Gene transfer is an investigational approach that aims to significantly slow or even halt the progression of Duchenne muscular dystrophy (Duchenne) by delivering a modified yet functional dystrophin 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.
A Different Way to Restore What’s Missing
Gene transfer is potentially beneficial in Duchenne because it aims to address the root cause of the disease. While the risks and benefits of gene transfer in Duchenne 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 Duchenne, it has the potential to benefit those living with the disorder, regardless of a person’s disease mutation.
Potential to Slow or Stop Duchenne
Gene transfer may be a way to restore expression of a modified yet functional dystrophin in all important muscle groups, including the heart, diaphragm and skeletal muscles.
Potential Impact
While gene transfer is intended to promote ongoing expression of a modified dystrophin 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 Transfer
Solid chose to focus its initial efforts on gene transfer innovation due to the significant progress in our understanding of the dystrophin gene, recently reported successes in gene therapy clinical development and advances in manufacturing.
- The dystrophin protein, a critical component in muscle formation, is undetectable in Duchenne patients.
- The absence of dystrophin is the fundamental indicator of muscle degeneration.
- Skeletal and cardiac muscles all lack dystrophin and symptoms are pervasive.