Gene therapeutic applications, including ex vivo and in vivo gene replacement and editing, often require a long, single-stranded template or donor DNA. Conventional solid-phase DNA synthesis is limited to short oligonucleotides, and enzymatic synthesis is challenging to scale for quantities needed in pre-clinical and clinical studies. In the first part of the talk, the speakers will show how DNA can be used to fabricate virus-like particles for early stage, in vitro studies of prophylactic vaccine candidates in HIV and SARS-CoV-2, and then give an outlook on therapeutic, cancer vaccines. In the second part of this talk, they will present a scalable, biotechnological process developed to achieve production of grams and larger quantities of single-stranded DNA of custom sequence with lengths up to ten kilobases, and will then highlight important milestones for pharmaceutical-grade manufacturing on an industrial scale.
Mark Bathe is a Professor in the Department of Biological Engineering at MIT, Director of the MIT New Engineering Education Transformation, Member of the Harvard Medical School Initiative for RNA Medicine, and Associate Member of the Broad Institute of MIT & Harvard. He obtained his Doctoral Degree at MIT working in the Departments of Mechanical, Chemical, and Biological Engineering before moving to the University of Munich as an Alexander von Humboldt Fellow to carry out his postdoctoral research in Biological Physics. He returned to MIT in 2009 to join the faculty in the Department of Biological Engineering, where he runs an interdisciplinary research group focused on engineering nucleic acids for application to vaccines, therapeutics, structural biology, and computing. He is academic co-founder of Cache DNA, Inc. and Kano Therapeutics, Inc., and in his free time he enjoys running, biking, swimming, and skiing amongst other outdoor activities.
Dr. Engelhardt is Postdoctoral Researcher in the Department of Biological Engineering at MIT. She joined Professor Bathe’s lab in February 2020 after obtaining her Doctoral Degree from the Technical University of Munich (TUM). She holds a Bachelor’s degree in "Biochemistry and Molecular Biology" and Master´s degree in "Molecular Biotechnology". Throughout her PhD she worked with Professor Hendrik Dietz and focused on improving DNA origami design, developing application-specific DNA origami purification techniques, as well as biotechnological single-stranded DNA production in the range of 1 to 10kb. She is a mentor in the student project "Space Origami" and is part of the organizational team for the networking event "DNA Node Munich".