{"id":27109,"date":"2026-09-08T14:12:48","date_gmt":"2026-09-08T18:12:48","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=27109"},"modified":"2026-09-08T14:12:48","modified_gmt":"2026-09-08T18:12:48","slug":"emilys-entourage-awards-2026-translational-research-grant-to-advance-novel-gene-editing-delivery-for-cystic-fibrosis","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/pt\/emilys-entourage-awards-2026-translational-research-grant-to-advance-novel-gene-editing-delivery-for-cystic-fibrosis\/","title":{"rendered":"Emily's Entorage atribui Bolsa de Investiga\u00e7\u00e3o Translacional de 2026 para fazer avan\u00e7ar a administra\u00e7\u00e3o inovadora de edi\u00e7\u00e3o gen\u00e9tica para a fibrose qu\u00edstica"},"content":{"rendered":"

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<\/span><\/i><\/p>\n

\"\"

Emily\u2019s 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.<\/em><\/p><\/div>\n

Lower Merion, PA\u2014September 8, 2026\u2014<\/span>Emily\u2019s 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.<\/span><\/p>\n

Dr. Lu\u2019s project aims to develop a next-generation delivery platform for gene editing therapies\u2014addressing 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%.<\/span><\/p>\n

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%\u00a0 given its potential to address the root cause of disease and provide lasting therapeutic benefit for all CF mutations.<\/span><\/p>\n

Dr. Lu\u2019s research focuses on overcoming this barrier through the development of multifunctional ionizable lipid nanoparticles (LNPs)\u2014engineered 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.<\/span><\/p>\n

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.<\/span><\/p>\n

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.<\/span><\/p>\n

Using this platform, Dr. Lu\u2019s 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.<\/span><\/p>\n

\u201cGene editing has the potential to fundamentally change how we treat CF, but only if we can deliver these therapies safely and effectively,\u201d said Dr. Lu. \u201cThis work is focused on developing a delivery system that can make that possible, particularly for people with CF who currently have no treatment options.\u201d<\/span><\/p><\/blockquote>\n

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.<\/span><\/p>\n

\u201cThis project takes on one of the most critical challenges in the field: delivery,\u201d said Chandra Ghose, PhD, Chief Scientific Officer at EE. \u201cBy doing so, it has the potential to help unlock gene editing as a real therapeutic pathway\u2014not just in theory, but in practice\u2014for people with CF, including those in the final 10% who are still waiting for a treatment that addresses the root cause of their disease.\u201d<\/span><\/p>\n

 <\/p><\/blockquote>\n

Sobre o programa de subs\u00eddios da Emily's Entourage (EE)<\/b><\/p>\n

O Programa de Subs\u00eddios da Emily\u2019s Entourage (EE) financia a acelera\u00e7\u00e3o da investiga\u00e7\u00e3o e do desenvolvimento terap\u00eautico para pessoas com fibrose qu\u00edstica (FQ) que n\u00e3o beneficiam de terapias existentes direcionadas para muta\u00e7\u00f5es. A EE apoia uma variedade de mecanismos de financiamento, incluindo subs\u00eddios de transla\u00e7\u00e3o, subs\u00eddios colaborativos e subs\u00eddios explorat\u00f3rios pr\u00e9-cl\u00ednicos, bem como investimentos de filantropia de risco.<\/span><\/p>\n

At\u00e9 \u00e0 data, a EE atribuiu milh\u00f5es de d\u00f3lares a equipas multidisciplinares em todo o mundo e ajudou a garantir milh\u00f5es adicionais em financiamento subsequente. Para ver os subs\u00eddios atribu\u00eddos, visite <\/span>https:\/\/www.emilysentourage.org\/awarded-grants\/<\/span><\/a>. Para saber mais sobre as oportunidades de financiamento da EE, visite <\/span>https:\/\/www.emilysentourage.org\/funding-opportunities\/<\/span><\/a>.<\/span><\/p>\n

 <\/p>\n

Sobre a comitiva de Emily<\/b><\/p>\n

A Emily\u2019s Entourage (EE) \u00e9 uma organiza\u00e7\u00e3o inovadora com estatuto 501(c)3 que acelera a investiga\u00e7\u00e3o de novos tratamentos e de uma cura para os doentes na fase final da fibrose c\u00edstica (FC) que n\u00e3o beneficiam das terapias direcionadas para muta\u00e7\u00f5es atualmente dispon\u00edveis. Desde 2011, a EE j\u00e1 concedeu milh\u00f5es de d\u00f3lares em bolsas de investiga\u00e7\u00e3o, lan\u00e7ou uma empresa de terapia gen\u00e9tica para a FC em fase cl\u00ednica, desenvolveu a base de dados de doentes \u00abClinical Trial Connect\u00bb (CTC) e o programa de correspond\u00eancia de ensaios cl\u00ednicos para acelerar o recrutamento global para ensaios cl\u00ednicos, e liderou esfor\u00e7os a n\u00edvel mundial para impulsionar a investiga\u00e7\u00e3o de alto impacto e o desenvolvimento de medicamentos. A organiza\u00e7\u00e3o tem sido destaque nos meios de comunica\u00e7\u00e3o nacionais, incluindo o New York Times, a STAT, a CNN, a People e outros. Saiba mais em <\/span>emilysentourage.org<\/span><\/a>.<\/span><\/p>\n

 <\/p>\n

Contacto para a imprensa<\/b><\/p>\n

Hannah Kleckner<\/span><\/p>\n

Diretor de Marketing e Comunica\u00e7\u00e3o<\/span><\/p>\n

hannah@emilysentourage.org<\/span><\/p>\n



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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\u2014September 8, 2026\u2014Emily\u2019s 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… <\/p>\n

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