Emily's Entourage financia un proyecto de investigación en colaboración que conecta laboratorios de EE.UU. y los Países Bajos para explorar la tecnología de vanguardia ACE-tRNA

Work from the lab of Jeffrey Beekman, PhD, and Sacha Spelier at the University Medical Center Utrecht will be tested in the lab of John D. Lueck, PhD, at the University of Rochester Medical Center

The pre-clinical research project aims to leverage ACE-tRNA technology to produce a fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to correct nonsense mutations of CF

Lower Merion, PA — Nov. 3, 2022 — Emily’s Entourage (EE), an innovative 501(c)3 foundation that accelerates research for new treatments and a cure for the final 10% of people with cystic fibrosis (CF), including those with nonsense mutations, today announced it has awarded a total of $220,000 in funding to two teams at the University of Rochester Medical Center and University Medical Center Utrecht in the Netherlands. The collaborative research project will explore using new technology to produce a fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein in people with nonsense mutations of CF.

Approximately 90% of people with CF benefit from mutation-targeted therapies that restore the function of their CFTR mutations. However, the remaining 10% of people with CF do not benefit from these drugs because they produce too little or no functional protein.

This collaborative research project, titled, “Anticodon-engineered transfer-RNA suppression of CFTR nonsense mutations in patient-derived organoids,” aims to explore the use of ACE-tRNAs to produce full-length, fully functional CFTR protein. Previous studies on ACE-tRNA technology have shown promising results, indicating that further investigation of ACE-tRNAs in patient-derived cell models is now warranted.

“We are laser-focused on bringing therapies to the final 10% of people with CF as quickly as possible and that requires innovation and collaboration,” said Chandra Ghose, PhD, Chief Scientific Officer at Emily’s Entourage. “Attracting the next generation of scientists to the CF space, removing funding roadblocks, and supporting their work are all critical components to achieving our mission as quickly as possible.”

Jeffrey Beekman, PhD, of University Medical Center Utrecht and his team, including PhD student Sacha Spelier, are leading the way in the development and execution of state-of-the-art translational CF biology experiments. John D. Lueck, PhD, of the University of Rochester Medical Center is a pioneer of ACE-tRNA technology. The two teams will work collaboratively to characterize CFTR function in patient-derived cells after ACE-tRNA treatment.

“We are really excited about the opportunity to work with Dr. Lueck’s team and are highly grateful for the generous support of EE that makes this possible,” said Jeffrey Beekman, PhD. “Previous work has shown that CFTR function measurements in patient-derived cells can be important indicators for individual disease severity or therapeutic response in people from whom these cells are isolated. Using these patient-derived cell models, we can validate ACE-tRNA safety and how much of it is needed to efficiently restore CFTR function.”

Importantly, ACE-tRNA technology has the potential to also work for other diseases associated with nonsense mutations, such as other rare genetic diseases. Results from this work will provide critical information to potentially aid in moving ACE-tRNAs closer to the clinic for countless individuals with nonsense mutations around the world.

Más información sobre las subvenciones financiadas por Emily's Entourage en www.emilysentourage.org/awarded-grants. 

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Acerca del séquito de Emily
Emily's Entourage es una innovadora organización 501(c)3 que acelera la investigación de nuevos tratamientos y una cura para las personas en el 10% final de la población con fibrosis quística (FQ) que no se beneficia de las terapias dirigidas a mutaciones actualmente disponibles, incluidas las personas con mutaciones sin sentido de la FQ. Desde 2011, Emily's Entourage ha concedido millones de dólares en becas de investigación, ha puesto en marcha una empresa de terapia génica de la FQ, ha desarrollado un registro de pacientes y un programa de búsqueda de pacientes compatibles para ensayos clínicos con el fin de acelerar el reclutamiento de pacientes para ensayos clínicos, y ha liderado los esfuerzos mundiales para impulsar la investigación de alto impacto y el desarrollo de fármacos. La organización ha aparecido en medios de comunicación nacionales como el New York Times, STAT, CNN y People, entre otros. Más información en emilysentourage.org.

Contacto con los medios
Ashling Knight
Director de marketing y comunicaciones
El séquito de Emily
ashling@emilysentourage.org