Jason Z. Ye

This study evaluates the 12-month outcomes of vesemnogene lantuparvovec, an intrathecal gene therapy for SMA in children under 24 months in LMICs, showing safety and motor milestone improvements.

MuteFree eliminates ITR mutations in AAV transfer plasmids, achieving 0% mutation rates and resolving production issues in GMP manufacturing.

miniVec is a new plasmid backbone that eliminates antibiotic resistance genes and improves plasmid yield and functionality for clinical applications.

This paper reports on a phase 1 trial of vesemnogene lantuparvovec, an affordable gene therapy for SMA, showing safety and improved motor milestones in young children.