Emily’s Entourage Awards $220,000 Research Grant to Dr. Zheng-Rong Lu and Dr. Mitchell Drumm of Case Western Reserve University to Advance Delivery Mechanisms for Gene Therapy to Treat Cystic Fibrosis (CF)

The project is focused on the design and development of multifunctional ionizable lipid nanoparticles (LNP) for safe and efficient delivery of various nucleic acids for gene therapy to potentially treat CF. 

Lower Merion, PA – November 20, 2023 — 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), today announced it has awarded $220,000 in funding to Dr. Zheng-Rong Lu, PhD and Dr. Mitchell Drumm, PhD of Case Western Reserve University. The grant supports research on the design and development of multifunctional ionizable lipid nanoparticles (LNP) for safe and efficient delivery of various nucleic acids for gene therapy.

Mitch Drumm, PhD (left), and Zheng-Rong Lu, PhD (right)
Case Western Reserve University

CF is a rare, inherited genetic disease caused by mutations in a functional gene in the lungs. While 90% of the CF population can be treated with approved CFTR modulator therapies, the remaining 10% of people with CF do not respond to these therapies. Gene therapy holds the potential to normalize the functions of the mutated gene and effectively treat CF, but the challenge lies in safely delivering a copy of a normal gene into cells in the lungs.

The project, titled, “Multifunctional Ionizable Lipid Nanoparticles for Gene Therapy of Cystic Fibrosis,” aims to optimize and develop multifunctional ionizable lipid nanoparticles (LNP) as a platform technology for nonviral gene therapy. 

“This grant represents a significant step forward in our ongoing mission to find effective treatments for all individuals with CF,” said Chandra Ghose, PhD, Chief Scientific Officer of EE. “Gene therapy has the potential to be a game-changer for the final 10% of people with CF who do not respond to current therapies, and we are proud to support Dr. Zheng-Rong Lu and Dr. Mitchell Drumm’s innovative research in this area.”

The research team has shown that their multifunctional ionizable lipids are safe and efficient for delivery of nucleic acids of any size, including siRNA, miRNA, mRNA, and plasmid DNA, via repeated local and systemic administrations in animal models. Through this grant funding, the team will optimize the multifunctional lipids to further improve their safety and efficacy for repeated aerosol delivery of a therapeutic plasmid DNA encoding CFTR gene. Their LNP has demonstrated efficient gene transfection and expression in both in vitro and in vivo settings, showcasing its potential for effective treatment of monogenic disorders in preclinical disease models.

“Gene therapy is a promising therapeutic approach to treat the final 10% of the people with CF that do not respond to currently available pharmacotherapeutics. The challenge for successful gene therapy is the lack of safe and efficient delivery systems for repeated delivery of therapeutic nucleic acids to regulate, replace, or correct the mutated CFTR gene,” said Dr. Drumm.

“We are honored to receive this grant from Emily’s Entourage, which will allow us to further optimize and develop our LNP platform for gene therapy,” said Dr. Lu. “Our goal is to create a safe and efficient delivery system that can be utilized for repeated administrations of gene therapy to effectively treat CF in the final 10% of people with CF.”

In this project, the team will continue to refine and test the optimized LNP to deliver a copy of a normal gene in a preclinical CF model. The success of the project holds the promise of providing a safe and efficient delivery platform for effective gene therapy for the final 10% of people with CF.

EE remains committed to supporting cutting-edge research that leads to accelerated, transformative treatments for the final 10% of the CF population, 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 does 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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