Sudhir Agrawal is the president and founder of Arnay Sciences and an associate professor at UMass Medical School, with extensive research in RNA therapeutics and antisense technology.
In this presentation, Agrawal explained that the foundation of Arnay’s technology involves targeting RNA with complementary DNA or RNA molecules. These molecules bind to RNA at any stage in the cell machinery with the ultimate goal of modulating protein expression while managing immune responses to foreign nucleic acids.
This particular field has been met with difficulties, mainly due to unwanted immune responses tiggered by the recognition of foreign nucleic acids. How this kind of immune response works was not fully understood until recent discoveries about innate immune responses.
Antisense RNA or DNA therapeutics can now be designed fairly easily, but depending on its sequence, length, and composition, it could be implicated in machinery it was not supposed to. Agrawal’s focus for this presentation was on providing drug-like properties to nucleic acids – noting the growing list of approved drugs in this modality.
Agrawal then outlined the evolution of antisense technology, highlighting the significance of chemical modifications that enhance stability and efficacy. He particularly noted the creation of gapmers, which combine DNA and RNA features. He also discussed safety concerns related to immune activation and inflammatory responses, emphasising the need for careful design to avoid adverse effects while maintaining therapeutic efficacy.
The second half of the presentation outlines the recent innovations nucleic acid drug design. These include the development of spatially designed structures that improve antisense potency and specificity, addressing previous limitations in drug design. Agrawal then discussed the future advancements in RNA therapeutics, focusing on minimizing immune responses while enhancing the therapeutic potential through innovative chemical engineering approaches.