Shire’s haemophilia A gene therapy wins Orphan status
Shire’s gene therapy candidate SHP654, an investigational factor VIII (FVIII) gene therapy for the treatment of haemophilia A, has been awarded Orphan Drug Designation by US regulators
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Tag: SHP654
Shire applies to test haemophilia A gene therapy in humans
Shire has submitted a request to the US Food and Drug Administration to test its investigational factor VIII (FVIII) gene therapy SHP654 in patients with the bleeding disorder haemophilia A.
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