Daniel J. Siegwart

Dr. Daniel J. Siegwart is an Associate Professor in the Department of Biochemistry at the University of Texas Southwestern Medical Center and a co-founder of ReCode Therapeutics. His research laboratory utilizes materials chemistry to enable targeted delivery of genomic medicines. He has focused on overcoming difficult challenges in nucleic acid delivery. He recognized that although great advances had been made in the delivery of short RNAs, the ideal chemical and formulation composition was poorly understood for longer RNA cargo such as messenger RNA. Their efforts led to an understanding of the essential physical and chemical properties of synthetic carriers required for therapeutic delivery of genomic medicines including siRNA, miRNA, tRNA, pDNA, and mRNA.

He has been at the forefront in the design of synthetic carriers for gene editing and has applied these technologies for correction of genetic diseases. The Siegwart lab reported the first non-viral system for in vivo CRISPR/Cas gene editing in December 2016. Recently, they developed Selective ORgan Targeting (SORT), which is the first and only strategy for predictable tissue specific mRNA delivery and gene editing. SORT lipid nanoparticles are systematically engineered to exclusively edit extrahepatic tissues and therapeutically relevant cell types. SORT is compatible with multiple gene editing techniques, including Cas9 mRNA / sgRNA / ssDNA, and Cas9 ribonucleoprotein (RNP) complexes for gene editing in targeted tissues.