U of T Engineering Lab Partners with Moderna to Develop RNA-Based Tools to Treat and Prevent Disease

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A team of researchers from the University of Toronto’s School of Applied Sciences and Engineering has partnered with biotech company Moderna to develop next-generation RNA platform technologies.

This strategic research agreement between industry and university is the first academic partnership under the collaborative partnership framework agreement between U of T and Moderna.

“We are creating new types of nanotechnology and RNA to help prevent and cure disease. Together, we are driving new technological innovations to give patients even more options for highly effective RNA-based medicines,” he says. Omar F Khanassistant professor at the Institute of Biomedical Engineering who leads the U of T research team and holds the Canada Research Chair in Nucleic Acid Therapeutics.

“Moderna has an incredible track record of taking research from the idea to the clinic. This partnership is a great opportunity for us to reach our collective goal of efficiently engaging the body to treat and prevent disease.”

As evidenced by the success of its COVID-19 vaccine, Moderna is an established name in messenger RNA (mRNA) science. Now, Moderna and Khan’s lab group is harnessing fundamental knowledge from chemistry, engineering, biology, and immunology to design new types of RNAs and their safe and effective delivery to the body.

“We believe that mRNA is a platform that could significantly improve the way treatments and vaccines are discovered, developed and produced,” he says. Shehzad IqbalNational Medical Director of Moderna Canada.

“It is critical that the next generation of mRNA drugs be fully controllable; we need both the understanding and the ability to optimize delivery systems and their payloads to maximize the benefits of mRNA drugs while minimizing unwanted side effects.”

Ribonucleic acid, commonly known as RNA, is a nucleic acid in the same chemical family as DNA and is found naturally in the body. While DNA codes for all human genes, RNA is involved in the expression and regulation of those genes, including their translation into proteins. Certain viruses also use RNA as genomic material, including SARS-CoV-2.

Delivering custom RNA sequences to the body could offer a way to inhibit undesirable processes and stimulate beneficial ones. For example, researchers could use RNA constructs to block the biochemical processes that allow cancerous tumors to grow and metastasize, or to help the body’s immune system fight infection.

RNA-based therapies have the potential to treat many diseases, from diabetes to cancer to musculoskeletal diseases, through targeted approaches that target the biochemical pathways that exploit those diseases. RNA molecules could be used to combat genetic diseases, either by silencing some genes or enhancing the expression of others, all without genome editing or the use of small molecule drugs.

Despite this potential, RNA is a comparatively fragile molecule. To do its job, it must be delivered in a package that protects the material from damage and retains its potency as it is delivered and stored around the world. The packaging also allows cells to take in the RNA sequence and read its instructions.

The new partnership will design both those delivery vehicles and the custom RNA sequences they will contain.

“On the nanotechnology side, we are working on delivering molecules,” says Khan. “On the RNA side, we are working with mRNA, which people are familiar with. We are also going beyond mRNA to create new and advanced technology that can prevent and treat disease.”

Khan brings an effective blend of academic and industry experience to the Modern partnership. His research was recently supported by Medicine by Design’s Pivotal Experiment Fund, a program that supports a preclinical line of regenerative medicine-based therapies that have strong potential for clinical and/or commercial impact.

“Khan’s lab has a wealth of experience identifying ‘what comes next,’ and Moderna brings significant manufacturing and development experience to help bring to life the next big project Dr. Khan and his team are dreaming of,” he says. Iqbal.

“Working with Moderna, a leader in RNA medicine that has helped countless lives, is a wonderful opportunity for all of my students,” adds Khan. “My team can showcase their innovative talent and work toward our common goal of using science and engineering to improve the health of people around the world. The impact on global health is very tangible.”

“Together, we look to the future and envision the true value of nanotechnology and RNA in its many forms.”

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