RNA Targeted Therapies

RNA-targeted therapeutics have gained significant traction due to advancements in transcriptomic technologies that have unveiled the multifaceted roles of RNA in genetic regulatory mechanisms and cellular activities.

RNA, sometimes described as DNA’s lesser-known cousin, plays a key role in cells. If DNA is the instruction manual for making proteins, then RNA provides the means for reading that document. RNA plays a crucial role in numerous cellular processes and decision-making mechanisms. Therefore, any disruptions or malfunctions in RNA structures can significantly contribute to the onset and progression of various diseases.

Meanwhile, conventional approaches to targeting proteins encounter challenges due to the limited number of "druggable" protein targets.

The structure-based recognition of RNA tertiary structures by RNA-targeted small molecules (“rSM”) provides an alternative to sequence-based approaches, such as antisense oligonucleotides. rSM approaches enable novel therapeutic potential by allowing to target highly conserved parts of RNA, creating pathways in cases where the encoded protein cannot be targeted conventionally, and unlocking the largely unexplored field of non-coding RNAs, which can also be disease drivers.

Evotec's SM RNA targeting platform focuses on identifying RNA tertiary structural elements for selective and high-affinity binding of RNA-targeted small molecules developing orally available small molecule binders and validating their engagement with disease-relevant RNA structures.

Evotec’s innovative RNA small molecule platform builds on the well-established drug discovery routes within Evotec and combines them with novel, highly innovative technologies such as third generation sequencing, sequencing-based structure elucidation of RNA molecules, which constitute a first-class expertise in this area.

mRNA therapeutics for targeted protein delivery has fueled great hope to combat a wide range of incurable diseases and gained significant momentum in recent years, especially due to the field of mRNA-based Immunotherapy. Evotec’s mRNA therapeutic platform is set up to deliver mRNA designs to optimize mRNA stability and translational fitness. The platform is set up to support small scale to mid-scale IVT mRNA production (up to hundreds of mg) of highly purified mRNA with optimized 5’ / 3’ UTR sequences and optimized polyA length and 5’ capping.

The safe delivery of mRNA therapeutics can be achieved by combining Evotec’s outstanding platforms in molecular biology, RNA biology and delivery/formulation. The platforms combine the strength of Evotec’s state-of-the-art know-how and strengthen the delivery of mRNA-based therapeutics design principles, manufacture, applications, delivery, and effects. This necessitates contributions of different areas like formulation, in vitro, and in vivo, evaluation assays. This allows Evotec to deliver mRNAs as powerful and versatile tools to combat genetic, infectious, cancer and other disease areas.