Axovant Gene Therapies (NASDAQ:AXGT) has dosed the first patient in a clinical study of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis.
GM1 gangliosidosis is a rare genetic disease where mutations in the GLB1 gene impair production of beta-galactosidase, causing lipids to accumulate in cells of the nervous system.
AXO-AAV-GM1 is designed to deliver a functional copy of the GLB1 gene to restore normal beta-galactosidase production.
“GM1 gangliosidosis is a devastating disease in young children, for which there are no currently approved treatment options,” Dr. Cynthia Tifft, the trial’s principal investigator, said in a statement.
“The development of a safe and effective gene therapy for these patients would be a welcome advancement in the field of pediatric lysosomal storage disorders affecting the brain,” she added.
Axovant expects to report initial data from this trial in the second half of 2019.
