RNA: From Biology to Drug Discovery 2026
Symposium Explores the Biology and Therapeutic Potential of RNA

The Anderson lab studies the role of long noncoding RNA in muscle biology, specifically the regulation of striated muscle development and function, and how defects in those pathways can give rise to human disease. His research has demonstrated that many annotated lncRNAs encode small functional proteins, micropeptides, which play important roles in regulating intracellular signaling.

Brenda Bass, Ph.D., is known for her contributions in defining double-stranded RNA-mediated pathways, including the discovery of ADAR RNA editing enzymes, and models and experiments that established Dicer's role in RNA silencing. She has been recognized with election to the National Academy of Sciences, the American Academy of Arts and Sciences, and as a Fellow of the American Association for the Advancement of Science. Her lab is interested in the poorly understood functions of double-stranded RNA, and how cell distinguish "good" dsRNA from viral dsRNA.

Benjamin Blencowe, Ph.D., is a professor in the University of Toronto Department of Molecular Genetics, Canada Research Chair in RNA Biology and Genomics and the Banbury Chair in Medical Research. His team studies the mechanisms by which genes are regulated and coordinated to provide critical functions in mammalian cells, specifically RNA splicing. Alternative splicing dicisions allow cells to greatly expand the structurally and functionally distinct RNA transcripts and proteins from a limited number of genes. Current research is determining how alternative splicing networks are integrated with orthogonal gene regulatory layers – including chromatin/epigenetic modification, transcription, mRNA 3´end processing, turnover, and translation – to control fundamental normal and disease-associated biology.

As director of the Department of Pharmacology at Weill Cornell Medicine, Samie R. Jaffrey, M.D., Ph.D., explores RNA modifications that control gene expression, coning the term "epitranscriptomics." The Jaffrey laboratory is interested in how deregulation of RNA-regulatory pathways can lead to neurological disfunction, cancer, autoimmune diseases and other conditions. His mapping of the N6-methyladenosine and other modified nucleotides has attracted interest to the abundant chemical modifications of messenger and non-coding RNA molecules, with significant epigentic effects on gene expresion. His lab is also developing RNA aptamer technologies for probing and manipulating cellular biology.

Jeffrey Keift is executive director of the New York Structural Biology Center and an adjunct professor at Columbia University. His research focuses on understanding how RNA structure, RNA conformational changes and complex intermolecular interactions combine to enable diverse RNA function. He is particularly interested in viral RNAs that form complex three-dimensional structures that interact with and manipulate the cellular machinery. At the NYSBC, he oversees a leading center for structural biology, linking molecular structure and disease.

Dr. Maquat’s research focuses on the molecular basis of human diseases, with particular interest in mechanisms of mRNA decay. Maquat discovered nonsense-mediated mRNA decay (NMD) in human diseases in 1981 and, subsequently, the exon junction complex (EJC) and how the EJC marks mRNAs for a quality control “pioneer” round of protein synthesis. She also discovered Staufen mediated mRNA decay, which mechanistically competes with NMD and, by so doing, new roles for short interspersed elements and long non-coding RNAs. Additionally, she defined a new mechanism by which microRNAs are degraded, thereby regulating mRNAs so as to promote the cell cycle. One of her current interests focuses on the development of therapeutics for diseases that she has shown manifest hyperactivated NMD, including the most common single gene cause of intellectual disability and autism, Fragile X Syndrome. Maquat is an elected Fellow of the American Association for the Advancement of Science (2006); an elected Member of the American Academy of Arts & Sciences (2006), the National Academy of Sciences (2011), and the National Academy of Medicine (2017); and a Batsheva de Rothschild Fellow of the Israel Academy of Sciences & Humanities.

Nikhil C. Munshi, MD is Professor of Medicine at the Harvard Medical School and the Director of Basic and Correlative Science, and Associate Director of the Jerome Lipper Multiple Myeloma Center at the Dana Farber Cancer Institute and an attending physician at the Brigham and Women’s Hospital and the Boston VA Healthcare system, Harvard Medical School. His research focus spans both basic sciences to understand molecular mechanisms driving the genomic instability in cancer to translational approaches directed at improving diagnosis and prognosis as well as therapeutics including novel antigen-directed immunotherapy/vaccine approaches and small molecules for myeloma. His research has provided pivotal understanding of genomic changes in myeloma.

Dr. Muthiah (Mano) Manoharan serves as the Senior Vice President of Drug Innovation, a Scientific Advisory Board Member, and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts. In 2003, he was the founding chemist hired at Alnylam as the Head of the Drug Discovery. He and his team pioneered the discovery and development of numerous chemical modifications, GalNAc conjugation chemistry, lipid conjugates (the first in vivo demonstration of RNAi in 2004) and Lipid Nanoparticles (LNP) delivery platform that made RNA interference-based human therapeutics possible. This work led to the approval of five RNAi therapeutics: ONPATTRO® (patisiran, 2018), GIVLAARI® (givosiran, 2019), OXLUMO® (lumasiran, 2020), LEQVIO® (inclisiran, 2020, 2021) and AMVUTTRO® (vutrisiran, 2022). Prior to joining Alnylam, he worked at Isis (Ionis) Pharmaceuticals from 1990-2003 in the field of antisense oligonucleotides.

The Ken lab is interested in combining computational and experimental approaches to examine RNA structure-function relationships in particular, both in vitro and in the cellular context. We aim to build quantitative models of cellular RNA-protein interactions that allow us to gain deeper understanding of fundamental biology as well as develop RNA-targeting strategies. While these tools and approaches can be be applied broadly, we are currently focused on viral RNAs and antiviral drug development.

Physician-scientist Duane Mitchell, M.D., Ph.D., serves as director of the UF Health Cancer Institute. As the Co-Director of the Preston A. Wells, Jr. Center for Brain Tumor Therapy at UF Health, he has pioneered an adoptive cellular therapy platform that uses total tumor RNA-pulsed dendritic cells to expand polyclonal tumor-specific T cells for the treatment of invasive and refractory brain cancers. His team is now refining the approach to add patient-specific antigen profiling using a novel cancer immunogenomics-based algorithm developed in his lab, allowing the targeting of hundreds of tumor-specific antigens in a single pool of RNA, thereby addressing tumor heterogeneity.

Elias Sayour, MD, PhD, is the Stop Children’s Cancer/Bonnie R. Freeman Professor for Pediatric Oncology Research in the UF departments of neurosurgery and pediatrics at the University of Florida. He is also a principal investigator of the ribonucleic acid engineering laboratory at the Preston A. Wells, Jr. Center for Brain Tumor Therapy. Primary high-grade gliomas (pHGGs) in children are almost uniformly lethal. While there is promise for immunotherapy, gliomas are overwhelmingly immunosuppressive, posing an access and activity challenge for the immune activation required for immunotherapy. Dr. Sayour and colleagues have developed a novel treatment platform which leverages the use of nanoparticles (NPs) combined with messenger RNA (mRNA) taken from the patient’s tumor to make a product that functions as both a vaccine and an agent that is capable of increasing activity of the immune system.