Sixth Annual RNA Medicine Symposium Celebrates Real World Successes

The 6th annual RNA Medicine Symposium — sponsored by the HMS Initiative for RNA Medicine (HIRM) and the Cancer Center at Beth Israel Deaconess Medical Center (BIMDC) — was held online due to the COVID-19 pandemic. Since the last Symposium, however, two RNA-based vaccines were developed, tested and deployed against the virus. The real-world successes of the relatively new field of investigation lent the virtual proceedings — attended by more than 1,000 participants logging in from around the world — a celebratory feel.

"At the first RNA medicine symposium back in 2015, we celebrated a large amount of exciting pre-clinical data that pointed to a very bright future for RNA medicine," said Frank J. Slack, PhD, Director of the Harvard Medical School Initiative for RNA Medicine and Director of BIDMC's Cancer Research Institute and the Shields Warren Mallinckrodt Professor of Medical Research Departments of Pathology and Medicine at BIDMC and Harvard Medical School. "This year, we've witnessed the roll out of the first RNA-based vaccines. RNA medicine is now preventing and curing diseases and we are so fortunate to have some of the pioneers in those areas speaking at our symposium today."

"None of us envisioned that 2020 would entail combating a virus of such magnitude, ramping down our research labs, transforming our homes into offices and reimagining the way we teach and work, and I am incredibly proud the way HIRM has navigated these stormy waters," said George Q. Daley, MD, PhD, Dean of the Faculty of Medicine at Harvard Medical School. "Many of you steered your research endeavors toward understanding, treating and preventing SARS-CoV-2 through the possibilities offered by RNA medicine, and I thank you for your efforts — both in the COVID-19 realm and throughout RNA discovery. I can't wait to hear more about what you've accomplished and what lies just beyond the horizon."

Jeffrey E. Saffitz, MD, PhD, Chair of the Department of Pathology at BIDMC, noted that the seeds for a visionary RNA medicine program were planted in 2012 when it became apparent to him that advances in this area would transform the way we understand diagnose and treat human disease.

"Dr. Slack has done a spectacular job developing a world-class multi-institute, multi-disciplinary program, both within HMS and the larger Boston academic and biotech community," said Saffitz, who is also the Mallinckrodt Professor of Pathology at HMS. "We are clearly at a defining moment in RNA medicine and the future is very exciting."

Noting that BIDMC is delighted to serve as HIRM's home base, Gyongyi Szabo, MD, PhD, Chief Academic Officer at BIDMC and Beth Israel Lahey Health and Professor of Medicine and Faculty Dean for Academic Affairs at HMS, added that the Initiative is currently home to 54 faculty members from 11 schools and hospitals and works with 35 industry partners. "Under Dr. Slack, HIRM created the first comprehensive precision medicine core facility in the country to enable the translation of basic science discovery into potential clinical applications," she added. "Undoubtedly, this is place where collaboration and discovery can flourish with the support of core facilities and expert scientists."

Szabo, who is also a member of the HIRM faculty, presented on her research focusing on microRNAs in both alcoholic and non-alcoholic fatty liver disease — two increasingly common conditions in industrialized nations and beyond with no long-term treatments or cure. Since 2012, Szabo and her team have been untangling the relationships among micro RNAs, alcohol, and inflammation. Szabo's findings suggest that manipulating the expression of specific micro RNAs could mitigate inflammation — the central cause of most of the cellular damage in fatty liver disease — and open the door to potential therapeutic targets.

A second representative from BIDMC, Ioannis Vlachos, PhD, Co-Director of the Bioinformatics Program of the Cancer Research Institute at BIDMC and an Assistant Professor in Department of Pathology presented on a project he and colleagues across the Harvard ecosystem, MIT and the Broad Institute pivoted to as the COVID-19 crisis hit the northeast in the U.S. last spring.

Using tissues from individuals who succumbed to COVID-19 and were cared for at BIDMC, Brigham and Women's Hospital, and Massachusetts General Hospital, Vlachos and colleagues including HIRM members Szabo and Winston Hide, PhD, Co-Director of the Non-Coding RNA Precision Diagnostics and Therapeutics Core Facility at BIDMC, used single-cell and spatial RNA sequencing to investigate how COVID-19 manifests at the cellular level across tissues. With samples taken from different organs—including lung, heart, liver, and kidney—they created a comprehensive "cell atlas" capturing in high granularity SARS-CoV-2 pathology and revealed potential cellular targets.

The team did so using cutting-edge spatial technologies that allowed them to quantify gene expression from single cells or directly in the tissue. "This is a new scalpel that can redirect the way we approach research in health and disease," said Vlachos, who is also Director of the Spatial Technology and Bioinformatics Units, Precision RNA Medicine Core at HIRM, and an Associate Member, Broad Institute of MIT and Harvard.

Additional presenters included Melissa J. Moore, PhD, Chief Scientific Officer, Platform Research, Moderna, Inc; Katalin Karikó, PhD, Senior Vice President, BioNTech SE; Elsa Flores, PhD, Chair, Department of Molecular Oncology, Leader of the Cancer Biology and Evolution Program, H. Lee Moffitt Cancer Center and Research Institute; Brett P. Monia, PhD, Chief Executive Officer, Ioannis Pharmaceuticals, Inc.; Harry F. Noller, PhD, Sinsheimer Professor of Molecular Biology and Director of the Center for Molecular Biology of RNA in the Department of MCD Biology at University of California, Santa Cruz; Daniel G. Anderson, PhD, Professor, Department of Chemical Engineering, Institute for Medical Engineering and Science, Harvard-MIT Division of Health Sciences and Technology, David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology; Sohail Tavazoie, MD, PhD, Leon Hess Professor & HHMI Faculty Scholar, Meyer Laboratory of Systems Cancer Biology, Directors, Black Family Metastasis Center, The Rockefeller University; Tracy Johnson, PhD, Professor, Department of Molecular, Cell and Developmental Biology, Dean, Division of Life Sciences, University of California, Los Angeles; Andrea Ventura, MD, PhD, Member, Cancer Biology and Genetics Program, Memorial Sloan Kettering Cancer Center; Victoria D'Souza, PhD, Professor of Molecular and Cellular Biology, Harvard University; and Carl D. Novina, MD, PhD, Associate Professor of Medicine, Dana-Farber Cancer Institute, Harvard Medical School, Associate Member, Broad Institute of Harvard and MIT.

For more information about the HIRM, please visit the HMS Initiative for RNA Medicine page.