ABEONA THERAPEUTICS RECEIVES ORPHAN DRUG DESIGNATION IN THE EUROPEAN UNION FOR ABO-101 GENE THERAPY IN SANFILIPPO SYNDROME TYPE B

NEW YORK and CLEVELAND, Jan 20 (Bernama-GLOBE NEWSWIRE) -- 

• ABO-101, Abeona’s third AAV gene therapy program to receive EMA Orphan Designation
• Clinical trials anticipated to begin enrolling in second quarter 2017
• Natural History Study in 25 patients has established efficacy outcome measures
• FDA previously granted Orphan Drug and Rare Pediatric Disease Designations   

Abeona Therapeutics Inc. (Nasdaq:ABEO), a clinical-stage biopharmaceutical company focused on developing gene therapies for life-threatening rare diseases, announced today that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation (EMA/OD/226/16) for Abeona’s gene therapy program ABO-101 for children impacted by Sanfilippo syndrome type B (MPS IIIB), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death in children.

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