Paul McCray Jr., a professor of pediatrics-pulmonary medicine at the University of Iowa Carver College of Medicine, was the 2024 recipient of the prestigious Trailblazer Award presented by Emily’s Entourage, a nonprofit supporting funding and awareness for rare mutations of cystic fibrosis.

According to the Cystic Fibrosis Foundation, there are close to 40,000 children and adults living with cystic fibrosis in the U.S.

Cystic fibrosis is a genetic disease caused by a mutation in the cystic fibrosis transmembrane conductance regulator, also known as CFTR.

The disease causes mucus to clog airways in the lungs and prevent the release of enzymes and nutrients in the pancreas. People with cystic fibrosis are more susceptible to infections, inflammation, and respiratory failure and often suffer from malnutrition and poor growth.

​​Emily’s Entourage focuses on the 10 percent of cystic fibrosis patients who do not respond to new medications due to gene variants.

McCray received the Trailblazer Award on Sept. 27 at the North American Cystic Fibrosis Conference in Boston.

McCray has been working on gene therapy for cystic fibrosis since the early 1990s, he said, and got involved with Emily’s Entourage in 2015. His work focuses on improving gene delivery systems to respiratory epithelial cells.

“Most of my research has been focused on CF — either trying to understand basic mechanisms of the disease, like how does loss of function of this particular gene result in the illness and then how to treat it,” he said.

Emily’s Entourage co-founder Emily Kramer-Golinkoff wrote in an email to The Daily Iowan that the nonprofit first met McCray when he attended a key opinion leaders meeting in September 2015.

“It was the famous meeting of the minds that sparked what later became Spirovant, the CF gene therapy company that EE seed funded and launched and in which Dr. McCray is an academic co-founder,” she wrote.

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McCray also received a research grant from Emily’s Entourage for $858,932 in May 2024 in collaboration with researchers at the Children’s Hospital of Philadelphia and the University of Rochester. The project McCray is exploring develops and tests a new group of viral vectors to deliver genetic cargoes to the linings of the lungs or respiratory epithelial cells, he said.

Emily’s Entourage gave out the Trailblazer Award for the first time in 2017.

“The Trailblazer Award is incredibly close to our hearts,” Kramer-Golinkoff wrote. “We are so lucky to work with such brilliant, committed, passionate researchers who care so deeply and so personally about accelerating lifesaving research and drug development for those in the final 10 percent of the CF community that do not benefit from existing CFTR modulators. This award is a small way to celebrate those researchers.”

Chief Scientific Officer at Emily’s Entourage Chandrabali Ghose wrote to the DI that McCray’s work has significantly advanced the frontiers of cystic fibrosis research.

“Dr. McCray’s receipt of the Emily’s Entourage Trailblazer Award is a testament to his groundbreaking approach to advancing therapeutics for the CF community, with particular emphasis on the [people with CF] who do not benefit from currently approved modulators,” she wrote.

Ghose also wrote that McCray is a committed mentor, physician, and lifelong advocate for individuals with cystic fibrosis and their families.

McCray expressed gratitude for receiving the prestigious award.

“It’s just incredibly gratifying to have people recognize me and the team of people that have worked with me for the years on this,” he said. “It’s really motivating to myself and the people that I work with to get this kind of recognition.”