Emily’s Entourage Awards Translational Research Grant to Accelerate Prime Editing for Hard-to-Treat Cystic Fibrosis Mutations
The newly funded project will harness high-throughput gene editing and computational modeling to develop tailored therapies for people with cystic fibrosis unable to benefit from currently available mutation-targeted therapies.
David R. Liu, PhD, a world-renowned gene editing expert and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of MIT and Harvard.
Lower Merion, PA—June 24, 2025—Emily’s Entourage (EE), an innovative 501(c)(3) organization accelerating research for individuals with cystic fibrosis (CF) who do not benefit from existing CFTR modulator therapies, has awarded a 2025 Translational Research Grant to David R. Liu, PhD, a world-renowned gene editing expert and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of MIT and Harvard.
This grant supports Dr. Liu’s groundbreaking project to develop prime editing strategies—an advanced gene editing technology—to permanently correct a broad range of CF-causing mutations that cannot currently be treated by approved CFTR modulator therapies.
While CFTR modulators have transformed care for many with CF, nearly 10% of people with CF cannot benefit from these therapies due to their specific genetic mutations or due to side effects from or suboptimal responses to currently approved modulators. For these individuals, there is a dire need for innovative approaches that address the root cause of their disease. Dr. Liu’s work offers new hope for this group with urgent unmet medical needs.
Prime editing is a breakthrough CRISPR-based “find-and-replace” technology that can precisely correct the genetic mutations responsible for CF—potentially providing a one-time, permanent treatment that restores lung function and quality of life.
Dr. Liu’s team recently demonstrated that prime editing could fix the most common CF mutation, F508del, in lung cells donated by people with CF, restoring function to levels comparable to or higher than current modulator therapies. Building on this breakthrough, the new project aims to develop personalized prime editing solutions for approximately 100 different modulator-ineligible CF mutations, accelerating the path to personalized gene editing treatments.
To achieve this, Dr. Liu’s team will use spooled lentiviral screening to test thousands of candidate prime editing strategies in parallel, rapidly identifying the most effective approaches for each mutation. They will also develop computational models to predict and prioritize these strategies, speeding progress toward personalized treatments. Finally, they will validate the best techniques in airway cells from people with CF to confirm restored production of functional CFTR protein.
“Our lentiviral screening approach, combined with computational modeling, enables rapid optimization of prime editing strategies for many mutations—a process that previously required years of painstaking work,” said Dr. Liu. “This project aims to develop highly personalized gene editing therapies, tailored to the unique genetic makeup of each patient. By harnessing the power of precision medicine, we hope to deliver one-time, life-changing treatments for those who have not benefited from existing options.”
“While modulators have transformed the lives of many with CF, too many people with CF still face the harsh reality of having no effective treatments simply by virtue of their genetic mutations,” said Chandra Ghose, PhD, Chief Scientific Officer at EE. “By investing in this groundbreaking gene editing research, we are fiercely committed to closing that gap—pushing the boundaries of science to bring hope, healing, and ultimately fast breakthroughs and a cure to the most underserved and overlooked members of our community, who are waiting desperately with each passing day.”
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 clinical-stage CF gene therapy company, developed the Clinical Trial Connect (CTC) patient database and a 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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