Understanding Our Science
Advancing science to tackle challenges
Advancing science to tackle challenges
At Sarepta, we are always looking for new ways to tackle the challenges of rare diseases that you and your loved ones are facing. We use the most advanced scientific tools to find targets for new treatments and to develop products that have the potential to improve patients’ health.
Exploring health at the most basic level: the cell
Sarepta science includes many fields of study, including biology, genetics, and chemistry. These are all parts of molecular biology—the study of how genetic information is created, and how it affects the way cells work. This includes DNA, RNA, and protein creation.
Sarepta is using the tools of molecular biology to develop a variety of investigational products in the areas of gene therapy, RNA technology, and gene editing. We are continually seeking new ways to treat rare genetic conditions, develop safe and effective products as quickly as possible, and streamline manufacturing and other processes.
Deoxyribonucleic acid, where genetic information is stored.
Ribonucleic acid, a transcript of information from DNA that has several functions, many of which relate to making proteins.
Large, complex molecules required for the structure, function, and regulation of the body’s cells, tissues, and organs.
Gene therapy: making history for 60 years
The science of gene therapy advances how we think about and create new products for you and your family every day. Yet this technology has actually resulted from decades of research, done by dedicated physicians and scientists.
See a summary of gene therapy history on this timeline of key events.
