Sio Gene Therapies Gets FDA Fast-Track Designation for AXO-AAV-GM1 in GM1 Gangliosidosis
21 Ottobre 2021 - 2:56PM
Dow Jones News
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 colin.kellaher@wsj.com
(END) Dow Jones Newswires
October 21, 2021 08:41 ET (12:41 GMT)
Copyright (c) 2021 Dow Jones & Company, Inc.
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