US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder

Reuters

2025/07/12

July 11 (Reuters) - The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's RARE.O experimental gene therapy to treat a rare genetic disorder, the company said on Friday.

(Reporting by Kamal Choudhury in Bengaluru; Editing by Shailesh Kuber)

((Kamal.Choudhury@thomsonreuters.com;))

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