Locked Nucleic Acid (LNA) Platform
RNA-targeting medicines are designed to access disease targets that are difficult to reach with current medicines. Typically, RNA-targeted therapies come in two varieties: synthetic single-stranded DNA molecules, often referred to as "antisense"; and synthetic double-stranded RNA molecules, often referred to as "siRNA". Roche recently acquired the RNA-targeting Locked Nucleic Acid (LNA) platform that could deliver LNA-based drug candidates for diseases that are difficult, or impossible, to target with antibodies and small molecules.
LNA-based molecules overcome the limitations of earlier antisense and siRNA technologies through a unique combination of small size and very high affinity that could allow this new class of drug candidates to potently and specifically inhibit RNA targets in many different tissues without the need for complex delivery vehicles. The LNA platform may therefore provide the means to efficiently discover and develop an important new class of medicines that may address the significant needs of patients across multiple therapeutic areas.