BioTuesdays

FDA grants Sio Gene fast track for gene therapy for GM2 gangliosidosis

SioGene-Therapies

Sio Gene Therapies (NASDAQ:SIOX) received fast track designation for AXO-AAV-GM2, an investigational gene therapy for the treatment of early infantile, late infantile, and juvenile-onset Tay-Sachs and Sandhoff disease, two categories of GM2 gangliosidosis, a rare genetic disorder that progressively destroys nerve cells in the brain and spinal cord.

In a statement, Pavan Cheruvu, M.D., Sio Gene’s CEO, said fast track designation complements the previously granted rare pediatric disease and orphan drug designations for AXO-AAV-GM2, along with similar designations for AXO-AAV-GM1 for GM1 gangliosidosis, 

“We look forward to working closely with the FDA as we continue enrollment in our ongoing, registration-enabling trial in Tay-Sachs and Sandhoff diseases to bring AXO-AAV-GM2 to patients and families,” Dr. Cheruvu said.

The current Phase 1/2 study is an open-label, two-stage clinical trial designed to evaluate safety and dose-escalation (Stage 1) and safety and efficacy (Stage 2) of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal cord of pediatric participants with both infantile and juvenile GM2 gangliosidosis.

Sign up for the BioTuesdays weekly newsletter

Get the latest news on the healthcare industry’s corporate and clinical developments, executive moves, and market updates. Every Tuesday, in your inbox.