Abeona Therapeutics Announces New MRI Data Showing Increased Brain Volume in Young Patients with Sanfilippo Syndrome Type A (MPS IIIA) After Treatment with ABO-102 Gene Therapy
NEW YORK and CLEVELAND, July 26, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene therapy, today announced magnetic resonance imaging (MRI) data from the Phase 1/2 Transpher A clinical study indicating that ABO-102 increased grey matter, corpus callosum and amygdala volumes in the brain in three young patients with Sanfilippo Syndrome Type A (MPS IIIA) at 24 months as compared to afflicted patients without treatment. The new data was presented during an oral presentation at the 16th International Symposium on MPS and Related Diseases.
“Brain volume loss is characteristic in children with MPS IIIA and is associated with long-term cognitive and physical disability. Specifically, grey matter is important for cognitive development, corpus callosum for motor function, and amygdala for fear learning as well as social/emotional development,” said Vishwas Seshadri, Ph.D., M.B.A., Head of Research & Clinical Development of Abeona. “The new MRI data shows the potential of ABO-102 to increase brain grey matter, corpus callosum and amygdala volumes and is consistent with previously reported results of preservation of neurocognitive development in these three young patients in the Transpher A study.”
The Transpher A study primary endpoints are neurodevelopment and safety. Secondary endpoints include brain volume, behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and liver volume.
About the Transpher A Study
About Sanfilippo Syndrome Type A (MPS IIIA)
About Abeona Therapeutics
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