Ractigen Therapeutics Receives FDA Orphan Drug Designation for RAG-21 to Treat ALS
Ractigen Therapeutics has announced that the FDA has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy for treating amyotrophic lateral sclerosis (ALS). RAG-21 specifically targets FUS-ALS, one of the most aggressive ALS subtypes, by leveraging RNA interference (RNAi) technology to reduce FUS protein levels. Preclinical studies have shown its ability to mitigate FUS cytoplasmic mis-localization and aggregation, addressing the root cause of motor neuron degeneration in FUS-ALS. This marks Ractigen’s second FDA ODD for ALS, following RAG-17 for SOD1-ALS.
The O...
