Dual Vector Gene Therapy Trial Offers Hope for Tay-Sachs and Sandhoff Diseases

Dual vector gene therapy trial offers hope for Tay-Sachs and Sandhoff diseases

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.

Dual-Vector rAAVrh8 Gene Therapy for GM2 Gangliosidosis: A Phase I/II Trial

Investigators initiated a Phase I/II, single-dose, dose-escalation of combined BiT, intra-cisterna magna and intrathecal infusion in children with Tay–Sachs and Sandhoff diseases [Nature Medicine] Full Article