COMPOSITIONS AND METHODS FOR MUSCULAR DISORDERS
PE0016642026A1Pending Publication Date: 2026-08-19KATE THERAPEUTICS INC
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Patent Information
- Application Number
- PE2026000678
- Authority / Receiving Office
- PE · PE
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-10-11
- Filing Date
- 2024-10-11
- Publication Date
- 2026-08-19
Abstract
The present invention provides nucleic acid molecules and methods that result in changes in DUX4 expression leading to the treatment of FSHD. The nucleic acid molecules of the invention comprise a miR scaffold and a guide miR sequence targeting a double homeobox 4 (DUX4) transcript. By targeting DUX4 transcripts, the nucleic acid molecules of the invention result in an appropriate expression profile for the DUX4 gene.
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