Harnessing Small Interfering RNA for Rare Diseases: present and future possibilities

Details

Supervisors

Revencu, Nicole

Faculty

Faculté de pharmacie et des sciences biomédicales

Degree label

Master [120] en sciences pharmaceutiques, à finalité spécialisée

Abstract

Rare diseases are medical conditions that represent significant challenges. Their impact is not only experienced by the affected patient but be family, friends and healthcare system as well. The complex panel of symptoms, delayed diagnosis and limited therapeutical options cause a decline in quality of life. Curative treatments and tailored healthcare plans may ensure better patient medical management. A solution could be small interfering RNA (siRNA) technology. In fact, their gene-silencing ability offers the opportunity to target specific genes and inhibit disease-causing proteins. Three approved siRNA-based drugs were presented through Real World Data to explore present clinical use of these molecules (Patisiran, Givosiran, Lumasiran) in rare diseases. One phase III trial siRNA-based drug was selected to present future possibilities concerning this novel therapeutic class. Challenges and future perspectives related to these drugs were discussed.