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RNA: the Medicine of the Future
The RNA Therapeutics initiative at the University of Michigan is pioneering the future of personalized medicine by developing cutting-edge RNA-based treatments. Harnessing the power of Antisense Oligonucleotides (ASOs), we aim to target and correct specific genetic mutations responsible for a variety of diseases. Our mission goes beyond scientific discovery; we are committed to addressing wealth disparities by making personalized RNA-based therapies accessible and affordable for all, ensuring no one is left behind. Led by a team of world-class researchers, we are transforming groundbreaking discoveries into real-world therapies that offer new hope and improved outcomes for patients around the globe.
Interested in partnering on a project or case?
Contact us at info.rnatx@umich.edu for more information.
Michelle Hastings, Ph.D.
RNA Therapeutics Director
As the Pfizer Upjohn Research Professor of Pharmacology, Dr. Hastings brings over two decades of expertise in the design and discovery of RNA therapeutics, particularly Antisense Oligonucleotides (ASOs). Her work aims to bridge foundational RNA research discoveries and the development of medicines to treat genetic, infectious, and other diseases, creating a seamless “bench to bedside” pipeline. Dr. Hastings has been instrumental in easing bottlenecks in the journey from lab to clinic by navigating the complexities of clinical trials and intellectual property. Under her leadership, the initiative is fostering a synergistic community of researchers and establishing an in-house RNA production system, enabling rapid, cost-effective development of personalized therapies. Dr. Hastings’ work has already made significant strides, such as developing an ASO-based treatment for juvenile Batten disease, and she is committed to empowering collaborations that advance RNA therapeutics into real-world applications, ultimately transforming the landscape of personalized medicine.
Peter Todd, M.D., Ph.D.
RNA Therapeutics Clinical Director
Dr. Peter Todd, the clinical director of the RNA Therapeutics initiative, also plays a pivotal role in advancing the mission. Dr. Todd is dedicated to developing novel therapeutics for patients with repeat expansion disorders and understanding how nucleotide repeats contribute to human disease. His research focuses on how instability and expansion of specific nucleotide repeats lead to neurological disease, with a view to developing innovative treatments. With a background as a physician scientist, Dr. Todd embodies the “bench-to-bedside” approach, combining fundamental research with clinical application to benefit patients. His work includes studying the role of repetitive elements in gene function and disease, using advanced sequencing and bioinformatic approaches to uncover new therapeutic targets. Dr. Todd’s leadership and collaborative spirit are key to driving the initiative’s success and fostering the growth of future scientific leaders.
Katelyn Lacy, Ph.D.
RNA Therapeutics Managing Director
Katelyn received her B.S. from the University of Michigan in Cellular and Molecular Biology and went on to earn her Ph.D. in Molecular Biophysics at UT Southwestern Medical Center. Her thesis work in mechanistic studies of RNA modification enzymes led her to join the Core Antisense Research Group at Ionis Pharmaceuticals. There she studied fundamental aspects of antisense oligonucleotide (ASO) function, from both the drug (ASOs) and the target (RNA) angles including the characterization of how naturally occurring RNA modifications affect ASO activity and evaluating structure activity relationships (SAR) of ASO chemical modifications on oligo activity and selectivity. She also investigated other modalities of RNA therapeutics evaluating them for further development. Katelyn has joined the UM RNA Therapeutics team as the Director of Operations and will facilitate the development of the program and support the design and optimization of ASO medicines.
ASOs are powerful tools use to correct gene expression
Antisense oligonucleotides (ASOs or AONs) are chemically modified, single-stranded nucleic acids that can target and fix genetic issues at their root. They work by binding to RNA—the genetic instructions for making proteins—and either turning its activity up or down, depending on the disease being treated. Currently, 11 ASO-based treatments have been approved by the FDA, with many more in clinical trials. These therapies are used to treat conditions affecting the nervous system, metabolism, and heart. Because of their versatility and safety record, ASOs are also the designated platform by the FDA for treating ultra-rare genetic diseases. ASO therapies are particularly effective for conditions with a known genetic basis, and have been effectively delivered in the liver, kidney, brain, or eyes. However, ongoing advancements are pushing the boundaries, making it possible to target more diseases and tissues in the future.
Find below a collection of articles and publications authored or featuring the work of our directors
U-M RNA Therapeutics Director Michelle Hastings, Ph.D., utilizes ASOs in breakthrough treatment for young twins suffering from rare form of Batten disease
In a remarkable display of bench to bedside science, a therapeutic developed by Michigan Medicine’s Michelle Hastings, Ph.D., is now being used to treat twin girls with a rare form of juvenile Batten disease.
Hastings, who is the Pfizer Upjohn Research Professor of Pharmacology, director of RNA Therapeutics at U-M’s Center for RNA Biomedicine, has been working on the drug platform, called antisense oligonucleotides, for nearly twenty years.
Hastings explains it can be used to “fix” genes with specific mutations, like the CLN3 mutation responsible for the girls’ disease.
Click here to read more.
Publications
- Michaels WE, Pena-Rasgado C, Kotaria R, Bridges RJ, Hastings ML. Open reading frame correction using splice-switching antisense oligonucleotides for the treatment of cystic fibrosis. Proc Natl Acad Sci U S A. 2022 Jan 18;119(3).
- Centa JL, Jodelka FM, Hinrich AJ, Johnson TB, Ochaba J, Jackson M, Duelli DM, Weimer JM, Rigo F, Hastings ML. Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease. Nat Med. 2020 Sep;26(9):1444-1451.
- Wang L, Kempton JB, Jiang H, Jodelka FM, Brigande AM, Dumont RA, Rigo F, Lentz JJ, Hastings ML, Brigande JV. Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction. Nucleic Acids Res. 2020 May 21;48(9):5065-5080.
- Hinrich, A.J., Jodelka, F.M., Chang, J.L., Brutman, D., Bruno, A.M., Briggs, C.A., James, B.D.,Stutzmann, G.E., Bennett, D.A., Miller, S.A., Rigo, F., Marr, R.A., and Hastings, M.L. (2016)Therapeutic correction of ApoER2 splicing in Alzheimer’s disease mice using antisense oligonucleotides. EMBO Mol. Med. 8: 328-345. PMCID: 4818756.
- Vijayakumar, S., Depreux, F., Jodelka, F.M., Lentz, J.J., Rigo, F., Jones, T.A., Hastings, M.L. (2017) Rescue of peripheral vestibular function in Usher syndrome mice using a splice-switching antisense oligonucleotide. Hum. Mol. Genet. 1093/hmg/ddx234. PMCID: 5886244.
- A native function for RAN translation and CGG repeats in regulating Fragile X protein synthesis Rodriguez CM#, Wright SE#, Kearse MG, Haenfler JM, Flores BN, Liu Y, Ifrim MF, Glineburg MR, Krans A, Jafar-Nejad P, Sutton MA, Bassell GJ, , Parent JM, Rigo F, Barmada SJ, Todd PK. Nature Neuroscience, 2020 Mar;23(3):386-397.