University of Toronto 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 Faculty of Applied Science and Engineering has partnered with biotech company Moderna to develop next-generation RNA platform technologies.

This industry-university strategic research agreement is the first university partnership under the U of T’s Collaborative Partnership Framework Agreement with Moderna.

“We are making new kinds 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,” 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 for taking research from idea to clinic. This partnership is a great opportunity for us to achieve our collective goal of effectively engaging the body to treat and prevent disease.

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

“We believe that mRNA is a platform that could dramatically improve the way treatments and vaccines are discovered, developed and produced,” says Chehzad IqbalCountry Medical Director at Moderna Canada.

“It is essential that the next generation of mRNA-based drugs be fully controllable – we need both the understanding and the ability to optimize delivery systems and their payloads to maximize drug benefits at mRNA base while minimizing unwanted side effects.”

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

Delivering personalized RNA sequences into the body could offer a way to inhibit unwanted processes and stimulate beneficial ones. For example, researchers could use RNA constructs to block 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 focus on the biochemical pathways exploited by these diseases. RNA molecules could be used to fight genetic diseases, either by silencing certain genes or enhancing the expression of others, all without editing the genome or using small molecule drugs.

Despite this potential, RNA is a relatively fragile molecule. To do its job, it must come in a package that protects the material from damage and preserves its potency when shipped and stored around the world. The packaging also allows cells to pick up the RNA sequence and read its instructions.

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

“On the nanotechnology side, we are working on carrier molecules,” says Khan. “On the RNA side, we work 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 industrial experience to the Moderna partnership. His research was recently supported by Medicine by Design’s Pivotal Experiment Fund, a program that supports a preclinical pipeline of regenerative medicine-based therapies that have strong potential for clinical and/or commercial impact.

“The Khan Lab has deep expertise in identifying ‘what comes next’ and Moderna brings significant manufacturing and development experience to help realize the next big thing that Dr. Khan and his team dream of,” says Iqbal. .

“Working with Moderna, a leader in RNA medicine that has helped countless lives, is a wonderful opportunity for all of my interns,” adds Khan. “My team can showcase their talent for innovation and work towards 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 foresee the true value of nanotechnology and RNA in its many forms.”

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