Oligonucleotide Therapies

Sequence-based approaches for targeting RNA, such as siRNA and ASOs, utilize synthetic oligonucleotides to bind specifically to RNA targets, leading to altered protein expression. These approaches can achieve downregulation of the target by degrading the RNA through RNAseH or RISC-mediated mechanisms. However, the field is rapidly expanding, and oligos can also have diverse mechanisms of action, including modulating splice variants and upregulating protein expression.

While the field has been around for over two decades, it has gained significant clinical success in the past six years, with 17 drugs already on the market for various indications. This success is expected to accelerate as the field gains momentum. We have been actively expanding our capabilities in this area and collaborates with multiple partners on projects involving therapeutic siRNAs and ASOs, both conjugated and unconjugated, throughout the drug discovery and development process. We offer expertise in design, synthesis, hit identification screening, lead optimization, modification chemistry, early toxicology, and IND-enabling studies across various therapeutic areas, including oncology, metabolic diseases, CNS diseases, rare diseases, infectious disease, and women's health. Furthermore, our teams explore extrahepatic delivery opportunities as well as the diverse range of mechanisms of action for oligonucleotide drugs.