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16.05.2025 10:14

RNA-based therapies: Hope for previously incurable diseases like Huntington’s disease

Bianka Jerke Presse- und Öffentlichkeitsarbeit
Else Kröner-Fresenius-Stiftung

    Anastasia Khvorova receives Else Kröner Fresenius Prize for Medical Research during celebratory event marking Else Kröner’s 100th birthday

    Bad Homburg v. d. Höhe, May 16, 2025 – Last night, the Else Kröner-Fresenius-Stiftung (EKFS) conferred the 2025 Else Kröner Fresenius Prize for Medical Research to Prof. Dr. Anastasia Khvorova for her pioneering work in the field of RNA-based therapies. At 2.5 million euros, the prize is one of the most prestigious and generously endowed of any medical research prize worldwide. Khvorova plans to use the prize money to advance the development of new RNA therapies targeting genetic and neurodegenerative diseases such as Huntington’s disease. The award ceremony took place at the Städel Museum in Frankfurt am Main as part of a joint celebration together with Fresenius SE & Co. KGaA marking what would have been the 100th birthday of foundation benefactress, Else Kröner.

    Anastasia Khvorova is a professor at the RNA Therapeutics Institute of UMass Chan Medical School. For more than 20 years now, she has been researching how RNA molecules can be used as medications to target disease processes. She focuses on chemical modifications that will improve the stability of RNA therapeutics, ensuring that they reach specific organs.

    Thanks to Khvorova’s research, RNA medications are no longer be limited to just the liver, but can also reach other organs such as the brain, heart, or muscles. She plans to use the prize money to develop novel RNA therapies for genetic brain diseases such as Huntington’s disease. In this hereditary and as yet incurable neurological disease, a gene mutation leads to the formation of toxic proteins that damage a specific group of nerve cells, causing progressive movement disorders, paralysis, and eventually death. Khvorova aims in her work to prevent the underlying genetic changes and thus inhibit the production of toxic proteins. This would enable a targeted therapeutic approach for Huntington’s disease for the first time.

    “With the Else Kröner Fresenius Prize for Medical Research, we acknowledge scientific achievements that can pave the way for medical breakthroughs,” says Prof. Dr. Michael Madeja, Chairman of the Management Board at the EKFS, during the award ceremony. “We are delighted that Professor Khvorova plans to use the prize money to advance her research into therapies for this as yet incurable disease.”

    RNA-based therapies: A key technology for the medicine of the future

    The award not only honors Khvorova’s outstanding research achievements to date, but also recognizes the future potential of her work. RNA-based therapies are considered a key technology for the medicine of the future because they can intervene precisely in disease processes at the molecular level and modify them.

    “RNA therapies offer powerful new tools for treating human disease by targeting its root causes — correcting the genetic instructions that underlie and drive pathology,” said Craig Mello, PhD, Nobel Laureate in Medicine and chair of the jury for the Else Kröner Fresenius Prize for Medical Research. “Anastasia Khvorova’s pioneering work is expanding the reach of this technology beyond the liver, opening the door to transformative treatments for a wide range of conditions.”

    “Huntington’s disease is a devastating diagnosis, not only for those affected but also for entire families. With the prize money, we have the opportunity to develop a new generation of RNA therapies that could specifically target this disease and many similar neurodegenerative diseases,” said Anastasia Khvorova in her acceptance speech. “It is a great honor for me that the Else Kröner-Fresenius Foundation is recognizing my team and our research with this award and giving us the opportunity to continue making significant progress.”

    The award ceremony took place at the Städel Museum in Frankfurt during the celebrations marking what would have been the 100th birthday of the entrepreneur and foundation benefactress, Else Kröner. In addition to honoring the prizewinner, the event highlighted Else Kröner’s impressive legacy. Else Kröner not only shaped Fresenius as a company, but also perpetuated her commitment to medical research and humanitarian aid when she established the foundation.

    More information
    Prizewinner film: https://youtu.be/A6wbdyny2Hs
    Film on the occasion of Else Kröner's 100th birthday: https://youtu.be/tlIZtWcZh1Y
    EKFS website: https://ekfprize25.de
    Website of Anastasia Khvorova’s research lab: https://www.umassmed.edu/khvorovalab/

    Else Kröner-Fresenius-Stiftung (EKFS) – Advancing research. Helping people.
    Else Kröner-Fresenius-Stiftung is a non-profit foundation dedicated to the funding and advancement of medical research and the support of humanitarian projects. To date the foundation has funded around 2,800 projects. With an annual funding volume currently amounting around 60 million euros it is the largest foundation in Germany that actively funds and supports medicine. You can find more information at: www.ekfs.de/en

    Press Contact
    Else Kröner-Fresenius-Stiftung
    Bianka Jerke
    Public Relations
    Tel.: + 49 6172 8975-24
    Email: b.jerke@ekfs.de


    Wissenschaftliche Ansprechpartner:

    Dr. Alexander Lehmann
    Scientific Funding: Graduate and Research Schools and International Research Prize
    a.lehmann@ekfs.de
    +49 (6172) 8975-25


    Weitere Informationen:

    https://www.ekfs.de/en/scientific-funding/international-research-prize/ekfpm2025


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