Axovant doses first patient in gangliosidosis gene therapy trial


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.

Michelle Carr