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- 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…
- Ultragenyx secures first gene therapy approval for GSDIa as Sanfilippo asset decision pendingUltragenyx received accelerated FDA approval for its gene therapy DTX401, marketed as Genglycos, to treat glycogen storage disease type Ia (GSDIa). The approval comes as the company awaits a secon…