{"id":27114,"date":"2026-09-09T14:51:00","date_gmt":"2026-09-09T18:51:00","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=27114"},"modified":"2026-09-09T14:52:12","modified_gmt":"2026-09-09T18:52:12","slug":"emilys-entourage-breaks-new-ground-with-ai-powered-grant-to-identify-next-generation-cf-gene-therapy-delivery-systems","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/es\/emilys-entourage-breaks-new-ground-with-ai-powered-grant-to-identify-next-generation-cf-gene-therapy-delivery-systems\/","title":{"rendered":"Emily\u2019s Entourage abre nuevos caminos con una subvenci\u00f3n impulsada por inteligencia artificial para identificar sistemas de administraci\u00f3n de terapia g\u00e9nica para la fibrosis qu\u00edstica de pr\u00f3xima generaci\u00f3n"},"content":{"rendered":"

AI-guided lipid nanoparticle screening aims to overcome longstanding barriers to inhaled CF gene therapy<\/span><\/i><\/p>\n

\"\"

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

Lower Merion, PA\u2014September 9, 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 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).<\/span><\/p>\n

Dr. Witten\u2019s project aims to develop improved lipid nanoparticles (LNPs) designed specifically for inhaled gene therapy\u2014addressing a significant barriers to restoring CFTR function in people with CF who do not benefit from current mutation-targeted therapies.<\/span><\/p>\n

For these individuals\u2014known as the final 10%\u2014restoring 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.<\/span><\/p>\n

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\u2019s unique biological barriers, including thick mucus, limited cellular uptake, and tight junctions between airway cells.<\/span><\/p>\n

Dr. Witten\u2019s 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.<\/span><\/p>\n

A key innovation of the project is the use of apical-out airway organoids\u2014advanced 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.<\/span><\/p>\n

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

\u201cOne of the greatest challenges in CF gene therapy is getting treatments to the right cells in the lung,\u201d said Dr. Witten. \u201cThis 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.\u201d<\/span><\/p><\/blockquote>\n

For individuals in the final 10%, effective delivery of mRNA or gene editing tools could represent a transformative step toward restoring lung function.<\/span><\/p>\n

\u201cSolving the airway delivery challenge is essential to unlocking the full potential of nucleotide-based therapies therapy for CF,\u201d said Chandra Ghose, PhD, Chief Scientific Officer at EE. \u201cBy supporting innovative strategies that combine AI with advanced airway models, we\u2019re helping accelerate the development of delivery technologies designed specifically for those unable to benefit from current CFTR modulator therapies.\u201d<\/span><\/p><\/blockquote>\n

 <\/p>\n

Acerca del Programa de Subvenciones de Emily's Entourage (EE)<\/b><\/p>\n

El Programa de Subvenciones de Emily\u2019s Entourage (EE) proporciona financiaci\u00f3n para acelerar la investigaci\u00f3n y el desarrollo terap\u00e9utico para personas con fibrosis qu\u00edstica (FQ) que no se benefician de las terapias existentes dirigidas a mutaciones. EE apoya una variedad de mecanismos de financiaci\u00f3n, incluidas subvenciones translacionales, subvenciones colaborativas y subvenciones exploratorias precl\u00ednicas, as\u00ed como inversiones de filantrop\u00eda de riesgo.<\/span><\/p>\n

Hasta la fecha, EE ha otorgado millones de d\u00f3lares a equipos multidisciplinarios de todo el mundo y ha ayudado a asegurar millones m\u00e1s en financiaci\u00f3n posterior. Para ver las subvenciones concedidas, visite <\/span>https:\/\/www.emilysentourage.org\/awarded-grants\/<\/span><\/a>. Para saber m\u00e1s sobre las oportunidades de financiaci\u00f3n de EE, visite <\/span>https:\/\/www.emilysentourage.org\/funding-opportunities\/<\/span><\/a>.<\/span><\/p>\n

Acerca del s\u00e9quito de Emily<\/b><\/p>\n

Emily\u2019s Entourage (EE) es una innovadora organizaci\u00f3n sin \u00e1nimo de lucro 501(c)3 que impulsa la investigaci\u00f3n de nuevos tratamientos y una cura para las personas que se encuentran en la fase final de la fibrosis qu\u00edstica (FQ) y que no se benefician de las terapias dirigidas a mutaciones disponibles en la actualidad. Desde 2011, EE ha concedido millones de d\u00f3lares en subvenciones de investigaci\u00f3n, ha puesto en marcha una empresa de terapia g\u00e9nica para la FC en fase cl\u00ednica, ha desarrollado una base de datos global de pacientes y un programa de selecci\u00f3n de ensayos cl\u00ednicos para acelerar el reclutamiento de participantes, y ha liderado iniciativas a nivel mundial para impulsar la investigaci\u00f3n de gran impacto y el desarrollo de f\u00e1rmacos. La organizaci\u00f3n ha aparecido en medios de comunicaci\u00f3n nacionales, como el New York Times, STAT, CNN, People y otros. M\u00e1s informaci\u00f3n en<\/span> emilysentourage.org<\/span><\/a>.<\/span><\/p>\n

Contacto con los medios<\/b><\/p>\n

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

Director de marketing y comunicaciones<\/span><\/p>\n

hannah@emilysentourage.org\u00a0

<\/p>","protected":false},"excerpt":{"rendered":"

AI-guided lipid nanoparticle screening aims to overcome longstanding barriers to inhaled CF gene therapy Lower Merion, PA\u2014September 9, 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 proud to announce its sixth and final 2026 Translational Research Grant awarded to… <\/p>\n

<\/div>\n

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