Sio Gene Therapies Gets FDA Fast-Track Designation for AXO-AAV-GM1 in GM1 Gangliosidosis

By Colin Kellaher

Sio Gene Therapies Inc. on Thursday said the U.S. Food and Drug Administration granted fast-track designation to AXO-AAV-GM1 in the genetic disorder GM1 gangliosidosis.

The New York clinical-stage biopharmaceutical company said the designation covers the gene-therapy candidate for the treatment of Type I, or early infantile-onset, and Type II, or late infantile-onset and juvenile-onset, GM1 gangliosidosis.

The FDA's fast-track program is designed to facilitate the development and expedite the review of treatments for serious or potentially life-threatening illnesses with high unmet medical needs.

Sio said there are currently no FDA-approved treatment options for GM1 gangliosidosis, a progressive and fatal pediatric lysosomal storage disorder.

The company earlier Thursday reported positive interim data from its ongoing Phase 1/2 study of AXO-AAV-GM1 in GM1 gangliosidosis. Sio said it saw dose-dependent responses in two key biomarkers, including normalization of both biomarkers in the study's high-dose cohort.

Shares of Sio, which closed Wednesday at $2.15, rose more than 12% in premarket trading Thursday.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

10-21-21 0841ET