Platform approach

Actelion’s efforts in drug discovery have focused on developing platforms of expertise based on its core capabilities. This focus allows high productivity in the generation of innovative compounds potentially addressing a wide range of highly unmet medical needs.

The first focus is the design, synthesis and optimization of low molecular weight drug-like molecules. The productivity of Actelion’s drug discovery endeavors is demonstrated by the more than 2,500 pending patent applications and/or granted patents currently in Actelion’s portfolio.

Actelion also focuses on the choice of its molecular target families. Initially, the company looked solely at G-protein coupled receptors (GPCRs) and a specific enzyme family known as aspartic proteinases. As the company’s capabilities have expanded, so too have the target platforms to include anti-infectives, ion channels and a broad range of soluble enzymes.