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# Ractigen Gets Orphan Status for Duchenne’s
- URL: https://www.fdaweb.com/ractigen-gets-orphan-status-for-duchennes/
- Published: 2024-08-27T12:00:00.000Z
- Updated: 2026-09-14T14:39:35.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5157662

FDA has granted Ractigen Therapeutics an orphan drug designation for RAG-18, a saRNA product candidate for treating Duchenne Muscular Dystrophy (DMD). The therapy is designed to specifically target and activate UTRN gene expression in muscle cells via the RNAa mechanism, according to the company. “The utrophin protein encoded by the UTRN gene is structurally and functionally similar to dystrophin, and its upregulation could potentially serve as a functional replacement for the missing dystrophin in DMD muscle cells, providing treatment for all DMD patients regardless of the specific mutation location,” it says.

The designation is based on preclinical data showing that RAG-18, delivered through subcutaneous injection utilizing Ractigen’s proprietary LiCO (lipid-conjugated oligonucleotide) technology, has “effectively mitigated muscle damage, demonstrating significant potential in treating DMD patients,” the company says.

RNAa is described as a clinically validated platform technology that uses saRNAs to target gene regulatory domains to activate gene expression and restore therapeutic protein levels.