The Else Kröner Fresenius Foundation is awarding the 2.5 million euro prize in recognition of Khvorova's pioneering work in the field of RNA-based therapies. Her research has contributed significantly to the development of new approaches for treating genetic and neurodegenerative diseases. The award ceremony will take place on May 15, 2025, at the Städel Museum in Frankfurt am Main.
Anastasia Khvorova, PhD, is a professor of RNA therapeutics at UMass Chan Medical School. The Khvorova Lab develops and modifies RNA molecules to improve their effectiveness in the human body. RNA-based therapeutics use a variety of RNA molecules to fight diseases at the molecular level. These innovative approaches make it possible to inhibit the production of toxic proteins and stimulate the production of missing proteins. Despite great progress in the field, there are still major challenges.
Khvorova's work focuses on chemical modifications that improve the delivery, stability, efficacy, and specificity of such therapeutics. In practice, RNA molecules often remain inaccessible to organs other than the liver. Much of Khvorova's research to date has focused on enabling RNA therapeutics to reach other organs, including the brain, muscle, heart, lung, and placenta, so that they can effectively carry out their function.
Khvorova plans to use the prize money to develop novel RNA molecules that can prevent the production of toxic proteins in the brain. Such proteins occur due to the expansion of repeating sequences in certain genes and are the basis of more than 50 neurodegenerative diseases, one being Huntington's disease.
Learn more about Anastasia Khvorova in the interview.
Anastasia Khvorova, PhD, has more than two decades of experience pioneering oligonucleotide-based drug development. She began her scientific career in Moscow, where she completed her academic training and earned her doctorate in 1994. In 1995, Khvorova moved to the United States, where she has conducted research at various universities and held senior positions in the pharmaceutical industry. She holds more than 150 patents and is an elected fellow of the National Academy of Inventors. She has been a professor in the RNA Therapeutics Institute at UMass Chan Medical School in Worcester, Massachusetts since 2012.
Further information here.
Prof. Craig Mello (Chair of the Jury, 2006 Nobel Prize Laureate, Massachusetts), Prof. Elizabeth Blackburn (2009 Nobel Prize Laureate, San Francisco), Prof. Stefan Endres (Chairman of the EKFS Scientific Commission, Munich), Dr. Jeremy Farrar (WHO Chief Scientist, Geneva), Dr. Joao Monteiro (Chief Editor of Nature Medicine), Prof. Ole Petter Ottersen (Former President of the Karolinska Institutet, Oslo), Prof. Christian Reinhardt (Member of the EKFS Scientific Commission, Essen), Dr. Orla Smith (Editor of Science Translational Medicine), Prof. Fiona Watt (Director of the European Molecular Biology Organization, EMBO, Heidelberg).
The press release of the Else Kröner Fresenius Prize for Medical Research 2025 can be found here.