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Developing Breakthrough RNA Therapeutics

By interweaving nucleic acid scientists with clinicians dedicated to finding new cures, our goal is to create a new paradigm for organizing molecular research that enables the rapid application of new biological discoveries to solutions for unmet challenges in human health.

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RTI Spotlight

BRIDGE Innovation and Business Development at UMass Chan Medical School has committed nearly $2 million in funding this year for six UMass Chan Medical School investigator-led research projects that hold promise for translation to clinical application and commercialization. Dr. Li Li, assistant professor of RNA Therapeutics, was selected as a recipient for his project, Developing nucleoside-modified circular mRNAs as a durable and non-immunogenic platform for mRNA therapeutics.  Read more»

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2025 RNA Therapeutics: From Concept to Clinic


Save the dates
for our 7th annual RNA Therapeutics Symposium, June 25-27, 2025!


Abstract submission
is CLOSED.

Onsite registration is sold-out. Please email us if you would like to be placed on the waitlist.

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ScienceLIVE Educational Outreach Program

ScienceLIVE is an educational science outreach program for Worcester area middle schools. We provide opportunities for students to engage with our diverse postdoctoral and graduate student trainees through interactive, exciting virtual and hands-on STEM activities.

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Support Our Next Breakthrough

For decades, scientists at UMass Chan have been pioneers in RNA biology and leading innovators in the development of information-based therapeutics: cutting-edge therapeutic tools that leverage our understanding of the human genome in ways that are revolutionizing how we treat disease. With your support, we are poised to unleash their power, and change the world for the better.

 

Anastasia Khvorova wins the Else Kröner Fresenius Prize for Medical Research

This year's Else Kröner Fresenius Prize for Medical Research goes to RNA researcher Anastasia Khvorova, PhD. The Else Kröner Fresenius Foundation is awarding the 2.5 million euro prize in recognition of Khvorova's pioneering work in the field of RNA-based therapies. Her research has contributed significantly to the development of new approaches for treating genetic and neurodegenerative diseases.

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UMass Chan scientists receive $2.3 million from Rett Syndrome Research Trust for research

Erik Sontheimer, PhD, the Pillar Chair in Biomedical Research and professor of RNA therapeutics; Jonathan Watts, PhD, professor of RNA therapeutics; and Scot Wolfe, PhD, professor of molecular, cell & cancer biology, were awarded $2.3 million in new funding from the Rett Syndrome Research Trust to utilize base-editing and prime-editing technologies to correct mutations in MECP2.

Current treatments for Rett syndrome symptoms are palliative, but gene therapy clinical trials aimed at addressing the disease’s underlying causes are underway.  

“I am cautiously optimistic there’s a good chance that these gene therapy approaches are going to be successful at making some difference. The editing approaches we’re working on have the chance to change disease outcomes even more profoundly, and perhaps enable patients to get much closer to normalcy, especially if the disease can be caught earlier,” Dr. Watts said.  

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