Oligonucleotide Therapeutics Society

Nucleic acid-based therapies have become the third major drug class after small molecules and antibodies. The role of nucleic acid-based therapies has been strengthened by recent regulatory approvals and tremendous clinical success. In this review, we look at the major obstacles that have hindered the field, the historical milestones that have been achieved, and what is yet to be resolved and anticipated soon. This webinar provides a view of the key innovations that are expanding nucleic acid capabilities, setting the stage for the future of nucleic acid therapeutics.Speakers: Hassan Fakih, PhD (UMass Chan Medical School) and Jillian Belgrad, MD, PhD Candidate (UMass Chan Medical School)

Monoclonal antibodies represent a highly successful class of therapeutics that are used to treat complex diseases across a wide range of therapeutic areas. However, their high cost and variable patient response underscores the need for new modalities that can be used to activate or suppress the immune system. Aptamers have long been viewed as a simpler alternative to antibodies, but challenges with biostability, effectiveness, and throughput have slowed their progress in the clinic. In this talk, I will discuss a path for democratizing antibody therapeutics that relies a class of molecules called threomers, which are highly functionalized synthetic nucleic acids assembled on a biologically stable threose nucleic acid (TNA) framework. I will describe the underlying chemistry and enzyme engineering developed to support a high throughput discovery pipeline and provide examples of recently discovered threomers that mirror the binding properties of antibodies.Speaker: John Chaput, PhD, Professor - University of California, Irvine