Abeona Therapeutics Announces Initial European Regulatory Approvals for Phase 1/2 Gene Therapy Clinical Studies; Sanfilippo Syndromes Type A (MPS IIIA) and Type B (MPS IIIB)

Abeona Therapeutics, Inc.

, a biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, today announced the Interministerial Council of Genetically Modified Organisms has approved the Genetically Modified Organism (GMO) Voluntary Release regulatory filings for both Phase 1/2 Gene Therapy Clinical Studies to treat patients with AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH) for patients with Sanfilippo syndrome type A (MPS IIIA) or type B (MPS IIIB). Additionally, the Comite Etico De Investigacion Clinica de Euskadi (CEIC-E) has approved the ethical committee regulatory filings for both ABO-101 and ABO-102. The Company plans to file CTAs for both programs shortly for the upcoming clinical studies to be conducted at Cruces University Hospital (Bilbao, Spain). "The GMO and Ethical committee approvals -- which are similar to the Recombinant DNA Advisory Committee (RAC) and hospital Institutional Review Board processes in the United States -- support our planned path to clinical trials in the EU for the treatment of Sanfilippo syndromes type A and B. Pre-clinical studies have demonstrated that a single intravenous injection of ABO-101 or ABO-102 restored the enzyme activity in the cerebral spinal fluid and serum, and also corrected the lysosomal storage pathology throughout the CNS and body," noted Tim Miller, PhD, President & CEO. "We look forward to advancing these potentially life-changing therapies into global clinical trials and grateful for the support and guidance from regulators, clinicians, and patient community." "Sanfilippo syndromes type A and B are devastating lysosomal storage diseases that affect children around the world. These gene therapies, delivered as a single, non-invasive intravenous injection, offer a new and promising treatment paradigm for patients with this relentless disease," commented Luis Aldámiz-Echevarría, M.D., PhD., Principal Investigator of the clinical trials and Associate Professor in the Faculty of Medicine at the University of the Basque Country (Spain), Paediatrician in the Department of Paediatrics at Cruces University Hospital (Spain) and Principal Investigator of the Metabolic Inherited Disorders Group and the Metabolomics and Proteomics Platform at BioCruces Health Research Institute (Spain). Sanfilippo syndromes (or mucopolysaccharidosis (MPS) type III) are a group of four inherited genetic diseases each caused by a single gene defect, described as type A, B, C or D, which cause enzyme deficiencies that result in the abnormal accumulation of glycosaminoglycans (sugars) in body tissues. MPS III is a lysosomal storage disease, a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. The incidence of MPS III (all four types combined) is estimated to be 1 in 70,000 births. Mucopolysaccharides are long chains of sugar molecule used in the building of connective tissues in the body. There is a continuous process in the body of replacing used materials and breaking them down for disposal. Children with MPS III are missing an enzyme which is essential in breaking down the used mucopolysaccharides called heparan sulfate. The partially broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. Babies may show little sign of the disease, but as more and more cells become damaged, symptoms start to appear. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. Importantly, there is no cure for MPS III and treatments are largely supportive care.