{"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\/fr_ca\/emilys-entourage-breaks-new-ground-with-ai-powered-grant-to-identify-next-generation-cf-gene-therapy-delivery-systems\/","title":{"rendered":"Emily\u2019s Entourage ouvre de nouvelles voies avec une subvention propuls\u00e9e par l'IA pour identifier des syst\u00e8mes de d\u00e9livrance de th\u00e9rapie g\u00e9nique de nouvelle g\u00e9n\u00e9ration pour la FK"},"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

\u00c0 propos du programme de subventions de l'Entourage d'Emily (EE)<\/b><\/p>\n

Le programme de subventions d'Emily's Entourage (EE) fournit des financements pour acc\u00e9l\u00e9rer la recherche et le d\u00e9veloppement th\u00e9rapeutique pour les personnes atteintes de fibrose kystique (FK) qui ne b\u00e9n\u00e9ficient pas des th\u00e9rapies cibl\u00e9es sur les mutations existantes. EE soutient une gamme de m\u00e9canismes de financement, notamment des subventions translationnelles, des subventions collaboratives et des subventions exploratoires pr\u00e9cliniques, ainsi que des investissements en capital-risque philanthropique.<\/span><\/p>\n

\u00c0 ce jour, EE a octroy\u00e9 des millions de dollars \u00e0 des \u00e9quipes pluridisciplinaires \u00e0 travers le monde et a aid\u00e9 \u00e0 obtenir des millions de plus en financements compl\u00e9mentaires. Pour consulter les subventions accord\u00e9es, visitez <\/span>https:\/\/www.emilysentourage.org\/awarded-grants\/<\/span><\/a>. Pour en savoir plus sur les possibilit\u00e9s de financement de l'EE, consultez le site suivant <\/span>https:\/\/www.emilysentourage.org\/funding-opportunities\/<\/span><\/a>.<\/span><\/p>\n

\u00c0 propos de l'entourage d'Emily<\/b><\/p>\n

Emily\u2019s Entourage (EE) est une organisation innovante de type 501(c)3 qui acc\u00e9l\u00e8re la recherche de nouveaux traitements et d\u2019un rem\u00e8de pour les personnes atteintes de mucoviscidose (CF) qui ne tirent aucun b\u00e9n\u00e9fice des th\u00e9rapies ciblant les mutations actuellement disponibles. Depuis 2011, EE a octroy\u00e9 des millions de dollars de subventions de recherche, lanc\u00e9 une soci\u00e9t\u00e9 sp\u00e9cialis\u00e9e dans la th\u00e9rapie g\u00e9nique de la mucoviscidose au stade clinique, d\u00e9velopp\u00e9 une base de donn\u00e9es mondiale de patients et un programme de mise en relation pour les essais cliniques afin d\u2019acc\u00e9l\u00e9rer le recrutement des participants, et men\u00e9 des initiatives \u00e0 l\u2019\u00e9chelle mondiale pour favoriser la recherche \u00e0 fort impact et le d\u00e9veloppement de m\u00e9dicaments. L\u2019organisation a fait l\u2019objet de reportages dans les m\u00e9dias nationaux, notamment le New York Times, STAT, CNN, People et bien d\u2019autres. Pour en savoir plus, rendez-vous sur<\/span> emilysentourage.org<\/span><\/a>.<\/span><\/p>\n

Contact presse<\/b><\/p>\n

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

Directeur du marketing et de la communication<\/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

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