ABO-102, which has previously received rare pediatric disease designation, fast track designation, and orphan drug designation from the U.S. Food and Drug Administration (FDA), was the recipient of another acknowledgement this morning when it was granted regenerative medicine advanced therapy (RMAT) designation.
Abeona Therapeutics, Inc. announced the news about its adeno associated virus (AAV)-mediated gene therapy for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome).
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