means of sgRNAs targeting endogenous utrophin, the sgRNAs can efficiently activate the transcription and translation expression of human and mouse utrophin, thus providing a therapeutic option for the treatment of Duchenne muscular dystrophy.
CPC Classifications
A61K 48/005 A61K 31/7105 C12N 15/113 C12N 15/8645 C12N 2750/14143 C12N 2800/107
Filing Date
2024-01-18
Application No.
19144537
Related changes
Get daily alerts for USPTO Patent Applications - Pharma (A61K)
Daily digest delivered to your inbox.
Free. Unsubscribe anytime.
Source
About this page
Every important government, regulator, and court update from around the world. One place. Real-time. Free. Our mission
Source document text, dates, docket IDs, and authority are extracted directly from USPTO.
The summary, classification, recommended actions, deadlines, and penalty information are AI-generated from the original text and may contain errors. Always verify against the source document.
Classification
Who this affects
Taxonomy
Browse Categories
Get alerts for this source
We'll email you when USPTO Patent Applications - Pharma (A61K) publishes new changes.
Subscribed!
Optional. Filters your digest to exactly the updates that matter to you.