Emily's Entourage inova com subsídio impulsionado por IA para identificar sistemas de entrega de terapia gênica para fibrose cística de próxima geração
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.”
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 com status 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 um banco de dados global de pacientes e um programa de correspondência para ensaios clínicos a fim de acelerar o recrutamento 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
