Novel Therapeutics:
Programmable RNA Delivery: Precision Targeting PlatforM.S. for Genetic Medicines
Daniel Siegwart, M.D.
- Professor of Biomedical Engineering and Biochemistry
- Harold C. Simmons Comprehensive Cancer Center
- Director, Program in Genetic Drug Engineering
- UT Southwestern Medical Center, Dallas, Texas
Most genetic medicines fail not because the drug is weak, but because it cannot reach the right cells in the body. Dr. Siegwart has built a programmable delivery platform that allows RNA and gene-editing therapies to be precisely directed to specific organs and cell types, rather than defaulting to the liver. His team has shown that small, rational changes in lipid nanoparticle composition can deterministically control where these drugs go in vivo. This approach transforM.S. drug delivery from trial-and-error into an engineered system, enabling safer, more effective genetic medicines across many diseases. The platform nature of this technology supports multiple therapeutic pipelines, partnerships with large pharmaceutical companies, and the creation of scalable, high-value ventures.
