Katalin Kariko

Adjunct Professor
​University of Pennsylvania

Katalin Karikó is professor at University of Szeged and adjunct professor of neurosurgery at the Perelman School of Medicine, University of Pennsylvania, where she worked for 24 years. She is former senior vice president at BioNTech SE, Mainz, Germany, where she worked between 2013-2022. She received her Ph.D. in biochemistry from University of Szeged, Hungary, in 1982. Research-funding problems in Hungary drove her to the United States, where she did postdoctoral fellowships before accepting a faculty position at the University of Pennsylvania. There, Dr. Karikó collaborated with several clinical investigators as her work progressed, despite lack of grants and institutional support. During the 1990s, she persevered even though, by this time, conventional wisdom held that delivering intact mRNA to cells in the human body was infeasible.
For four decades, her research has been focusing on RNA-mediated mechanisms with the ultimate goal of developing in vitro-transcribed mRNA for protein therapy. She investigated RNA-mediated immune activation and co-discovered that nucleoside modifications suppress immunogenicity of RNA, which widened the therapeutic potentials of mRNA. She is co-inventor on mRNA-related patents for application of non-immunogenic, nucleoside-modified RNA. Fourteen of those are granted by the US. She co-founded and from 2006-2013 served as CEO of RNARx, a company dedicated to develop nucleoside-modified mRNA for therapy. Her patents, co-invented with Drew Weissman on nucleoside-modified uridines in mRNA is used to create the FDA-approved COVID-19 mRNA vaccines by BioNTech/Pfizer and Moderna to fight the pandemic. For her achievement she received many prestigious awards, including the Japan Prize, the Horwitz Prize, the Paul Ehrlich Prize, the Benjamin Franklin Medal, the Canada Gairdner International Award, the Kovalenko Medal, the Tang Prize, the Warren Alpert Prize, the Prince Mahidol Award, the BBVA Frontiers Award, the VinFuture Prize, the Breakthrough Prize and the Lasker-DeBakey Clinical Medical Research Award.

Speech title & Synopsis

Developing mRNA for therapy

Messenger RNA was discovered in 1961 and it took 60 years until the first mRNA became FDA-approved product in the form of COVID-19 mRNA vaccine developed by BioNTech/Pfizer and Moderna. During those years a lot of progress has been made by hundreds of scientists. For the first two decades, scientists analyzed mRNA isolated from cells and viruses. It was 1978 when the first-time isolated mRNA encapsulated into liposome was delivered into mammalian cells and the encoded protein was produced. In vitro transcription introduced in 1984 made it possible to generate any desired mRNA from the encoding plasmid using phage RNA polymerases. In the early 90s mRNA was used in animal studies for therapy as well as vaccine against infectious diseases and cancer. Inflammatory nature of the mRNAs limited their in vivo use. Replacing uridine with a naturally occurring alternative, pseudouridine made the mRNA non-immunogenic, more stable and highly translatable. Delivery of the lipid nanoparticle-formulated nucleoside-modified mRNA encoding viral antigens became a platform for effective vaccine. Labile nature of the mRNA is ideal for transient production of the viral antigen, to generate effective antibody and cellular immune response. These discoveries eventually led to the development of the mRNA vaccine that has now helped to fight the pandemic and opened the door for developing breakthrough therapeutics for unmet medical needs. Today, more than 150 clinical trials are running using in vitro-transcribed mRNA for cure. The mRNA platform is revolutionizing the delivery of effective and safe vaccines, therapeutics and gene therapies.