Sio Gene Therapies (NASDAQ: SIOX) stock was up by 20% since April 20 without any direct positive news from the company during this period. However, the company is focusing on the development of the drugs related to GM1 gangliosidosis, Tay-Sachs/Sandhoff disease, and Parkinson’s disease. The company will disclose the clinical research data about these drugs at the 24th Annual Meeting of the American Society of Gene & Cell Therapy to be held next week, which may bring some good news for investors.
AXO-AAV-GM1 is currently the most advanced GM1 gangliosidosis therapy from the company, and it has the potential to be the first approved treatment for this devastating disease, which currently in clinical Phase 1/2 stage. The FDA has previously granted Orphan Drug Designation and Rare Pediatric Disease Designation for AXO-AAV-GM1 for the treatment of GM1 gangliosidosis.
AXO-Lenti-PD is the only investigational gene therapy for Parkinson’s disease from the company. The company's initial clinical data showed that a single dose of AXO-Lenti-PD “turns back the clock” for patients by improving motor function and activities of daily living, and now, it's in clinical Phase 2 stage.
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