Athma Pai
UMass Chan Medical School

RNA Therapeutics: Recoding Drug Design, One Gene at a Time

JULY 18, 6:30 PM
Telluride conference center


Presented by Athma Pai
RNA Therapeutics Institute Assitant Professor
UMass Chan Medical School
The advent of genomic sequencing has revolutionized our ability to identify disease-causing genetic sequences. With this advancement comes the potential for personalized medicines tailored to an individual’s genetic information, targeting specific genes to modulate their activity. To achieve this, it is necessary to have drugs capable of precise but customizable genetic targeting. In recent years, scientists have made remarkable progress in harnessing the intrinsic targeting abilities of RNA molecules for these purposes. RNAs play several roles in cells, including serving as regulatory molecules that fine-tune genetic outputs by selectively binding and targeting specific sequence elements. Thus, unlike traditional small molecule drugs, small RNAs can be designed based on sequence information to target nearly any gene in the human genome, enhancing or suppressing functional activity with minimal off-target effects. While still in the early stages, RNA therapies have exhibited promising results and represent one of the most rapidly advancing fields in medicine. This talk will describe the various classes of RNA therapeutics, including small interfering RNAs (siRNAs), antisense oligonucleotides (ASOs), and CRISPR guide RNAs, and discuss their unique mechanisms of action. Second, it will discuss promising new applications of these molecular modulators and the challenges that remain in designing, developing, and testing these therapies.