New research core removes barriers to RNA-based research and drug discovery efforts at U-M

The Nucleic Acids Therapeutics (NATx) Core opened at the University of Michigan Life Sciences Institute this week, expanding opportunities for investigators across the university and around the world to access and discover nucleic acid–based therapies and research tools.

Nucleic acids — DNA and RNA — provide promising platforms for developing both new treatments to address unmet medical needs in the clinic and tools to investigate challenging scientific questions in the lab. 

The field of nucleic acids–based drug discovery is fairly nascent but rapidly growing, with fewer than 30 RNA-based therapies receiving FDA approval since their first introduction in 1998, and most of those coming in the past ten years. Recent advances, including the rapid development and deployment of antisense oligonucleotides and gene editing medicines for ultra-rare genetic diseases, have drawn new attention to the power of RNA-based therapies.

“Nucleic acids as research tools and therapeutics are enabling rapid advancement in science and medicine, we want to provide a service to help these tools reach their full potential,” explained NATx Core Faculty Director Michelle Hastings, Ph.D. “With this new core, U-M is now one of the only places where researchers can source these types of molecules at the academic level at an accessible price point.”

The core offers comprehensive services to both university and external partners that has not been traditionally available through commercial services, including the design, synthesis and early testing of custom-made oligonucleotides. Beyond molecule production and testing, the core also provides consultations and hands-on scientific support to guide experimental design and help investigators identify new strategies and guide experimental design for using nucleic acids to answer important scientific and disease-related questions.

“Our goal is to be an end-to-end resource that lowers the barriers — both cost barriers and knowledge barriers — to nucleic acid research for the U-M biomedical community,” said Katelyn Lacy, Ph.D., operations manager of the NATx Core. “Our approach allows for lower costs and greater support in terms of working with investigators on oligo design, synthesis of custom modifications and iterative optimization of candidate molecules.”

The core is already fielding both clinical and discovery science projects, including a drug development campaign targeting a rare disease that has been found in current U-M patients.

The LSI is home to a suite of four other research cores and facilities that support drug discovery and basic science projects from across campus — including the Center for Chemical Genomics, the Center for Structural Biology, the Natural Products Discovery Core, and the Cryo-EM Facility — as well as the campuswide Michigan Drug Discovery program.

This co-location with Michigan Drug Discovery and other LSI cores, as well as the proximity to both central campus and the medical campus, will enable NATx staff to accommodate more users from across U-M, as well as non-profit, government, and industry researchers, Lacy said.

“Having this resource embedded within U-M will enable more research with real impacts on research and ultimately patients,” added Hastings, who is also the Pfizer Upjohn Research Professor of Pharmacology in the U-M Medical School and a professor of medicinal chemistry in the College of Pharmacy. “I think it's going to be a gamechanger in terms of advancing the whole field of nucleic acids research.”