Scholar Spotlight: Transforming Cystic Fibrosis treatment with RNA-based therapeutics

May 15, 2025

Cystic fibrosis is a life-limiting genetic disease that affects the lungs and other organs, caused by mutations in the CFTR gene that impair the production of a critical protein. While advances in treatment have improved outcomes for some patients, many still face limited options. 

As a 2025 Oxford-Harrington Rare Disease Scholar, Dr Bowen Li is working on an innovative approach aimed at the root cause of the disease. In this Q&A, he discusses his research into RNA-based therapies that could potentially expand therapeutic pathways for patients. 

What inspired you to focus your research on rare diseases, and how did you come to specialise in RNA therapies? 

My interest in rare diseases stems from their profound unmet medical needs. As a scientist, I’m drawn to challenges where innovation can significantly improve lives.

Dr Bowen Li: I began focusing on nucleic acid therapies during my doctoral studies at MIT under the supervision of Profs Daniel Anderson and Bob Langer, where I was focused on developing nonviral delivery systems for RNA medicines such as mRNA and CRISPR-Cas9 to address diseases at the genetic level. This therapeutic approach aligns perfectly with the needs of rare disease populations, offering hope for conditions that lack effective treatments. 

Tell us about your awarded project and its potential impact on patients. 

B.L: My awarded project focuses on developing RNA-based therapies for cystic fibrosis, a congenital lung disease caused by genetic mutations that result in CFTR protein dysfunction. By leveraging lipid nanoparticles as delivery vehicles, we aim to deliver therapeutic RNA molecules that can overcome these genetic mutations, enabling patients to restore the production of functional CFTR proteins. 

This innovative approach has the potential to transform treatment by addressing the root cause of cystic fibrosis, significantly improving quality of life and clinical outcomes, especially for patients who currently have limited therapeutic options.

How does the Oxford-Harrington Rare Disease Centre, OHC, model help advance your work? 

B.L: The OHC’s unique model of combining funding with therapeutic development support is instrumental in advancing our research. Their expertise helps us navigate critical preclinical challenges, such as optimising lipid nanoparticle formulations, improving therapeutic RNA stability, and validating efficacy in relevant disease models.  

Additionally, their support fosters collaboration with experts in regulatory science and translational research, which is essential for designing robust preclinical studies and identifying key milestones for eventual clinical translation.  

While clinical application remains a long-term goal, the OHC’s structured guidance ensures we remain on a strategic path, focusing on rigorous and scalable solutions for cystic fibrosis treatment. 

What excites you most about your therapeutic approach? 

B.L: What excites me most is the potential of lipid nanoparticles to precisely deliver RNA therapies to affected cells. This platform is versatile and can be tailored for other rare diseases as well, making it a foundational technology for future innovations. 

What role do collaborations play in advancing your project? 

Academia brings innovation, industry provides scale, and patients shape the priorities. Collaboration ensures we stay grounded in real-world impact.

B.L: Collaborations are crucial for success. Academia brings innovation, industry provides resources for scaling, and patient organisations offer invaluable insights into the real-world needs of those we aim to serve. By working together, we can ensure the therapies we develop are not only scientifically sound but also patient-centred and accessible. 

Where do you see your project in the next 2–5 years? 

B.L: In the next 2-5 years, my focus will be on completing rigorous testing in rodent models and exploring opportunities to validate our therapy in large animal models. These steps are critical to building the foundation for eventual clinical translation. I am determined to drive our research closer to the bedside, bridging the gap between laboratory discoveries and tangible therapies for patients. 

About Dr Bowen Li 

Dr Bowen Li is Assistant Professor at the University of Toronto and Canada Research Chair in RNA Vaccines & Therapeutics. His research focuses on lipid nanoparticle-based RNA delivery technologies for treating genetic diseases, with a focus on cystic fibrosis.