Exegenesis Bio, a leading genetic medicines company, unveiled encouraging clinical efficacy and safety results for their EXG001-307 gene therapy in SMA Type 1 patients at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.
EXG001-307 is a novel recombinant adeno-associated virus (rAAV) gene therapy designed to address the genetic defect responsible for SMA. Nine patients treated with EXG001-307 exhibited significant improvements in motor function, including increased head control and the ability to sit independently within three months of dosing.
Notably, EXG001-307 demonstrated a superior safety profile compared to existing gene therapies, with reduced off-target expression in liver and heart tissue. This unique AAV design, featuring a pro-NS promoter, enhances target gene expression in the spinal cord while minimizing risks in other organs.
Based on the promising data, Exegenesis Bio plans to file an Investigational New Drug (IND) application for EXG001-307 in SMA Type 1 in the United States in the fourth quarter of 2024. The company is also considering an accelerated development pathway for SMA Type 2/3 patients.
SMA is a rare genetic disorder that affects the motor neurons, leading to muscle weakness, atrophy, and ultimately respiratory failure. Type 1, the most severe form, affects infants and has a high mortality rate. EXG001-307 represents a promising therapeutic option with the potential to significantly improve outcomes for these young patients.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.