Emily’s Entourage Breaks New Ground with AI-Powered Grant to Identify Next-Generation CF Gene Therapy Delivery Systems
AI-guided lipid nanoparticle screening aims to overcome longstanding barriers to inhaled CF gene therapy
Emily’s Entourage (EE) has announced its sixth and final 2026 Translational Research Grant awarded to Jacob Witten, PhD, Postdoctoral Associate in the Anderson Lab at the Massachusetts Institute of Technology (MIT).
Lower Merion, PA—September 9, 2026—Emily’s Entourage (EE), an innovative 501(c)(3) organization accelerating research for individuals with cystic fibrosis (CF) who do not benefit from current mutation-targeted therapies, is proud to announce its sixth and final 2026 Translational Research Grant awarded to Jacob Witten, PhD, Postdoctoral Associate in the Anderson Lab at the Massachusetts Institute of Technology (MIT).
Dr. Witten’s project aims to develop improved lipid nanoparticles (LNPs) designed specifically for inhaled gene therapy—addressing a significant barriers to restoring CFTR function in people with CF who do not benefit from current mutation-targeted therapies.
For these individuals—known as the final 10%—restoring CFTR activity through gene therapy or gene editing represents one of the most promising potential treatment strategies. However, decades of clinical trials have shown that safely and effectively delivering genetic therapies to the lungs remains a major challenge.
Lipid nanoparticles have recently emerged as powerful delivery tools for mRNA-based therapies, including COVID-19 vaccines and treatments for genetic diseases. Yet translating this success to the lung has proven difficult due to the airway’s unique biological barriers, including thick mucus, limited cellular uptake, and tight junctions between airway cells.
Dr. Witten’s research seeks to overcome these challenges by designing and screening new LNP formulations optimized specifically for airway delivery. His team will develop biodegradable lipid components to improve safety, use artificial intelligence (AI) to identify promising formulations, and optimize surface chemistry to enhance mucus penetration with efficient cellular uptake.
A key innovation of the project is the use of apical-out airway organoids—advanced laboratory models that closely mimic human airway biology. These models enable rapid, high-throughput screening of large numbers of LNP formulations, accelerating the discovery of delivery systems tailored for the lung.
By combining AI-guided lipid design with physiologically relevant airway models, the team aims to identify LNPs that are both more effective and better tolerated for inhaled delivery. The most promising candidates will then be evaluated in additional translational models, including CF airway cultures and human lung explants.
“One of the greatest challenges in CF gene therapy is getting treatments to the right cells in the lung,” said Dr. Witten. “This work focuses on developing delivery systems designed specifically for the airway, with the aim of enabling more effective inhaled genetic therapies for people who currently lack treatment options.”
For individuals in the final 10%, effective delivery of mRNA or gene editing tools could represent a transformative step toward restoring lung function.
“Solving the airway delivery challenge is essential to unlocking the full potential of nucleotide-based therapies therapy for CF,” said Chandra Ghose, PhD, Chief Scientific Officer at EE. “By supporting innovative strategies that combine AI with advanced airway models, we’re helping accelerate the development of delivery technologies designed specifically for those unable to benefit from current CFTR modulator therapies.”
About Emily’s Entourage’s (EE’s) Grant Program
Emily’s Entourage’s (EE’s) Grant Program provides funding to accelerate research and therapeutic development for people with cystic fibrosis (CF) who do not benefit from existing mutation-targeted therapies. EE supports a range of funding mechanisms, including translational grants, collaborative grants, and preclinical exploratory grants, as well as venture philanthropy investments.
To date, EE has awarded millions of dollars to multi-disciplinary teams around the world and helped secure millions more 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 (EE) 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, EE has awarded millions of dollars in research grants, launched a clinical-stage CF gene therapy company, developed a global patient database 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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