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Meet the Penn engineer who delivers mRNA inside human cells

Tom Avril, The Philadelphia Inquirer on

Published in Science & Technology News

It’s believed that only 2% to 5% of all the LNP gets out of the endosomes into the cell to translate the mRNA therapeutic.

We made a new LNP in a way that mimics a space shuttle. You can think of a space shuttle that has rocket boosters that break off over time. These lipid nanoparticles have lipids on the side that almost mimic what happens in the space shuttle. Those lipids can degrade and fall off over time.

As these tails are degrading and falling off, we believe that it’s helping to disrupt the endosomes to release the RNA cargo inside the cell more efficiently.

To test it, we fed mice a high-fat diet, then we injected them with this very potent LNP that can induce the secretion of lots of a weight-loss drug. They dramatically lost weight and returned to normal body weight levels.

Those LNPs traveled to the liver. What about targeting other parts of the body?

The RNA technology is now fairly mature. It’s now a delivery challenge.

 

How do we get RNA to other parts of the body? How do we get into the brain? How do we get into the lungs for cystic fibrosis and lung cancer?

My lab has bioengineers, materials scientists, and chemists who are working on this.

Tell me about that study where you got mRNA into the lungs of mice.

We wanted something that has a positive charge, which would cause the particles to travel to the lungs. But we wanted something that degrades as well. If it’s chopped up in the body, it would be safe.

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