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Abeona Therapeutics Receives Orphan Drug Designation in the European Union for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa

Abeona’s Fourth Gene Therapy Program to Receive EMA Orphan Designation

EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) has Demonstrated Promising Efficacy and Safety in Ongoing Phase 1/2 Clinical Trial

NEW YORK and CLEVELAND, March 08, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (ABEO), a leading 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 EB-101 gene therapy program for patients with recessive dystrophic epidermolysis bullosa (RDEB), a devastating, life-threatening genetic skin disorder that is characterized by skin blisters and erosions all over the body.

“EB-101 is Abeona’s fourth gene therapy program to be granted EMA Orphan Designation and it further builds on our commercial portfolio of clinical-stage gene therapies that have received FDA and EMA orphan drug designations, which is an important validation of the clinical translation of these treatments for severely underserved patient populations,” stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. “The orphan designation also provides 10 years of market exclusivity in the European Union from similar medicines for similar indications, an important value driver for Abeona as we expand our clinical programs in Europe.”

The ongoing phase 1/2 clinical trial with gene-corrected skin grafts has shown...