{"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\/pt_br\/emilys-entourage-breaks-new-ground-with-ai-powered-grant-to-identify-next-generation-cf-gene-therapy-delivery-systems\/","title":{"rendered":"Emily's Entourage inova com subs\u00eddio impulsionado por IA para identificar sistemas de entrega de terapia g\u00eanica para fibrose c\u00edstica de pr\u00f3xima gera\u00e7\u00e3o"},"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

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

O Programa de Subs\u00eddios da Emily\u2019s Entourage (EE) fornece financiamento para acelerar a pesquisa e o desenvolvimento terap\u00eautico para pessoas com fibrose c\u00edstica (FC) que n\u00e3o se beneficiam de terapias existentes direcionadas a muta\u00e7\u00f5es. O EE apoia uma variedade de mecanismos de financiamento, incluindo subs\u00eddios translacionais, subs\u00eddios colaborativos e subs\u00eddios explorat\u00f3rios pr\u00e9-cl\u00ednicos, bem como investimentos de filantropia de risco.<\/span><\/p>\n

At\u00e9 o momento, a EE concedeu milh\u00f5es de d\u00f3lares a equipes multidisciplinares em todo o mundo e ajudou a garantir milh\u00f5es adicionais em financiamento subsequente. Para ver as bolsas concedidas, visite <\/span>https:\/\/www.emilysentourage.org\/awarded-grants\/<\/span><\/a>. Para saber mais sobre as oportunidades de financiamento da EE, acesse <\/span>https:\/\/www.emilysentourage.org\/funding-opportunities\/<\/span><\/a>.<\/span><\/p>\n

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

A Emily\u2019s Entourage (EE) \u00e9 uma organiza\u00e7\u00e3o inovadora com status 501(c)3 que acelera a pesquisa de novos tratamentos e de uma cura para indiv\u00edduos na fase final da fibrose c\u00edstica (FC) que n\u00e3o se beneficiam das terapias direcionadas a muta\u00e7\u00f5es dispon\u00edveis atualmente. Desde 2011, a EE j\u00e1 concedeu milh\u00f5es de d\u00f3lares em subs\u00eddios de pesquisa, lan\u00e7ou uma empresa de terapia g\u00eanica para FC em fase cl\u00ednica, desenvolveu um banco de dados global de pacientes e um programa de correspond\u00eancia para ensaios cl\u00ednicos a fim de acelerar o recrutamento para esses ensaios, al\u00e9m de liderar esfor\u00e7os mundiais para impulsionar pesquisas de alto impacto e o desenvolvimento de medicamentos. A organiza\u00e7\u00e3o tem sido destaque na m\u00eddia nacional, incluindo o New York Times, STAT, CNN, People e outros. Saiba mais em<\/span> emilysentourage.org<\/span><\/a>.<\/span><\/p>\n

Contato com a m\u00eddia<\/b><\/p>\n

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

Diretor de Marketing e Comunica\u00e7\u00f5es<\/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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