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Sio Gene Therapies Inc. (NASDAQ: SIOX) announced a receipt of $11.6 million from the sale of Arvelle Therapeutics to Angelini Pharma. The company anticipates an additional $4.8 million by mid-2021 upon European approval of cenobamate, totaling over $16.4 million in net proceeds this year. This boost supports Sio's commitment to advancing gene therapies for neurodegenerative diseases, including GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases. The company emphasizes its goal of developing solutions for highly prevalent conditions such as Parkinson's disease.
Sio Gene Therapies (NASDAQ: SIOX) has announced the dosing of the first patient in a Phase 1/2 trial for AXO-AAV-GM2, a gene therapy targeting GM2 gangliosidosis, including Tay-Sachs disease. This marks the first potentially disease-modifying treatment for this condition. The trial aims to evaluate the safety and efficacy of the therapy, which has received Orphan Drug and Rare Pediatric Disease Designations from the FDA. Through partnerships and collaborative research, Sio continues patient enrollment and screening, highlighting a milestone in addressing the urgent need for effective treatments for this rare disorder.
Sio Gene Therapies has agreed to sell its stake in Arvelle Therapeutics to Angelini Pharma for up to $960 million. The transaction, closing in Q1 2021, will provide Sio with $11.8 million upfront and up to $8.2 million in milestone payments, totaling $20 million. This non-dilutive capital will extend Sio's cash runway into 2022, supporting their clinical-stage programs. The sale represents a substantial gain without incurring income tax liabilities, reinforcing Sio's commitment to advancing gene therapies for neurodegenerative diseases.
Sio Gene Therapies Inc. (SIOX) reported promising six-month follow-up results from its Phase 1/2 study of AXO-AAV-GM1, a gene therapy for GM1 gangliosidosis. The low-dose cohort (1.5x1013 vg/kg) involved five patients, demonstrating a favorable safety profile with no serious adverse events related to the therapy. At six months, serum beta-galactosidase enzyme activity was restored to an average of 38% of normal levels, with all patients showing clinical stability. The company is progressing to a high-dose cohort, further supporting the therapy's potential to treat this severe condition.