On October 21st, Sio Gene Therapies Inc. made two major announcements featuring one of its lead gene therapy candidates. The New York-based company presented positive interim data for AXO-AAV-GM1, its AAV9-based gene therapy for GM1 gangliosidosis, at the ESGCT Virtual Congress 2021. On the same day, it also announced the FDA's Fast Track Designation granted for the drug.

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."