Craig Duvall, Cornelius Vanderbilt Professor, Department of Biomedical Engineering; Professor, Ophthalmology and Visual Sciences; Professor, Chemical and Biomolecular Engineering
The central dogma of biology is that genomic DNA encodes for messenger RNA (mRNA), which is then translated into proteins. Proteins are the fundamental units that drive the functions of cells and tissues in the body, both in health and in disease. Historically, drugs have been developed as compounds that can modulate the function of mature proteins. New classes of drugs have emerged that are based on RNA. Variations of RNA-based therapies have now been designed and applied that can either block or supplement translation of the protein encoded by a desired target gene. Recently, both RNA interference (gene silencing) and mRNA-based (gene expression promoting) drugs have progressed into clinical use, making it clear that genetic medicines have great potential for impacting human health. RNA-based drugs, in particular, allow one to modulate gene targets that are not “druggable” by traditional approaches and also to correct genetic mutations (i.e., gene editing). The Duvall Advanced Therapeutics Laboratory (ATL) specializes in design and application of smart polymer-based technologies for intracellular delivery of biological drugs such as peptides and nucleic acids. We also have established strengths in molecular design and chemistry of protein and RNA therapeutics, many of which have proven successful in preclinical models of diseases like breast cancer and osteoarthritis.
“While the Covid-19 pandemic was clearly horrific, a positive outcome has been the broader recognition of the power of RNA-based drugs such as the mRNA-based lipid nanoparticle vaccines. While it is less publicized, there are also now 5 approved gene silencing drugs, all of which have been approved in the last 5 years. Now that the proof of concept is established in specialized situations for gene silencing and gene supplementation, the race is on to expand RNA platform technologies for application for new diseases that are not well-managed by currently-available drugs. I anticipate exponential growth in the clinical use of RNA-based therapies in the near future, and hope that my lab can contribute to this groundbreaking and rapidly expanding area of research.”