New Symposium Highlights Research on Targeting Incurable Diseases Via RNA

JUPITER, Fla.—Sept. 20, 2019—On Dec. 5, Chemistry Professor Matthew D. Disney, PhD, will host a new symposium aimed at bringing together leading scholars in RNA biology at the Florida campus of Scripps Research. The conference, titled “RNA: From Biology to Drug Discovery.” will feature talks by noted scientists in RNA biophysics, sequence, structure, function, therapeutic targeting and computation.

Basic understanding of RNA biology has accelerated recently as the role of RNAs in disease has become clearer. Approaches to designing effective and selective RNA-focused therapeutic strategies have followed, and are showing great promise, opening diseases once deemed “undruggable” to potential treatment strategies. At Scripps Research in Jupiter, Florida, Professor Disney has pioneered multiple RNA-focused therapeutic approaches and helped launch a company, Expansion Therapeutics, to move them into the clinic.

“It is now known that RNA is a key driver in nearly every disease,” Disney says. “Bringing together leading scholars in this emerging field will make all of us better and improve efforts to find treatments for diseases for which there are no known cures or have a poor prognosis.”

Speakers include Chemistry Professor Dale Boger, PhD, of Scripps Research in La Jolla, Calif.; Chemistry and Biochemistry Professor M.G. Finn, PhD, of Georgia Tech University; Biochemistry and Biophysics Professor Lynne Maquat, PhD, Director of the University of Rochester Medical Center’s Center for RNA Biology; Professor of Biochemistry Robert Batey, PhD, of the University of Colorado, Boulder; Associate Professor of Integrative, Structural & Computational Biology Katrin Karbstein, PhD of Scripps Research in Jupiter, Florida; Assistant Professor of Molecular Genetics & Microbiology Eric Wang, PhD, of University of Florida’s Center for NeuroGenetics; Professor of Biochemistry and Biophysics David Matthews, MD, PhD, of the University of Rochester Medical Center; Mathematical Sciences Professor Emeritus Michael Zuker, PhD, of the Rensselaer Polytechnic Institute in New York, and Professor of Neuroscience Leonard Petrucelli, PhD, of the Mayo Clinic, Jacksonville.

A poster session will take place during the lunch break. All registrants are eligible to present a poster. See the registration portal to submit an abstract:
The universe of human proteins consists of about 20,000 varieties, while the universe of human RNAs is closer to 200,000, potentially offering new effective opportunities to intervene.
While about 2 percent of the human genome encodes proteins, 70 to 80 percent of is transcribed into RNA, potentially offering significantly more druggable targets. Until recently, however, most researchers considered RNAs undruggable.

“I am excited to hear more about where these scientists are taking RNA-focused research,” Disney says.

Symposium: “RNA – From Biology to Drug Discovery”
Dec. 5, 2019
Scripps Research Rodney B. Fink Auditorium, 120 Scripps Way, Bldg. B
8:15 a.m. to 5:15 p.m.
For more information or to submit your poster abstract: Contact Kathleen O’Brien,
To register, go to


Robert Batey, PhD
Department of Biochemistry
The University of Colorado at Boulder
“Targeting RNA by Small Molecules: A Perspective from Nature”

M.G. Finn, PhD
Professor and James A. Carlos Family Chair for Pediatric Technology
Department of Chemistry and Biochemistry
Georgia Tech University
“Using the RNA Inside Virus-Like Particles for Unnatural Purposes”

David Mathews, MD, PhD
Department of Biochemistry and Biophysics; Department of Biostatistics and Computational Biology
University of Rochester Medical Center
“Predicting RNA Structure with Physics and Sequence Comparison”

Eric Wang, PhD
Assistant Professor
Department of Molecular Genetics & Microbiology
Center for NeuroGenetics
University of Florida
“RNA Regulation in Repeat Expansion Diseases”

Dale Boger, Ph.D.
Richard and Alice Cramer Professor of Chemistry
Department of Chemistry & the Skaggs Institute for Chemical Biology
The Scripps Research Institute
“Mechanistic Studies on Natural Products that Bind and Alkylate or Cleave Nucleic Acids”

Katrin Karbstein, PhD
Associate Professor
Department of Integrative, Structural & Computational Biology
Scripps Research
“Quality Control During Ribosome Assembly”

Lynne Maquat, PhD
Department of Biochemistry and Biophysics
Center for RNA Biology
University of Rochester Medical Center
“Nonsense-Mediated mRNA Decay in Health and Disease”

Michael Zuker, PhD
Professor Emeritus
Department of Mathematical Sciences
Rensselaer Polytechnic Institute
“Algorithms for Predicting RNA Secondary Structure: Making the Most of an Ill-Conditioned Problem”

Leonard Petrucelli, PhD
Department of Neuroscience
Mayo Clinic, Jacksonville
“Understanding the link between C9Orf72 FTD/ALS and TDP-43”


Media contact:
Stacey Singer DeLoye
Sr. Communications Manager
Scripps Research
(561) 228-2551