Particle Technology Delivers Medicines and Vaccines

Basic research leads to advancements in medical treatments.

+ More

By Marlene Cimons, National Science Foundation

When Joseph DeSimone met Bill Gates about 18 months ago at a conference in California, he knew he had about ten seconds to engage him. So he told him, right away, how his new company was using computer industry manufacturing techniques to develop innovative vaccines and medicines.

“Obviously, he knew something about the computer industry, and he knew something about vaccines,” says DeSimone, a professor of chemistry at the University of North Carolina at Chapel Hill and chemical engineering at North Carolina State University. “That ten-second hello turned into a 30-minute conversation.”

Ultimately, that 30-minute conversation turned into a $10 million investment last March from the global health philanthropy, the Bill & Melinda Gates Foundation, the first of its kind from a foundation to a for-profit biotech company. The money is part of a Gates Foundation initiative that commits $400 million to promote the foundation’s work, in this case supporting the development of new and more effective vaccines and drugs.

The company, Liquidia Technologies, founded in 2004, is using an innovative approach, by bringing semiconductors to medicine with a process invented by DeSimone’s lab. PRINT®, which stands for Particle Replication in Non-wetting Templates, is a particle-molding technology that uses imprint soft lithography and a “liquid Teflon” substance to produce nano- and micron scale particles with a controlled size and shape.

The basic research to develop the technology was funded by the National Science Foundation Science and Technology Center that DeSimone co-directed at the University of North Carolina at Chapel Hill and North Carolina State University from 1999 to 2009. In recent years, however, as the lab’s research has focused on drugs and vaccines, the National Institutes of Health, specifically the National Cancer Institute, has provided major support.

The PRINT process, at its essence, is simply a molding technology that takes place on films that are processed in a roll-to-roll machine similar in many ways to the photographic film industry of yesteryear. PRINT creates particles within structures that resemble the cavities in ice cube trays or muffin pans but on the nanoscale.

“We brought for the first time to medicine the precision and uniformity of the semiconductor industry into the medical world,” DeSimone says.

Scientists now are engineering these exquisitely small particles to deliver more potent and less toxic vaccines and medicines, including chemotherapy, as well as developing synthetic blood, agents to treat respiratory disorders, such as asthma and cystic fibrosis, and diseases of the brain and nervous system. The particles also could prove valuable in diagnostics, for example, by using them to deliver imaging agents, such as dyes, contrast or radio-markers.

The fact that researchers can control the size and uniformity of the tiny particles could make a significant difference in their efficacy. For example, current powdered compounds that are inhaled often have an imprecise and irregular shape, which can affect dosage size and penetration.

“We’re making particles--delivery vehicles--that are extremely uniform,” DeSimone says. “They have all the precision and uniformity of transistors. We started thinking about using these particles for applications in biology. We became enamored with it. And we decided to launch a company.”

Liquidia began its first Phase I (safety) clinical trial last year for its experimental influenza vaccine, and hopes to move into new vaccine research areas soon, including for malaria, cancer and pneumococcol pneumonia. Last February the company announced it would collaborate with the PATH Malaria Vaccine Initiative (MVI), an international nonprofit global health organization, to study the use of PRINT particle technology in the next generation of malaria vaccines.

DeSimone’s research involves creating a hybrid of the earlier generations of vaccines that will be safer and better able to stimulate the immune system. The first vaccines commonly used were “live attenuated” vaccines, which contain live but weakened versions of the microbe. Later vaccines used specific pieces of the microbes--subunits--to provoke an immune response.