RNA: From Biology to Drug Discovery 2025
Explore the therapeutic potential of RNA with thought leaders in the field.

“Dr. Darnell developed methods to investigate how RNA binding proteins modulate gene expression. He discovered neuron-specific RNA-binding proteins in the mammalian brain, and studies their link to intellectual function and human disease…The lab has expanded its work to consider RNA regulation over time, and apply this to the study of autoimmune disease (rheumatoid arthritis) and infectious disease including COVID-19.” Dr. Darnell is the Robert and Harriet Heilbrunn Professor, Laboratory of Molecular Neuro-Oncology, The Rockefeller University; Howard Hughes Medical Institute.

“Guttman’s integrated team of experimental and computational biologists work together to understand how nuclear compartments control gene expression programs and cell state decisions. Guttman’s team has explored the long non-coding RNA Xist, which plays a crucial role in X chromosome inactivation. They discovered critical proteins like SHARP are involved in X chromosome silencing…Guttman Lab’s SPRITE method has opened the door to discovery-driven proximity interactions.” Guttman is Professor of Biology, Division of Biology and Biological Engineering, California Institute of Technology.

“Adrian Krainer’s lab studies the mechanisms of RNA splicing, ways in which they go awry in cancer and genetic diseases, and the means by which faulty splicing can be corrected. For example, the Krainer lab found a way to correct spinal muscular atrophy, a neuromuscular disease that is a leading genetic cause of death in infants by stimulating SMN protein production through the introduction of antisense oligonucleotides.” Krainer is St. Giles Foundation Professor and Cancer Center Program Co-Leader, Cold Spring Harbor Laboratory.

“The majority of the human genome is transcribed to non-protein-coding RNA (ncRNA), which is often considered as the “dark matter” or junk material. However, ncRNAs play diverse regulatory roles and represent critical elements that make humans highly complex and distinct from other animals. The brain, the most complex of human organs, is characterized by the extreme number and diversity of ncRNA species. Our Laboratory investigates various classes of ncRNA, such as microRNA, lncRNA, and enhancer RNA, involved in the pathogenesis of malignant brain tumors and neurodegenerative diseases.” Krichevsky is Professor of Neurology, Brigham and Women’s Hospital, Harvard Medical School and a Co-Director of HMS initiative for RNA Medicine.

“Dr. Manoharan is an organic chemist who transforms complex chemistry into medicines for people with serious illnesses. As the first chemist to join Alnylam in 2003, Dr. Manoharan has been instrumental in developing cell-penetrating, tissue-specific methods to deliver small interfering RNAs designed to silence disease-causing genes.” Manoharan is SVP, Innovation Chemistry, Alnylam Pharmaceuticals.

“Research in my lab focuses on RNA decay pathways. One pathway, called nonsense-mediated mRNA decay (NMD) or mRNA surveillance, surveys all newly synthesized mRNAs during what we call a "pioneer" round of translation…The Maquat lab defined the ‘50-55-nucleotide rule.’ Generally, if translation terminates more than 50-55 nucleotides upstream of an exon-exon junction that is marked by a splicing-dependent ‘mark’, then the mRNA will be subject to NMD.” Maquat is the J. Lowell Orbison Endowed Chair and Professor of Biochemistry & Biophysics, founding Director of the Center for RNA Biology, University of Rochester; Member of the National Academy of Sciences and the National Academy of Medicine.

“Our research bridges computational and experimental sciences to discover new regulatory aspects in the human genome: Specifically, how long noncodng RNA (lncRNA) genes regulate numerous biological processes… The Rinn lab has identified dozens of lncRNA genes that play key roles in diverse cellular functions, fertility, development and viability. ” Prof. Rinn is Leslie Orgel Professor of RNA Science, University of Colorado, Boulder.

Elias Sayour, M.D., PhD, is Associate Professor of Neurosurgery and Pediatrics at University of Florida Health, as well as Principal Investigator of the RNA Engineering Laboratory within the Preston A. Wells, Jr. Center for Brain Tumor Therapy and UF Brain Tumor Immunotherapy Program, and Co-Leader of the UFHCC Cancer Therapeutics & Host Response program. He is working to develop lipid-nanoparticles to train the immune system to fight cancer.