Anastasia Khvorova, PhD
Pioneering work in the field of RNA-based therapies for Huntington's disease
RNA researcher Anastasia Khvorova, PhD, professor of RNA therapeutics at UMass Chan Medical School, will receive this year's Else Kröner Fresenius Prize for Medical Research on May 15, 2025. The award ceremony will be a part of the festive event together with the Fresenius SE & Co. KGaA celebrating the 100th birthday of Else Kröner, at the Städel Museum in Frankfurt am Main.
Image source: RNA Therapeutics Institute
The foundation Else Kröner-Fresenius-Stiftung (EKFS) is honoring Khvorova's pioneering work in the field of RNA-based therapies. Her research contributes significantly to the development of new approaches for treating genetic and neurodegenerative diseases such as Huntington's disease. At 2.5 million euros, the Else Kröner Fresenius Prize for Medical Research is one of the biggest research prizes in the world.
Anastasia Khvorova, PhD, heads a multidisciplinary research team of chemists, biologists, pharmacologists, and biochemists. In her laboratory, she 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.
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 to prevent the production of toxic proteins in the brain. Such abnormal proteins are behind aging and many neurodegenerative diseases, such as Huntington's disease. Huntington's disease is a rare and fatal disease characterized by progressive movement disorders with impaired breathing, bedriddenness and susceptibility to infections. The symptoms typically begin when people are in their 30s to 50s. The disease is caused by a genetic mutation of autosomal dominant inheritance.
Profile:
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.
Source: Else Kröner-Fresenius-Stiftung
08.04.2025
