Emily’s Entourage concede Bolsa de Pesquisa Translacional de 2026 para Avançar Nova Entrega de Edição Genética para Fibrose Cística

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

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.”

 

Sobre o programa de subsídios do Emily's Entourage (EE)

O Programa de Subsídios da Emily’s Entourage (EE) fornece financiamento para acelerar a pesquisa e o desenvolvimento terapêutico para pessoas com fibrose cística (FC) que não se beneficiam de terapias existentes direcionadas a mutações. O EE apoia uma variedade de mecanismos de financiamento, incluindo subsídios translacionais, subsídios colaborativos e subsídios exploratórios pré-clínicos, bem como investimentos de filantropia de risco.

Até o momento, a EE concedeu milhões de dólares a equipes multidisciplinares em todo o mundo e ajudou a garantir milhões adicionais em financiamento subsequente. Para ver as bolsas concedidas, visite https://www.emilysentourage.org/awarded-grants/. Para saber mais sobre as oportunidades de financiamento da EE, acesse https://www.emilysentourage.org/funding-opportunities/.

 

Sobre a comitiva de Emily

A Emily’s Entourage (EE) é uma organização inovadora sem fins lucrativos, registrada como 501(c)3, que acelera a pesquisa de novos tratamentos e de uma cura para indivíduos na fase final da fibrose cística (FC) que não se beneficiam das terapias direcionadas a mutações disponíveis atualmente. Desde 2011, a EE já concedeu milhões de dólares em subsídios de pesquisa, lançou uma empresa de terapia gênica para FC em fase clínica, desenvolveu o banco de dados de pacientes Clinical Trial Connect (CTC) e o programa de conexão com ensaios clínicos para acelerar o recrutamento global para esses ensaios, além de liderar esforços mundiais para impulsionar pesquisas de alto impacto e o desenvolvimento de medicamentos. A organização tem sido destaque na mídia nacional, incluindo o New York Times, STAT, CNN, People e outros. Saiba mais em emilysentourage.org.

 

Contato com a mídia

Hannah Kleckner

Diretor de Marketing e Comunicações

hannah@emilysentourage.org