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.

The Burke lab uses biochemistry, molecular biology, and advanced microscopy to understand the RNA biology of cellular responses to pathogenic viral infections, including Dengue virus, Zika virus, SARS-CoV-2, and influenza A virus. Current work has focused on demonstrating that dsRNA sensors condense on viral dsRNA to promote their activation. For example, oligoadenylate synthetase 3 (OAS3) condenses on viral dsRNA (OAS3-DRIF), which leads to activation of RNase L, rapidly degradeing all cellular mRNA. The lab additionally found that RNase L-induced bodies promote RNA decay.

Dr. Hacisuleyman’s laboratory studies how RNA localization and local translation shape neuronal responses and synaptic plasticity. Her group develops and applies proximity- labeling approaches, transcriptomics, ribosome profiling, proteomics, and advanced imaging to define how activity-dependent RNA regulation remodels the local synaptic proteome and mitochondrial function. Her laboratory has identified genes that are locally translated in response to synaptic activity and uncovered a previously unrecognized translational program involving upstream open reading frames and micropeptides.

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.

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.

Jeffrey Keift, Ph.D., 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.

The research of Yanjun Li, Ph.D., spans the fields of deep learning, AI-driven drug discovery and computational biology, with a particular emphasis on deep generative modeling for de novo molecule design and geometric deep learning for molecular recognition. His work aims to develop innovative AI algorithms to tackle foundational life-science challenges with broad scientific impacts and to optimize and automate real-world drug discovery and design pipelines. Before joining the University of Florida, Dr. Li successively worked as a Senior Research Scientist at the Institute of Deep Learning, Baidu Research USA, Sunnyvale, California, and at Calico Life Sciences, an Alphabet-founded research lab for human aging research.

Lynne 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 in human diseases in 1981 and, subsequently, the exon junction complex 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. Dr. 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.

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.

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.

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 KEYNOTE 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.

Artem Nemudryi is an assistant professor in the University of Florida College of Medicine Biochemistry and Molecular Biology department. His group asks, "How do cells cope with RNA damage?" His team discovered that human cells repair RNA breaks induced by CRISPR ribonucleases and leverage this mechanism to make programmable deletions in the human transcriptome. They use programmable CRISPR ribonucleases to break RNA, study how it is repaired, and develop RNA editing tools with therapeutic potential.

Lien T. Nguyen, Ph.D., identifies and studies pathogenic or functional tandem genetic repeats and repeat expansions. Her research aims to identify novel repeat expansions that contribute to Alzheimer’s disease, ALS and other neurological disorders. It also investigates how novel repeat expansion mutations contribute to these diseases and the involvement of stress in triggering and exacerbating disease. She is also studying if and how tandem repeats regulate gene expression and function of the central nervous system.

The non-coding functions performed by nucleic acids depend on exquisite secondary and tertiary structures. The Passalacqua lab is focused on the discovery and structural characterization of functional nucleic acids. Current topics of research include discovery and characterization of structured non-coding RNAs involved in gene regulation, and host-pathogen/microbe interactions, such as ribozymes, riboswitches, RNA Thermometers and other non-coding RNAs. It also includes deciphering how complex DNA structures in R-loops impact genome stability.

The Pipkin Lab focuses on CD4 and CD8 T cells, and how transcription factors (TFs) and chromatin regulatory factors (CRFs) govern transcriptional programs that establish gene expression which defines distinct effector and memory T cell states, functions and tissue-adaptations. The laboratory developed one of the first first-ever pooled RNA interference (RNAi) mediated approaches in T cells to conduct pooled in vivo phenotypic screens to elucidate genes that control T cell mediated immunity during viral infection. We have applied these and other tools to define the roles of individual factors among entire gene families in CD4 and CD8 T cells as they participate in various immune-mediated processes in vivo.

Dr. Puthanveettil's laboratory investigates how RNA regulation shapes synaptic plasticity, long-term memory storage, and neuronal function. His research has uncovered roles for long noncoding and circular RNAs in regulating local protein synthesis, mitochondrial function, and activity-dependent synaptic remodeling, with a particular interest in how kinesin-mediated transport delivers regulatory RNAs to specific neuronal compartments. By integrating RNA biology with intracellular transport and synapse biology, his laboratory seeks to define fundamental mechanisms of memory storage and understand how their disruption contributes to neurological and neurodegenerative disorders.

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 combined with messenger RNA 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.

The Xie lab's overarching goal is to understand how non-coding RNAs and RNA-binding proteins contribute to gene regulation in human health and disease. Xie's research has expanded an unusual pathway that is distinct from canonical miRNA biogenesis in pre-miRNA synthesis, nuclear-cytoplasmic transport, Dicer processing and guide strand selection. It therefore impacts a broad spectrum of research areas from RNA pol II transcription, RNA processing and export, to studies of miRNA function and RNAi vector development.

Ling Xu, Ph.D., is an assistant professor in the chemistry department at The Wertheim UF Scripps Institute. The Xu lab investigates the structure, dynamics, and regulatory mechanisms of RNA and RNA–protein complexes that govern genome integrity, innate immunity, and human health. The lab combines cryo-electron microscopy (cryo-EM), biochemistry, and cell biology to visualize how RNA molecules fold, assemble with proteins and execute complex cellular functions.