Florian Eichler, MD, on Promising Efficacy of AXO-AAV-GM2 in GM2 Gangliosidosis

“We have a certain window within which we need to treat as soon as these patients are diagnosed and the genetics are clear, but before the disease has advanced too far, because at that point... it is likely these children will not benefit from the kind of gene replacement that we're performing here.”

Bithalamic, intra-cisterna magna (ICM) and intrathecal delivery of AXO-AAV-GM2 (Sio Gene Therapies) gene therapy has been found to be well-tolerated in all 5 participants with GM2 gangliosidosis dosed so far, with MRI evidence of accurate bithalamic targeting.

Updated data from a phase 1/2 trial (NCT04669535) were presented at the 2022 American Society of Gene and Cell Therapy Annual Meeting, May 16-19, 2022, in Washington DC, by Florian Eichler, MD, assistant professor of neurology, Harvard Medical School. The study is being conducted at Worcester General Hospital and Massachusetts General Hospital, where Eichler serves as the director of the leukodystrophy service.

CGTLive spoke with Eichler during the meeting to learn more about the phase 1/2 trial and its current progress with enrollment and dosing. He discussed promising enzyme activity and reduction of gangliosides.

REFERENCE

Eichelr F, Flotte T, Andonian H, et al. AXO-AAV-GM2 gene therapy for infantile- and juvenile-onset GM2 gangliosidosis: Preliminary results from an ongoing phase 1/2 trial. Presented at: ASGCT 25th Annual Meeting, May 16-19, 2022, Washington DC.