Orchard secures RMAT designation for novel gene therapy
UK and USA-based clinical-stage biotech Orchard Therapeutics (Nasdaq: ORTX) has received a Regenerative Medicine Advanced Therapy (RMAT) designation for OTL-103, a gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS), from the US Food and Drug Administration.
WAS is a life-threatening inherited immune disorder characterized by autoimmunity, eczema, abnormal platelet number and function.
Chief regulatory officer Anne Dupraz-Poiseau said: “Securing RMAT designation for OTL-103 is an important step in expediting the product development and review of our planned biologics license application.”
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