Abeona Stock Moves Higher After MPS IIIA Gene Therapy Shows Increase In Brain Volume

  • Abeona Therapeutics Inc ABEO announced magnetic resonance imaging (MRI) data from the Phase 1/2 Transpher A study of ABO-102 gene therapy in Sanfilippo Syndrome Type A (MPS IIIA).
  • Data indicated that ABO-102 increased grey matter, corpus callosum, and amygdala volumes in the brain in three young patients at 24 months as compared to afflicted patients without treatment. 
  • The new data were 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 a long-term cognitive and physical disability. 
  • The Transpher A study's 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, urine, and liver volume.
  • Price Action: ABEO shares are up 5.73% at $1.39 during the market session on the last check Monday.
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