Sio Gene Therapies Touts Positive Safety and Biomarker Data for GM1 Gangliosidosis Candidate
GM1 gangliosidosis is a rare inherited disease with no FDA-approved treatments currently. Safety is a big concern in the field of AAV gene therapy and several promising endeavors have been shut down recently due to adverse events. However, AXO-AAV-GM1 has shown promising safety and efficacy in both the low-dose and high-dose cohorts.
"The results from the GM1 gangliosidosis program is one of the light bulb moments for me and I think this gene therapy could really make a difference," Pavan Cheruvu told GeneOnline. "With these important data in hand, we anticipate a meeting with the FDA in the first half of 2022 to discuss the next steps for clinical development for AXO-AAV-GM1."