ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT1100
ReCode Therapeutics, a clinical-stage genetic medicines company specializing in tissue-specific delivery to advance mRNA and gene correction therapeutics, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RCT1100 for the treatment of primary ciliary dyskinesia (PCD). RCT1100 is an innovative investigational therapy aimed at treating PCD caused by pathogenic mutations in the DNAI1 gene. PCD is a rare, progressive genetic disorder characterized by the malfunction of cilia, which are microscopic hairlike structures lining the respiratory tract. This defect prevents the clearance of mucus from the airways, leading to the entrapment of bacteria and viruses. Consequently, patients suffer from chronic infections and impaired respiratory function, with a small percentage requiring lung transplants. Mutations in the DNAI1 gene are responsible for approximately seven percent of PCD cases.
ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT1100