UMass Chan Researchers Report Positive Phase I/II Trial Data for GM2 Gangliosidosis AAV Gene Therapy

A Phase I/II clinical trial at UMass Chan Medical School has shown promising results for a dual vector AAV gene therapy for GM2 gangliosidosis, a rare genetic disorder that includes Tay-Sachs and Sandhoff diseases. The AAV gene therapy demonstrated biochemical correction of the disease with minimal adverse reactions in patients.

Key AAV Trial Findings

• Positive Efficacy: The study confirmed that the dual AAV vector gene therapy successfully addressed the genetic deficiencies of the disease. Researchers observed measurable improvements in biochemical markers, indicating the AAV therapy’s potential therapeutic effect.
• Favorable AAV Safety Profile: The AAV treatment was well-tolerated by participants, with minimal adverse reactions reported. This is a crucial finding for an early-stage AAV clinical trial and suggests a positive safety profile for the AAV vectors used.

These results represent an important step in advancing AAV gene therapy as a potential treatment for these rare and severe neurodegenerative conditions.