Emily’s Entourage ouvre de nouvelles voies avec une subvention propulsée par l'IA pour identifier des systèmes de délivrance de thérapie génique de nouvelle génération pour la FK
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.”
À propos du programme de subventions de l'Entourage d'Emily (EE)
Le programme de subventions d'Emily's Entourage (EE) fournit des financements pour accélérer la recherche et le développement thérapeutique pour les personnes atteintes de fibrose kystique (FK) qui ne bénéficient pas des thérapies ciblées sur les mutations existantes. EE soutient une gamme de mécanismes de financement, notamment des subventions translationnelles, des subventions collaboratives et des subventions exploratoires précliniques, ainsi que des investissements en capital-risque philanthropique.
À ce jour, EE a octroyé des millions de dollars à des équipes pluridisciplinaires à travers le monde et a aidé à obtenir des millions de plus en financements complémentaires. Pour consulter les subventions accordées, visitez https://www.emilysentourage.org/awarded-grants/. Pour en savoir plus sur les possibilités de financement de l'EE, consultez le site suivant https://www.emilysentourage.org/funding-opportunities/.
À propos de l'entourage d'Emily
Emily’s Entourage (EE) est une organisation innovante de type 501(c)3 qui accélère la recherche de nouveaux traitements et d’un remède pour les personnes atteintes de mucoviscidose (CF) qui ne tirent aucun bénéfice des thérapies ciblant les mutations actuellement disponibles. Depuis 2011, EE a octroyé des millions de dollars de subventions de recherche, lancé une société spécialisée dans la thérapie génique de la mucoviscidose au stade clinique, développé une base de données mondiale de patients et un programme de mise en relation pour les essais cliniques afin d’accélérer le recrutement des participants, et mené des initiatives à l’échelle mondiale pour favoriser la recherche à fort impact et le développement de médicaments. L’organisation a fait l’objet de reportages dans les médias nationaux, notamment le New York Times, STAT, CNN, People et bien d’autres. Pour en savoir plus, rendez-vous sur emilysentourage.org.
Contact presse
Hannah Kleckner
Directeur du marketing et de la communication
hannah@emilysentourage.org
