Researchers from the University of Ottawa Faculty of Medicine announced on Apr. 17 that they have found a new way to deliver RNA and gene-editing therapies more precisely to specific organs using small extracellular vesicles (sEVs). These tiny, bubble-like structures act as natural messengers in the body, carrying RNA and other molecules between cells.
The study suggests that sEVs could help overcome one of the main challenges in developing next-generation treatments: delivering them safely and accurately to hard-to-reach tissues such as the brain and kidneys. This discovery could open new paths for precision medicine, especially for diseases with limited treatment options.
Dr. Derrick Gibbings, senior author of the study published in Cell Biomaterials, said his team took inspiration from biology by studying how sEVs naturally target specific tissues. "Our approach was to learn from nature-and work with nature – to find sEVs that could deliver to the tissues and cells where there were the most compelling targets for siRNA therapeutics," he said. Gibbings explained that this strategy focuses on matching vesicle origin with intended destination instead of assuming all sEVs behave alike.
The researchers found that certain sEVs could carry siRNA directly to kidneys when injected into mice, reducing symptoms in models of chronic kidney disease. They also showed success delivering treatments into the brain through direct administration into the central nervous system, improving outcomes in a model of neurodegenerative disease. The team observed similar results in larger animal models, suggesting their approach might be scalable for human use.
Gibbings highlighted both progress and ongoing challenges: producing enough sEVs at scale and ensuring long-lasting effects remain obstacles before clinical application can begin. "We have collected a lot of data that shows that sEVs can be effective, safe and scalable delivery vehicles. We are hoping to convince investors or industry partners to work with us to advance these towards clinical trials," he said.
He added particular interest in targeting chronic kidney disease caused by genetic variations in APOL1: "There are a very large number of patients, and they frequently progress to needing transplants and are also dying of this disease." Gibbings described research on extracellular vesicles as an exciting field offering new insights into cell communication—and potential ways to treat complex diseases: "It's kind of like we just discovered that cells use a new media like phones or TikTok for communication... So we're discovering what information and messages they are sharing, and how we can reprogram that to treat disease."
