Scientists have achieved a significant advancement in the treatment of respiratory diseases with the development of a new drug delivery system that transports genetic therapies directly to the lungs. This innovation opens up promising possibilities for patients with conditions such as lung cancer and cystic fibrosis.

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

Through testing more than 150 different materials, the researchers discovered a novel nanoparticle capable of safely and effectively delivering messenger RNA and gene-editing tools to lung cells. In preclinical studies using mice, this approach slowed lung cancer growth and improved lung function impaired by cystic fibrosis, a genetic condition caused by a single defective gene.

The team also developed a chemical strategy to synthesize a broad range of lung-targeting lipids, which serve as the foundation for the nanocarriers. These materials can be tailored to deliver therapies to various organs, making the system highly adaptable for future treatments.

This streamlined method of synthesis simplifies the design of next-generation therapies for a wide array of diseases. The results demonstrate the potential of targeted delivery systems in genetic medicine, with the ability to both stimulate immune responses against cancer and restore function in genetic lung disorders, all without causing harmful side effects.

Key contributors to the Nature Communications paper from Oregon State included K. Yu Vlasova, D.K. Sahel, Namratha Turuvekere Vittala Murthy, Milan Gautam, and Antony Jozic. The second study featured work by OSU researchers Murthy, Jonas Renner, Milan Gautam, Emily Bodi, and Antony Jozic alongside Sahay.

The long-term goal of this research is to develop safer, more effective treatments by precisely delivering genetic tools to specific locations in the body. This work marks a major step forward in that mission.

By Impact Lab