A team of researchers from the University of Toronto’s Faculty of Applied Sciences and Engineering has partnered with biotechnology company Moderna to develop next-generation RNA platform technologies.
This strategic industry-university research agreement is the first academic partnership under the framework collaboration agreement between U of T and Moderna.
“We are making 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,” says Omar F. Khan, assistant professor at the ‘Institute of Biomedical Engineering who leads the research team at the University of T. holds the Canada Research Chair in Nucleic Acid Therapeutics.
“Moderna has an incredible track record of bringing research from the idea to the clinic. This partnership is a great opportunity to achieve 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 leveraging fundamental knowledge of chemistry, engineering, biology and immunology to engineer new types of RNA and their safe and effective delivery into the body.
“We believe mRNA is a platform that could significantly improve the way treatments and vaccines are discovered, developed and produced,” says Shehzad Iqbal, Country Medical Director of Moderna Canada.
“It is critical that the next generation of mRNA medicines be fully controllable: we need the understanding and ability to optimize delivery systems and their payloads to maximize the benefits of mRNA medicine while minimizing side effects unwanted”.
Ribonucleic acid, commonly known as RNA, is a nucleic acid in the same chemical family as DNA and occurs 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 genomic material, including SARS-CoV-2.
Delivering customized 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 biochemical processes that allow cancer tumors to grow and metastasize, or to help the body’s immune system fight infections.
RNA-based therapeutics have the potential to treat many diseases, from diabetes to cancer to musculoskeletal diseases, through targeted approaches that focus on the biochemical pathways that these diseases exploit. RNA molecules could be used to fight 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 relatively fragile molecule. To do its job, it must be delivered in a package that protects the material from damage and preserves its potency as it is delivered and stored around the world. Packaging also allows cells to take 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’re working on delivery molecules,” says Khan. “Next to RNA, we’re working with mRNA, which people are familiar with. We’re also going beyond mRNA to create new and advanced technology that can prevent and treat disease.”
Khan brings an effective combination of academic and industry experience to the Moderna partnership. His research was recently supported by Medicine by Design’s Pivotal Experimentation Fund, a program that supports a preclinical portfolio of regenerative medicine-based therapies that have strong potential for clinical and/or commercial impact.
“The Khan lab has a wealth of experience in identifying ‘what’s next,’ and Moderna brings significant manufacturing and development experience to help realize the next big thing that Dr. Khan and his team they are dreaming,” says Iqbal.
“Working with Moderna, a leader in RNA medicine that has helped countless lives, is a wonderful opportunity for all of my trainees,” adds Khan. “My team can showcase their innovative talent and work toward our mutual goal of using science and engineering to improve the health of people around the world. The impact on global health is very tangible.”
“Together, let’s look ahead and envision the true value of nanotechnology and RNA in its many forms.”