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November 20, 2024

FDA grants ODD to Ractigen Therapeutics’ RAG-21 for ALS treatment

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Ractigen Therapeutics' new therapy, RAG-21, targeting the fused in sarcoma (FUS) gene, for amyotrophic lateral sclerosis (ALS) treatment.

The therapy is designed to combat one of the ALS subtypes, FUS-ALS by reducing FUS protein. Credit:  JOSHUA COLEMAN on Unsplash.