Scientists Develop Targeted Genetic Treatment

Researchers have developed a lung-targeted nanoparticle delivery system for genetic therapies.

In a major step forward for respiratory medicine, scientists have developed a new way to deliver genetic therapies directly to the lungs. This innovative system could open the door to powerful new treatments for conditions like lung cancer and cystic fibrosis.

The research was led by Gaurav Sahay at Oregon State University’s College of Pharmacy, in partnership with Oregon Health & Science University and the University of Helsinki. The findings were recently published in Nature Communications and the Journal of the American Chemical Society.

To create this new approach, scientists tested more than 150 materials before discovering a novel type of nanoparticle. This tiny carrier is designed to safely and efficiently transport messenger RNA (mRNA) and gene-editing tools directly to cells in the lungs. In lab tests with mice, the treatment slowed the growth of lung tumors and improved lung function that had been impaired by cystic fibrosis, a genetic disorder caused by a single faulty gene.

Customizable Lipid Carriers

Researchers also developed a chemical strategy to build a broad library of lung-targeting lipids used in the nanocarriers. These materials form the foundation for the new drug delivery system and could be customized to reach different organs in the body, Sahay said.

“The streamlined synthesis method makes it easier to design future therapies for a wide range of diseases,” he said. “These results demonstrate the power of targeted delivery for genetic medicines. We were able to both activate the immune system to fight cancer and restore function in a genetic lung disease, without harmful side effects.”

Oregon State’s K. Yu Vlasova, D.K. Sahel, Namratha Turuvekere Vittala Murthy, Milan Gautam, and Antony Jozic were co-authors of the Nature Communications paper, which also included scientists from OHSU and the University of Helsinki. OSU’s Murthy, Jonas Renner, Milan Gautam, Emily Bodi, and Antony Jozic teamed with Sahay on the other study.

“Our long-term goal is to create safer, more effective treatments by delivering the right genetic tools to the right place,” said Sahay. “This is a major step in that direction.”

References: “Synthesis of ionizable lipopolymers using split-Ugi reaction for pulmonary delivery of various size RNAs and gene editing” by K. Yu. Vlasova, A. Kerr, N. D. Pennock, A. Jozic, D. K. Sahel, M. Gautam, N. T. V. Murthy, A. Roberts, M. W. Ali, K. D. MacDonald, J. M. Walker, R. Luxenhofer and G. Sahay, 29 April 2025, Nature Communications.
DOI: 10.1038/s41467-025-59136-z

“Synthesis of Ionizable Lipids for Gene Delivery to the Lung Using an Ugi Four Component Reaction” by Jonas Renner, Namratha Turuvekere Vittala Murthy, Milan Gautam, Emily Bodi, Antony Jozic and Gaurav Sahay, 8 May 2025, Journal of the American Chemical Society.
DOI: 10.1021/jacs.5c04123

These studies were funded by the Cystic Fibrosis Foundation, the National Cancer Institute, and the National Heart, Lung and Blood Institute.

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