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Abstract: The Ekker Precision Gene Editing Laboratory developed morpholinos, gene-breaking and other transposon tools, and the first HDR editing in zebrafish. Recent gene editing advances include precision targeted large knockins using GeneWeld, smaller knockins using DonorGuide, and new programmable edits in the mitochondrial genome. This presentation will include technical and application updates for each approach, enabling the massive parallel assessment of AI-based CAR-T therapies, and the establishment of the first unconstrained mitochondrial TALE base editors. Recent advances in AI- prediction tools enable rational structure-based design of next generation gene editing tools including Zippered Donor Guide and mitoFUSX TALE base editors.

Biography: Stephen C Ekker PhD lead the Precision Gene Editing Laboratory and the Center for Rare Disease at the University of Texas at Austin. He uses zebrafish in vivo and human cells in vitro to develop new nucleic acid modifying tools to understand our genome and for new therapeutics. As Associate Dean of Innovation and Entrepreneurship at Dell Medical School, Ekker is leading the development of key innovation and entrepreneurship education and applications within Dell Medical School and the University of Texas Austin campus. Dr Ekker is a Professor of Pediatrics and Professor of Molecular Biosciences. Ekker leads the Dell Med Office of Innovation and Entrepreneurship that includes the Texas Health Catalyst program and portfolio, including the semi-annual Roundup and Rodeo. Ekker has been gene editing for over three decades at the University of Illinois Urbana-Champaign, Johns Hopkins University, University of Minnesota and Mayo Clinic. Ekker is the Editor-in-Chief of the Zebrafish journal, and he is a past President of the Zebrafish Disease Models Society. He was until recently the Dean of the Mayo Clinic Graduate School of Biomedical Sciences and founder and Director of the Mayo Clinic Office of Entrepreneurship. Ekker is an active inventor, has been continuously funded by the NIH for over two decades and has co-authored 180+ scientific publications. Dr Ekker was SAB leader for OpenTrons Labworks, fostering research using accessible lab automation, as it went from the YCombinator accelerator to unicorn. Ekker is co-founder of DGI (now Immusoft), a biotech company pioneering engineered B-cells as therapies with first-in-human clinical success. Ekker is a co-founder, Board Member and investor in LEAH Labs, the first gene editing company out of YCombinator. LEAH Labs is pioneering off-the-shelf CAR-T treatments for cancer, the first to put dogs with lymphoma into remission. Ekker recently founded UViiVE Inc that uses unique AI-based novel protein designs with cloud lab business models for rapid health technology startup iteration.