Emily’s Entourage Awards $858,932 in Inaugural Collaborative Research Grant for Novel Approach to Targeted Airway Delivery in Cystic Fibrosis Treatments

Through the newly-awarded funding from EE, research teams from the University of Iowa, CHOP, and the University of Rochester seek to overcome existing limitations in currently available cystic fibrosis therapies by leveraging advanced novel adeno-associated virus (AAV) capsid variants.

From Top to Bottom: Paul McCray, Jr., MD; Beverly Davidson, PhD; John Lueck, PhD

Lower Merion, PA – May 29, 2024 — Emily’s Entourage (EE), an innovative 501(c)3 that accelerates research for new treatments and a cure for the final 10% of people with cystic fibrosis (CF) who do not benefit from existing CFTR modulators, today announced it has awarded $858,932 in Collaborative Grant funding to Paul McCray, Jr., MD, professor of pediatrics-pulmonary medicine at the University of Iowa; Beverly Davidson, PhD, director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and chief scientific strategy officer Children’s Hospital of Philadelphia (CHOP), professor of pathology and laboratory medicine at the Perelman School of Medicine at the University of Pennsylvania; and John Lueck, PhD, associate professor of pharmacology and physiology at the University of Rochester School of Medicine and Dentistry. The award is the first of EE’s Collaborative Grant program, a newly-launched funding initiative to accelerate groundbreaking CF treatment research by bringing together multiple investigators with diverse areas of expertise to jointly tackle challenges in the development of novel CF therapeutics.

CF is a fatal genetic disease caused by mutations in the CFTR gene that affects the lungs, digestive system, and other organ systems. Despite significant advancements in CFTR modulator therapies, approximately 10% of individuals with CF still lack effective treatment options due to specific mutations, intolerance, or suboptimal response to existing medications.

Led by Dr. Paul McCray — together with Beverly Davidson, PhD, and John Lueck, PhD — the funded project, “Enhanced Airway Transduction and Therapeutic Cargo Delivery with Novel Adeno-associated Virus (AAV) Capsid Variants,” aims to advance novel CF therapeutic development by overcoming known barriers to effective treatments through enhanced therapeutic cargo delivery.

The research team aims to enhance the delivery of emerging technologies that restore CFTR function in people with CF. By using AAV capsid variants to address challenges like poor transduction efficiency, this approach has the potential to offer curative outcomes for the 10% of people with CF who currently lack effective treatments.

“We are honored to receive this generous grant from Emily’s Entourage, which will propel our research efforts towards developing more efficient and targeted treatments for cystic fibrosis,” said Dr. McCray. “Our research aims to address the critical unmet need in CF treatment by improving how therapeutics are delivered to airway epithelial cells — an important step in advancing treatments for the final 10% of people with CF.”

The project’s two-fold objectives include finalizing the capsid screen to identify the most efficient variants for cargo delivery to human airway epithelia and assessing the efficacy of leading capsids for therapeutic cargo delivery. By harnessing cutting-edge technologies and interdisciplinary collaboration, the research team is poised to advance this exciting science with the goal of developing a first-of-its kind targeted therapeutic option for individuals living with CF.

“Emily’s Entourage is proud to support innovative research initiatives that explore new approaches to treating people with CF with the goal of reaching 100% of the community,” said Chandra Ghose, chief scientific officer at Emily’s Entourage. “This inaugural award through our new Collaborative Grant program marks a significant milestone, underscoring our commitment to accelerating CF treatment breakthroughs and expediting the development of life-changing therapies for all individuals affected by this disease.”

EE remains committed to supporting cutting-edge research that leads to accelerated, transformative treatments for the final 10% of the CF population that do not benefit from existing CFTR modulators, ensuring that no one is left behind.

About Emily’s Entourage’s (EE’s) Grant Program 

Emily’s Entourage’s (EE’s) Grant Program provides grant funding to accelerate research and therapeutic development for people with cystic fibrosis (CF) who do not benefit from existing mutation-targeted therapies. EE provides funding through several grant funding mechanisms, including translational grants, collaborative grants, and preclinical exploratory grants, as well as through venture philanthropy investments.

To date, EE has awarded millions of dollars to multi-disciplinary teams around the world and helped to secure millions of dollars in follow-on funding. To view awarded grants, visit https://www.emilysentourage.org/awarded-grants/. To learn more about EE’s funding opportunities, visit https://www.emilysentourage.org/funding-opportunities/.

About Emily’s Entourage

Emily’s Entourage is an innovative 501(c)3 that accelerates research for new treatments and a cure for individuals in the final 10% of the cystic fibrosis (CF) population that do not benefit from currently available mutation-targeted therapies. Since 2011, Emily’s Entourage has awarded millions of dollars in research grants, launched a now-acquired CF gene therapy company, developed a patient registry and clinical trial matchmaking program to accelerate clinical trial recruitment, and led worldwide efforts to drive high-impact research and drug development. The organization has been featured in national media, including the New York Times, STAT, CNN, People, and more. Learn more at emilysentourage.org.

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