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.
Potential to Help Many
Because gene transfer targets the underlying cause of rare genetic diseases, it has the potential to benefit those living with the disorder.
Potential to Slow or Stop Genetic Diseases
Gene transfer may be a way to restore the expression of a functional protein throughout the body, including the heart, central nervous system, diaphragm, and skeletal muscles.
Potential Impact
While gene transfer is intended to promote ongoing expression of the missing protein after a single treatment, the duration of its potential effect is unknown and needs to be evaluated in clinical studies.
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.