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Ractigen Therapeutics’ RAG-18 Gains the US FDA’s Orphan Drug Designation to Treat DMD and BMD

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Ractigen Therapeutics

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

Related News:- Amylyx Pharmaceuticals’ AMX0035 Gains the EC’s Orphan Drug Designation to Treat Wolfram Syndrome

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Dipanshu Dixit

A passionate content writer with expertise in delivering high-quality and engaging content, Dipanshu is a keen reader and a versatile writer. Dipanshu dedicatedly covers news ranging from biopharma, life sciences, biotech, and MedTech to diagnostics and animal health companies. He can be contacted at connect@pharmashots.com

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