Emily’s Entourage abre nuevos caminos con una subvención impulsada por inteligencia artificial para identificar sistemas de administración de terapia génica para la fibrosis quística de próxima generación
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.”
Acerca del Programa de Subvenciones de Emily's Entourage (EE)
El Programa de Subvenciones de Emily’s Entourage (EE) proporciona financiación para acelerar la investigación y el desarrollo terapéutico para personas con fibrosis quística (FQ) que no se benefician de las terapias existentes dirigidas a mutaciones. EE apoya una variedad de mecanismos de financiación, incluidas subvenciones translacionales, subvenciones colaborativas y subvenciones exploratorias preclínicas, así como inversiones de filantropía de riesgo.
Hasta la fecha, EE ha otorgado millones de dólares a equipos multidisciplinarios de todo el mundo y ha ayudado a asegurar millones más en financiación posterior. Para ver las subvenciones concedidas, visite https://www.emilysentourage.org/awarded-grants/. Para saber más sobre las oportunidades de financiación de EE, visite https://www.emilysentourage.org/funding-opportunities/.
Acerca del séquito de Emily
Emily’s Entourage (EE) es una innovadora organización sin ánimo de lucro 501(c)3 que impulsa la investigación de nuevos tratamientos y una cura para las personas que se encuentran en la fase final de la fibrosis quística (FQ) y que no se benefician de las terapias dirigidas a mutaciones disponibles en la actualidad. Desde 2011, EE ha concedido millones de dólares en subvenciones de investigación, ha puesto en marcha una empresa de terapia génica para la FC en fase clínica, ha desarrollado una base de datos global de pacientes y un programa de selección de ensayos clínicos para acelerar el reclutamiento de participantes, y ha liderado iniciativas a nivel mundial para impulsar la investigación de gran impacto y el desarrollo de fármacos. La organización ha aparecido en medios de comunicación nacionales, como el New York Times, STAT, CNN, People y otros. Más información en emilysentourage.org.
Contacto con los medios
Hannah Kleckner
Director de marketing y comunicaciones
hannah@emilysentourage.org
