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Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-102 Gene Therapy in Sanfilippo Syndrome Type A

NEW YORK, NY, and CLEVELAND, OH--(Marketwired - October 18, 2016) - Abeona Therapeutics Inc. (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 for Abeona's lead gene therapy program ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death in children.

"Receiving European Union (EU) orphan drug designation is an important milestone that delivers significant commercial benefits to our company as we advance our innovative portfolio of gene therapy products," stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. "The benefits and incentives associated with these designations, including marketing exclusivity, are strategically important from a regulatory and commercial perspective and potentially value-creating for shareholders."

Abeona's MPS IIIA program, ABO-102, has previously been granted FDA Orphan Product Designation in the USA and received the Rare Pediatric Disease Designation as a pre-requisite part of the Priority Review Voucher (PRV) process.

About European Union (EU) Orphan Drug Designation: The European Commission grants orphan drug designation status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the European Union. The orphan drug designation provides Abeona with incentives and benefits in the EU, including reduced fees and protection from market competition once ABO-102 is approved for the treatment of MPS IIIA patients.

About ABO-102 (AAV-SGSH): ABO-102, the company's first-in-human, intravenously-administered AAV gene therapy, has been well tolerated through 30-day...