source
- FDA grants approval to Ultragenyx's Genglycos gene therapy for ultra-rare glycogen storage disorderUltragenyx announced that the U.S. Food and Drug Administration has approved its gene therapy called Genglycos. The therapy targets an ultra-rare glycogen storage disorder that can lead to severe…
- Trump nominates Heidi Overton as FDA Commissioner as agency releases CGT Q&A guidanceThe U.S. Food and Drug Administration issued final guidance that answers common questions about developing cellular and gene therapy products. The document aims to clarify regulatory expectations f…