Emily's Entourage apre nuove strade con un finanziamento basato sull'IA per identificare sistemi di rilascio di terapia genica per la fibrosi cistica di nuova generazione
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.”
Informazioni sul programma di sovvenzioni di Emily's Entourage (EE)
Il programma di sovvenzioni di Emily's Entourage (EE) fornisce finanziamenti per accelerare la ricerca e lo sviluppo terapeutico per le persone affette da fibrosi cistica (FC) che non traggono beneficio dalle terapie esistenti mirate alle mutazioni. EE sostiene una serie di meccanismi di finanziamento, tra cui sovvenzioni traslazionali, sovvenzioni collaborative e sovvenzioni esplorative precliniche, oltre a investimenti di venture philanthropy.
Ad oggi, EE ha assegnato milioni di dollari a team multidisciplinari in tutto il mondo e ha contribuito a garantirne altri milioni in finanziamenti successivi. Per visualizzare i sussidi assegnati, visitare https://www.emilysentourage.org/awarded-grants/. Per saperne di più sulle opportunità di finanziamento di EE, visitate il sito https://www.emilysentourage.org/funding-opportunities/.
Informazioni su Emily's Entourage
Emily’s Entourage (EE) è un’organizzazione innovativa senza scopo di lucro (501(c)3) che accelera la ricerca di nuovi trattamenti e di una cura per i pazienti affetti da fibrosi cistica (FC) che si trovano nella fase finale della malattia (10%) e che non traggono beneficio dalle terapie mirate alle mutazioni attualmente disponibili. Dal 2011, EE ha stanziato milioni di dollari in sovvenzioni per la ricerca, ha avviato un’azienda specializzata nella terapia genica per la FC in fase clinica, ha sviluppato un database globale dei pazienti e un programma di abbinamento alle sperimentazioni cliniche per accelerare il reclutamento dei partecipanti, e ha guidato gli sforzi a livello mondiale per promuovere la ricerca ad alto impatto e lo sviluppo di farmaci. L’organizzazione è stata citata dai media nazionali, tra cui il New York Times, STAT, CNN, People e altri. Per saperne di più, visita il sito emilysentourage.org.
Contatto con i media
Hannah Kleckner
Direttore marketing e comunicazione
hannah@emilysentourage.org
