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

Video

The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.

“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.
Recent Videos
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital
Mark Hamilton, MD, PhD, a hematology-oncology and bone marrow transplant (BMT) cell therapy fellow at Stanford University
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Related Content
© 2025 MJH Life Sciences

All rights reserved.