LEADING A RENAISSANCE IN RNA BIOLOGY
The origins of RNA-targeted medicines began with scientific discoveries more than 30 years ago. We are only now on the threshold of realizing their promise with mature technologies, including Sarepta’s phosphorodiamidate morpholino oligomer—or PMO—chemistry platform. Our company is a leader in the development of PMO-based,
RNA-targeted therapeutics, with multiple drug candidates for rare, infectious and other diseases in preclinical and clinical development.
RNA: THE BASICS
Humans have about 22,000 genes, which contain the blueprints for producing proteins that perform essential functions in the body.
Proteins are molecular workhorses involved in almost every function in our bodies, and defective proteins often result in disease. More specifically, some diseases may be caused by the over-production of one or more proteins, while other diseases are caused by protein deficiencies.
Proteins are produced in cells, where genes in the DNA are
"transcribed" into RNA templates, which are then processed
and "translated" into proteins by the cellular machinery.
Our RNA-targeted therapeutics direct the cellular machinery involved in making proteins. These drugs can be designed to increase or decrease the production of a protein involved in a disease.
By working at the genetic level, RNA-targeted therapeutics are powerful tools with
the potential to address rare, infectious and other diseases that otherwise
could not be treated with traditional small molecule or biologic drugs.