Emily's Entourage banar ny väg med AI-drivet anslag för att identifiera nästa generations leveranssystem för CF-genterapi
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.”
Om Emily's Entourages (EE's) stipendieprogram
Emily’s Enturages (EE) anslagsprogram tillhandahåller finansiering för att påskynda forskning och terapikutveckling för personer med cystisk fibros (CF) som inte har nytta av befintliga mutationsriktade terapier. EE stöder en rad finansieringsmekanismer, inklusive translationella anslag, samarbetsanslag och prekliniska utforskande anslag, såväl som riskkapitalinvesteringar (venture philanthropy).
Hittills har EE delat ut miljoner dollar to tvärvetenskapliga team runt om i världen och hjälpt till att säkra miljoner till i följdfinansiering. För att se beviljade anslag, besök https://www.emilysentourage.org/awarded-grants/. För att lära dig mer om EE:s finansieringsmöjligheter, besök https://www.emilysentourage.org/funding-opportunities/.
Om Emily's Entourage
Emily’s Entourage (EE) är en innovativ 501(c)3-organisation som påskyndar forskningen kring nya behandlingar och ett botemedel för personer i den sista fasen av cystisk fibros (CF) som inte har nytta av de mutationsinriktade behandlingar som finns tillgängliga idag. Sedan 2011 har EE beviljat forskningsanslag på flera miljoner dollar, startat ett företag som bedriver genterapi för cystisk fibros i klinisk fas, utvecklat en global patientdatabas och ett program för att matcha patienter med kliniska prövningar i syfte att påskynda rekryteringen till kliniska prövningar, samt lett globala insatser för att driva fram forskning och läkemedelsutveckling med stor genomslagskraft. Organisationen har uppmärksammats i nationella medier, bland annat i New York Times, STAT, CNN, People och flera andra. Läs mer på emilysentourage.org.
Kontakt med media
Hannah Kleckner
Direktör för marknadsföring och kommunikation
hannah@emilysentourage.org
