Accelerating Gene Therapy Delivery for Cystic Fibrosis: Emily’s Entourage to Convene Experts at 2025 Symposium

Philadelphia, PA—February 20, 2025—Emily’s Entourage (EE), a pioneering nonprofit accelerating research for the final 10% of the cystic fibrosis (CF) community, is set to host its 2025 Scientific Symposium titled “The Delivery of Genes: Advancements and Challenges Through a CF Lens,” from February 23-26, 2025, at the Quorum at the University City Science Center in Philadelphia, Pennsylvania.

This year’s symposium aims to push the boundaries of progress in the development of nucleotide-based therapies for CF, focusing on one of the most critical and complex challenges of gene-based treatments: their delivery. While advancements have been made, the obstacles related to delivering genetic material effectively remain a major hurdle. While the nucleotide-based therapies hold promise for the entire CF community, the need is particularly urgent  for those in the final 10% of the CF population who do not benefit from currently available CFTR modulator therapies. For this group, time is of the essence, and there is no room to wait for progress to unfold on traditional timelines.

“We believe in the power of bringing together the world’s most brilliant researchers and thought leaders in one room. Our goal is to encourage open sharing, spark collaboration, think big, and forge new, expedited pathways forward,” said Emily Kramer-Golinkoff, MBE, EE co-founder. “By convening the leading minds from within—and outside—the CF scientific community and industry, this symposium is poised to accelerate the delivery of these breakthrough therapies.”

Chaired by Kevin Foskett, PhD, Isaac Ott Professor of Physiology at the University of Pennsylvania’s Perelman School of Medicine; Joan Lau, PhD, MBA, CEO at Spirovant Sciences, Inc.; and Paul McCray, Jr, MD, professor of pediatrics-pulmonary medicine at the University of Iowa, the event will feature more than 40 globally renowned researchers, innovators, and thought leaders from academia, biotech, pharma—both within the CF space and beyond. These experts will examine cutting-edge advancements and dive into the unique challenges of safely and effectively delivering nucleotide-based therapies to individuals with CF, a critical step in unlocking the full potential of gene-based therapy. With an eye on translational opportunities, this symposium not only addresses the science but also the power of collaboration to transform the landscape of CF treatment.

The 2025 Symposium builds on EE’s ongoing efforts to speed therapeutic development for the final 10% of people with CF, who do not benefit from current CFTR modulators due to their rare genetic mutations. Highlights of EE’s work include:

• Awarding 38 research grants to top investigators worldwide, resulting in over $52.3 million in follow-on funding.
• Deploying a venture philanthropy model to spin out a CF gene therapy company now conducting a Phase 1/2 clinical trial.
• Developing 37 phages that have treated over 34 individuals with antibiotic-resistant infections.
• Launching CF Clinical Trial Connect (CTC), a global patient database, and Clinical Trial Matchmaking Program to accelerate recruitment for this ultra-rare population.

“We are incredibly proud of the ongoing momentum in CF gene therapy research, but we recognize that in order to truly make a difference and develop treatments, we need to face the tough questions and collaborate across disciplines,” said Chandra Ghose, Chief Scientific Officer at EE. “By convening such a diverse group of thought leaders, we aim to spark new ideas, push boundaries, and ultimately expedite the timeline for developing therapies for those who need them the most.”

EE thanks the 2025 symposium dream team sponsors, Viatris, Terapéutica molecular 4D, Vertex Pharmaceuticals, Inc., violet-level sponsors, ReCode Therapeutics and Boomer Esiason Foundation, and more whose support makes this critical event and the progress that it yields possible.

Acerca del séquito de Emily

Emily’s Entourage is an innovative 501(c)3 that accelerates research for new treatments and a cure for individuals in the final 10% of the cystic fibrosis (CF) population that does not benefit from currently available mutation-targeted therapies. Since 2011, Emily’s Entourage has awarded millions of dollars in research grants, launched a now-clinical stage CF gene therapy company, developed a patient database and clinical trial matchmaking program to accelerate clinical trial recruitment, and led worldwide efforts to drive high-impact research and drug development. The organization has been featured in national media, including the New York Times, STAT, CNN, People, and more. Learn more at emilysentourage.org.

Contacto con los medios

Hannah Kleckner

Director de marketing y comunicaciones

hannah@emilysentourage.org