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- FDA approves Ultragenyx gene therapy and Regeneron ultra-rare disease drugThe Food and Drug Administration gave the green light to a gene‑therapy product from Ultragenyx, marking a new treatment option in the company’s pipeline. In a separate decision, Regeneron secured…
- 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…