NEW YORK and CLEVELAND, Jan 20 (Bernama-GLOBE NEWSWIRE) --
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. “This designation builds on our commercial portfolio of AAV gene therapies that have received FDA and EMA orphan drug designations, which is an important validation of the scientific and clinical translation of these products for severely underserved patient populations,” stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. “Accomplishing the designation would not have been possible without the contributions of Nationwide Children’s Hospital researchers Drs. Doug McCarty and Haiyan Fu, the Stop Sanfilippo Fundacion, Fundacion Sanfilippo B, Red Sanfilippo Fundacion, the Sanfilippo Children’s Research Foundation, Ben’s Dream, the Sanfilippo Medical Research Foundation, Team Sanfilippo and the National MPS Society USA.” mrem.bernama.com/viewsm.php?idm=28311
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