Gene transfer is an investigational approach that aims to significantly slow or even halt the progression of rare genetic diseases by delivering a replacement gene throughout the body.
Scientists have been exploring the potential of using genes as medicine to treat disease for more than 35 years. Several recent advancements have made it possible to apply this technology to Duchenne, Friedreich’s Ataxia, and other rare genetic diseases
A Different Way to Restore What’s Missing
Gene transfer is potentially beneficial in rare genetic diseases because it aims to address the root cause of the disease. While the risks and benefits of gene transfer in these genetic diseases still need to be evaluated in human clinical trials, early research is encouraging.
THE SCIENCE:Innovating in Gene Therapy
Solid chose to focus its initial efforts on gene therapy innovation due to the significant progress in our understanding of Duchenne Muscular Dystropy and the dystrophin gene, recently reported successes in gene therapy clinical development and advances in manufacturing.