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Anastasia Khvorova, PhD

Professor, RNA Therapeutics Institute and Remondi Family Chair in Biomedical Research

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Research Focus - Medicinal Chemistry of Therapeutic RNAs

  • Development of novel oligonucleotide-based therapeutics
  • Drug development
  • Mechanism of small RNA trafficking
  • RNA medicinal chemistry and delivery

Representative Publications

  • An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis. Yenilmez B, Wetoska N, Kelly M, Echeverria D, Min K, Lifshitz L, Alterman JF, Hassler MR, Hildebrand S, DiMarzio C, McHugh N, Vangjeli L, Sousa J, Pan M, Han X, Brehm MA, Khvorova A, Czech MP.Mol Ther. 2021 Nov 10:S1525-0016(21)00578-5. doi: 10.1016/j.ymthe.2021.11.007. Online ahead of print.PMID: 34774753 Read Publication
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In the News

  • Novel siRNA backbone developed at UMass Chan enhances stability, durability of potentially therapeutic platform
    Aug 5, 2024

    Novel siRNA backbone developed at UMass Chan enhances stability, durability of potentially therapeutic platform

    A biochemical breakthrough using simple carbon atoms by Ken Yamada, PhD, and Anastasia Khvorova, PhD, has dramatically improved the stability and efficacy of a potential oligonucleotide therapeutic platform in mice.

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  • Local high school students compete in Central Mass. Brain Bee at UMass Chan
    Apr 4, 2024

    Local high school students compete in Central Mass. Brain Bee at UMass Chan

    Natick resident and Stanford Online High School student Rebecca Ahn outperformed other local high school students in the final round of the competition on March 16.

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  • UMass Chan helps ‘Crack the Code’ at biotech career day in Worcester
    Oct 25, 2023

    UMass Chan helps ‘Crack the Code’ at biotech career day in Worcester

    Representatives from UMass Chan presented on several topics and career paths, including RNA Therapeutics Institute, ScienceLive, community and government relations, diversity and inclusion and media and arts in sciences.

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  • Science for Living: Understanding preeclampsia, its toll on Black women and a promising therapeutic on the way
    Oct 2, 2023

    Science for Living: Understanding preeclampsia, its toll on Black women and a promising therapeutic on the way

    A new RNA therapeutic for preeclampsia is based on work by UMass Chan researchers and collaborating scientists. Researchers explain the importance of a multipronged approach to addressing factors underlying health disparities in this Science for Living article.

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  • MD/PhD student using NIH award to study onset of Huntington’s disease
    May 15, 2023

    MD/PhD student using NIH award to study onset of Huntington’s disease

    MD/PhD student Jillian Belgrad has received an award from the National Institute of Neurological Disorders and Stroke to study the pathology that drives the development of Huntington’s disease symptoms.  

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  • UMass Chan scientists deliver siRNA therapy to lung
    Apr 12, 2023

    UMass Chan scientists deliver siRNA therapy to lung

    A multidisciplinary team of UMass Chan scientists has developed a technology for delivering siRNA molecules locally to lung tissue, providing the first evidence of a platform capable of delivering chemically modified siRNAs to the lungs nasally and achieving robust genetic silencing.

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  • UMass Chan researcher Anastasia Khvorova named 2022 National Academy of Inventors Fellow
    Dec 12, 2022

    UMass Chan researcher Anastasia Khvorova named 2022 National Academy of Inventors Fellow

    Anastasia Khvorova, PhD, is one of 169 academic inventors selected as a 2022 fellow of the National Academy of Inventors.

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  • Boston Marathon runners raise thousands for UMass Chan ALS research
    Apr 5, 2022

    Boston Marathon runners raise thousands for UMass Chan ALS research

    ALS research at UMass Chan Medical School is the big beneficiary as select Boston Marathon runners raise tens of thousands of dollars each year. This year, five competitors in the April 18 event are running as part of the UMass Chan ALS Cellucci Fund Boston Marathon team and two sons of the late Jake Kennedy will run in support of the Jake Kennedy ALS Fund.

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  • UMass Cancer Center funds five UMass Chan research projects
    Nov 17, 2021

    UMass Cancer Center funds five UMass Chan research projects

    The UMass Cancer Center Pilot Project Program has awarded seed grants to five UMass Chan Medical School research projects to enable investigators to gather pilot data for external cancer-focused funding.

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  • Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium
    Apr 7, 2021

    Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium

    Six UMass Medical School scientists are among the members of the National Institutes of Health’s Somatic Cell Genome Editing Consortium to publish a paper in Nature outlining the program’s goals.

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  • Atalanta Therapeutics founded by UMass Medical School and three faculty members
    Jan 12, 2021

    Atalanta Therapeutics founded by UMass Medical School and three faculty members

    Atalanta Therapeutics, a biotech founded by UMass Medical School and three faculty research scientists to pioneer treatment options for neurodegenerative diseases, has launched with financing by venture capital fund F-Prime Capital and strategic collaborations with Biogen and Genentech.

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  • Inside the new mRNA vaccines for COVID-19
    Dec 18, 2020

    Inside the new mRNA vaccines for COVID-19

    The new vaccines by Pfizer/BioNTech and Moderna use messenger RNA to stimulate the immune system to protect against COVID-19. These vaccines are the first of their kind and researchers at UMass Medical School are among the leading RNA biologists in the world.

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  • UMass Medical School community mourns loss of Jake Kennedy
    Oct 14, 2020

    UMass Medical School community mourns loss of Jake Kennedy

    On behalf of the UMass Medical School community, we extend our sincere condolences to Jake Kennedy’s family and to all who knew and loved him. Jake Kennedy died Tuesday, Oct. 13, at age 65, in his Salem, N.H., home of amyotrophic lateral sclerosis.

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  • Anastasia Khvorova elected director of the American Society of Gene and Cell Therapy
    May 11, 2020

    Anastasia Khvorova elected director of the American Society of Gene and Cell Therapy

    UMass Medical School RNA therapeutics expert Anastasia Khvorova, PhD, has been elected by the membership of the American Society of Gene and Cell Therapy to a three-year term as director-at-large/scientific & research council chair, which will begin at the 2020 ASGCT virtual annual meeting.

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