CMM-affiliated investigators Zhenwei Song and Chengwen Li are part of a newly awarded four-year, $2.9 million collaborative R01 focused on improving the safety and effectiveness of gene therapies.
The multi-investigator team will study genetic and non-genetic factors that influence both therapeutic benefit and liver toxicity associated with AAV-based gene therapies. By strengthening understanding of how patients respond to these treatments, the project directly supports CMM’s mission to advance rigorous, translational research that moves gene therapies toward safer and more effective clinical applications.