FDA grants orphan drug designation to RNA therapy for Charcot-Marie-Tooth disease type 1A
The FDA has granted orphan drug designation to DTx-1252, an investigational small interfering RNA therapeutic to treat Charcot-Marie-Tooth disease type 1A.According to a DTx Pharma press release, the novel therapy is part of the company’s Fatty Acid Ligand Conjugated OligoNucleotides (FALCON) platform, which uses fatty acids to improve biodistribution and cellular uptake of RNA-based