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Sio Gene Shares Gain As Genetic Nerve Cell Disorder Gene Therapy Trial Shows Encouraging Action

Benzinga
Benzinga
 2021-10-21
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  • Sio Gene Therapies Inc (NASDAQ: SIOX) has announced interim data from its Phase 1/2 study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for GM1 gangliosidosis.
  • GM1 gangliosidosis is a genetic disorder that progressively destroys nerve cells in the brain and spinal cord.
  • Data were shared at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress.
  • Data showed that the gene therapy was generally well-tolerated at both low and high doses, with most adverse events considered mild to moderate.
  • Data demonstrated a dose-dependent improvement in key biomarkers of disease activity.
  • Serum β-galactosidase activity achieved a normal range, increasing by 12x and 17x pre-treatment levels, respectively, in both patients in the high-dose cohort at six months.
  • Levels of CSF GM1 ganglioside were normalized in both patients in the high-dose cohort with 42% and 72% reductions, respectively, at six months.
  • Total brain volume was maintained within ± 5% in all five patients.
  • Price Action: SIOX shares are up 13.90% at $2.45 during the premarket session on the last check Thursday.

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