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Abeona Therapeutics Receives European Medicines Agency PRIME Designation for ABO-102 Gene Therapy in MPS IIIA
PRIME is sixth regulatory designation for the ABO-102 clinical program NEW YORK and CLEVELAND, Dec. 20, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc.
About this update from Abeona Therapeutics Inc.
[{"type":"text","content":"PRIME is sixth regulatory designation for the ABO-102 clinical program\nNEW YORK and CLEVELAND, Dec. 20, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to the Company’s ABO-102 program studying its adeno-associated virus 9 (AAV9) gene therapy for Sanfilippo syndrome type A (MPS IIIA). The PRIME designation is based on nonclinical data and clinical data from the Transpher A Study, a global Phase 1/2 clinical trial evaluating a single-dose of ABO-102 for the treatment of children with MPS IIIA.\n “EMA’s PRIME designation for the ABO-102 program recognizes the urgent need for a treatment option for children suffering from MPS IIIA, and underscores the potential of ABO-102 to modify the course of this devastating lysosomal storage disease,” said João Siffert, M.D., Chief Executive Officer. The Transpher A Study is enrolling patients at sites in the U.S., Spain, and Australia. Additional information about the trial is available at AbeonaTrials.com and ClinicalTrials.gov (NCT02716246). The PRIME initiative provides access to enhanced support for the development of medicines targeting an unmet medical need. The designation affords sponsors with enhanced interaction and early dialogue regarding promising medicines, as well as the possibility of accelerated assessment of medicines applications. PRIME is intended to optimize development plans and speed up evaluation so these medicines can help patients to benefit as early as possible from therapies that may significantly improve their quality of life. About ABO-102ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using an AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds R...