RNA is found in the cells of all living organisms and is essential to biological function, and understanding it more fully could aid in everything from producing diagnostics to detecting diseases to contributing to treatments.

Mello discussed his work to harness RNA technology to create therapeutics for liver diseases. He referred more than once to RNA, his research focus for the past several decades, as “truly, truly amazing.” During a break, he found himself surrounded by attendees eager to keep the conversation going.

As Mello spoke, two fellow scientists intrigued by the possibilities of RNA science exchanged knowing looks: Dr. Mukesh Jain, senior vice president for health affairs and dean of medicine and biological sciences at Brown, and Juan Alfonzo, executive director of the Giuliani RNA Center. Both are leaders in advancing RNA research, and Jain spoke frankly about the importance of translating scientific findings into impact.

“We're here not just to share discoveries, but to spark collaborations that I hope will shape the future of RNA science and medicine,” Jain said. “As many of you know, the study of RNA holds enormous promise, not only for advancing our understanding of fundamental biology and disease pathogenesis, but also for novel diagnostics and therapeutics — and the potential impact of RNA extends to many sectors of life, from agriculture and food security to bio-manufacturing and bio-defense.”

The promise of RNA science

Nearly every seat in the medical school’s largest auditorium was filled for the symposium, which — like the Giuliani RNA Center itself — brought together scientists, faculty members, graduate students and scholars from a variety of disciplines, including molecular biology, biochemistry, computational biology, engineering, medicine, mathematicians and more.

Attendees heard from pioneering researchers like Mello as well as a range of Brown faculty, including Sergej Djuranovic, whose discoveries about RNA metabolism and gene regulation pathways are informing the development of biotechnological tools and potential therapeutics; Theresa Raimondo, who is researching delivery systems for RNA-based therapeutics such as vaccines and cancer immunotherapies; and Weihan Li, whose lab is using advanced single-molecule imaging techniques to study the precise localization of mRNAs and proteins and how their mispositioning can lead to disease.

The biopharmaceutical industry was well-represented by leaders and scientists from companies including Alnylam Pharmaceuticals, Flagship Pioneering, PTC Therapeutics and Moderna Therapeutics, among others. U.S. Sen. Jack Reed offered remarks that emphasized the importance of investing in RNA technology. 

During a panel on the challenges of RNA in science and medicine, the speakers acknowledged the need for cutting-edge tools to study RNA, that the ways to get RNA-based medicines to all the places in the body and brain where it needs to go haven’t yet been perfected, and that government funding can’t be taken for granted. However, their comments trended positive, prompting the moderator to joke that the RNA outlook sounds rather “rosy.”

“Ten years ago, there were only a few companies working on this — now you ask who is not working on this,” said Vasant Jadhav, chief technology officer for Alnylam Pharmaceuticals, which has partnered with Mello on developing therapeutics based on his RNA research. “That means there will be lot of discoveries and innovation.”

Entrepreneurs and researchers alike discussed how the two groups can continue to work together: university-based researchers can “think broadly” and identify drug targets and their roles in different diseases as well as varied applications of RNA discoveries, whereas pharmaceutical companies can specialize in focusing on specific goals like realizing an application of a major discovery.

Alfonzo cited RNA’s biggest challenge as one reason he and others feel so compelled by its potential.

“RNA is unpredictable,” Alfonzo said. “The one thing that I can tell you is predictable about RNA is that it's complicated to know everything about it, and we’re never going to get there in all of our lifetimes. But it’s super exciting because of the promise that it has.”