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 … Continue reading FDA Grants Orphan Drug Designation for Ractigen’s RAG-21 in ALS; Intellia’s Nexiguran RMAT for ATTR Amyloidosis; FDA Approves Acoramidis for ATTR-CM; Jazz Wins FDA Approval for Ziihera in HER2-Positive Biliary Cancer; J&J Seeks FDA Approval for Subcutaneous TREMFYA in Colitis