Ractigen Therapeutics’ RAG-18 Gains the US FDA’s Orphan Drug Designation to Treat DMD and BMD
Shots:
- The US FDA has granted orphan drug designation to the company’s RAG-18 for treating Duchenne Muscular Dystrophy (DMD) & Becker Muscular Dystrophy (BMD) caused by any mutation of the DMD gene
- RAG-18 is a saRNA candidate aimed at selectively stimulating UTRN gene expression in muscle cells through an RNA activation mechanism.
- Preclinical evidence suggests that RAG-18, which is administered via subcutaneous injection using Ractigen’s exclusive LiCO technology. Additionally, RAG-18 obtained RPDD from the US FDA in July of this year.
Ref: Ractigen Therapeutics | Image: Ractigen Therapeutics
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