FDA Orphan Drug Designation Granted to Hemophilia Gene Therapy; Clinical Studies to Begin Soon – Rare Disease Report (blog)
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FDA Orphan Drug Designation Granted to Hemophilia Gene Therapy; Clinical Studies to Begin Soon
Rare Disease Report (blog) The U.S. Food and Drug Administration (FDA) has granted Shire with orphan drug designation for its gene therapy candidate SHP654, intended to treat hemophilia A. The drug includes technology acquired from Chatham Therapeutics, LLC, a spin-out of … Shire Gets Orphan Drug Designation For Gene Therapy Candidate SHP654 |
