Orchard Therapeutics (NASDAQ:ORTX) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for OTL-103, its ex vivoautologous hematopoietic stem cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS) developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
RMAT is akin to Breakthrough Therapy status for drugs/biologics. It provides for the accelerated review of the marketing application and more intensive guidance from the FDA on development.
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