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Dosing Underway In Sio Gene's Early-Stage Trial For GM2 Gangliosidosis Gene Therapy

  • Sio Gene Therapies Inc (NASDAQ: SIOX) has dosed the first patient in Phase 1/2 trial evaluating AXO-AAV-GM2 investigational gene therapy to treat GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease, a rare genetic disorder that progressively destroys nerve cells in the brain and spinal cord.
  • The two-stage trial was designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal cord of pediatric participants with both infantile and juvenile GM2 gangliosidosis. Patient identification, screening, and enrollment in Stage 1 of the study are expected to continue throughout 2021.
  • AXO-AAV-GM2 has been granted Orphan Drug and Rare Pediatric Disease status by the FDA.
  • Price Action: SIOX shares gained 3.27% at $2.86 on the last check Wednesday.

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Posted-In: gene therapy Phase 1 TrialBiotech News Penny Stocks FDA General

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