Sio Gene Therapies jumps on early GM1 gangliosidosis results

Sio Gene Therapies (Nasdaq: SIOX) has reported positive six-month follow-up data from the low-dose cohort of the company’s dose escalation study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy for the treatment of GM1 gangliosidosis.

The encouraging results in this rare, progressive, and fatal pediatric disorder sent shares in Sio Gene upwards by 14% in Wednesday morning’s trading.

AXO-AAV-GM1 was able to stabilize disease progression and, in some cases, even improve it, to help these children retain the developmental milestones they achieved by the time of treatment.