Emily's Entourage assegna il Translational Research Grant 2026 per promuovere un nuovo sistema di somministrazione dell'editing genetico per la fibrosi cistica
Novel lipid nanoparticle platform aims to safely and efficiently deliver gene editors to correct CFTR mutations, bringing the field closer to a durable treatment for cystic fibrosis
Emily’s Entourage (EE) has awarded it’s fifth 2026 Translational Research Grant awarded to Zheng-Rong Lu, PhD, M. Frank Rudy and Margaret Domiter Rudy Professor of Biomedical Engineering at Case Western Reserve University.
Lower Merion, PA—September 8, 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 it’s fifth 2026 Translational Research Grant awarded to Zheng-Rong Lu, PhD, M. Frank Rudy and Margaret Domiter Rudy Professor of Biomedical Engineering at Case Western Reserve University.
Dr. Lu’s project aims to develop a next-generation delivery platform for gene editing therapies—addressing one of the most significant barriers in the field and helping unlock the potential of gene editing as a durable treatment approach for people with CF, including those in the final 10%.
Gene editing has long held promise as a transformative approach to CF treatment, with the potential to correct the underlying genetic mutation and restore normal CFTR function. However, safely and efficiently delivering gene editors into the appropriate cells in the lungs has posed challenges that have limited its progress. Solving this challenge is especially important for the final 10% given its potential to address the root cause of disease and provide lasting therapeutic benefit for all CF mutations.
Dr. Lu’s research focuses on overcoming this barrier through the development of multifunctional ionizable lipid nanoparticles (LNPs)—engineered delivery vehicles designed to transport gene editing tools directly into cells. While LNPs are already used safely in applications such as mRNA vaccines, existing formulations are not optimized to deliver protein-based gene editors for diseases like CF.
This project introduces a new class of biodegradable LNPs specifically designed to address these limitations. Unlike DNA- or mRNA-based approaches, this platform delivers gene editing machinery in the form of ribonucleoproteins (RNPs), which do not require expression of foreign genetic material and may reduce safety concerns associated with prolonged or uncontrolled gene activity.
The newly developed nanoparticles are designed to be stable in aqueous environments, avoid the use of harsh organic solvents, and degrade into biocompatible components in the body. Early studies suggest these LNPs can achieve more efficient intracellular delivery and gene editing at lower doses, while demonstrating improved safety profiles compared to earlier systems.
Using this platform, Dr. Lu’s team will optimize delivery of gene editors targeting CFTR mutations, with an initial focus on the W1282X mutation in preclinical models that are available at Case Western Reserve University. The project will evaluate both gene editing efficiency and restoration of CFTR function, with the goal of identifying a lead therapeutic candidate for further translational development. By enabling more efficient delivery of gene editors, this approach could move the field closer to treatments that correct CF at its source.
“Gene editing has the potential to fundamentally change how we treat CF, but only if we can deliver these therapies safely and effectively,” said Dr. Lu. “This work is focused on developing a delivery system that can make that possible, particularly for people with CF who currently have no treatment options.”
Approximately 10% of people with CF have genetic mutations that are ineligible for existing CFTR modulators. EE is committed to accelerating research and therapeutic development for all individuals who do not benefit from these therapies, ensuring that no one is left behind.
“This project takes on one of the most critical challenges in the field: delivery,” said Chandra Ghose, PhD, Chief Scientific Officer at EE. “By doing so, it has the potential to help unlock gene editing as a real therapeutic pathway—not just in theory, but in practice—for people with CF, including those in the final 10% who are still waiting for a treatment that addresses the root cause of their disease.”
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 il database dei pazienti Clinical Trial Connect (CTC) e il programma di abbinamento alle sperimentazioni cliniche per accelerare il reclutamento a livello globale per le sperimentazioni cliniche, e ha guidato gli sforzi mondiali volti a 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
