Cole K. Zheng

Twelve-Month Outcomes of Intrathecal Vesemnogene Lantuparvovec for Spinal Muscular Atrophy in Children Younger than 24 Months in Low- and Middle- Income Countries

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.

miniVec: A miniaturized plasmid backbone supporting antibiotic-free and additive-free fermentation with enhanced yield and functionality

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

Safety and Efficacy of Vesemnogene Lantuparvovec, an AAV-Based Gene Therapy in Young Children Under 24 Months with Spinal Muscular Atrophy in Low- and-Middle- Income Countries. Second Interim Report (August 2025).

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.