Emily’s Entourage Funds Synthetic Biology Approach to Enable Durable Gene Therapy for Cystic Fibrosis
Engineered synthetic receptor technology aims to selectively expand corrected airway stem cells and improve long-term treatment durability.
Emily’s Entourage (EE) first 2026 Translational Research Grant has been awarded to Sriram Vaidyanathan, PhD, Principal Investigator at Nationwide Children’s Hospital (left) and Assistant Professor of Pediatrics at The Ohio State University, and Kyle Cromer, PhD, Assistant Professor at the University of California, San Francisco (Right).
Lower Merion, PA—August 13, 2026—Emily’s 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, today announced its first 2026 Translational Research Grant awarded to Sriram Vaidyanathan, PhD, Principal Investigator at Nationwide Children’s Hospital and Assistant Professor of Pediatrics at The Ohio State University, and Kyle Cromer, PhD, Assistant Professor at the University of California, San Francisco (UCSF).
The newly funded project aims to address a key barrier to durable gene therapy for CF: ensuring that corrected airway stem cells can expand and persist long enough to repair lung tissue.
Gene-editing approaches that replace the faulty CFTR gene in people with CF have shown the potential to restore function in airway cells. However, even when gene correction is successful, corrected airway basal stem cells—the long-lived cells responsible for regenerating airway tissue—do not naturally outcompete uncorrected cells. As a result, corrected cells may fail to expand sufficiently to produce a lasting therapeutic benefit.
To overcome this challenge, Drs. Vaidyanathan and Cromer are developing engineered “synthetic receptors” designed to provide corrected cells with a temporary and controllable growth advantage. These receptors can be activated using a small-molecule compound that has already been evaluated in clinical settings. When activated, the receptors signal to corrected cells to expand, helping them repopulate the airway epithelium. Once expansion is achieved, the signal can be turned off.
The project will focus on engineering and testing two families of synthetic receptors that mimic natural growth-factor pathways involved in airway cell regeneration. The team will evaluate whether these receptors can selectively enrich CFTR-corrected airway basal stem cells while preserving their ability to differentiate into healthy airway tissue.
By enabling selective expansion of corrected cells, this approach seeks to achieve durable repair of the airway, one of the most persistent challenges in CF gene therapy.
“Gene correction alone may not be enough if corrected cells can’t outcompete diseased tissue,” said Dr. Vaidyanathan. “We’re developing a system that temporarily tilts the balance—allowing repaired cells to expand and potentially improve the durability of gene-based therapies.”
This synthetic biology approach is designed to work alongside existing gene-editing strategies and could be compatible with both cell-based and in vivo gene therapy approaches.
“One of the biggest challenges in gene therapy isn’t just fixing the gene—it’s ensuring those corrected cells can persist and repair tissue over time,” said Chandra Ghose, PhD, Chief Scientific Officer at EE. “This project introduces a creative synthetic biology strategy to address that challenge and help advance more durable treatment approaches for CF.”
Approximately 10% of people with CF do not benefit from current CFTR modulators. EE is committed to accelerating research that addresses both the underlying genetic causes of CF and the biological barriers that continue to limit treatment durability.
About Emily’s Entourage’s (EE’s) Grant Program
Emily’s Entourage’s (EE’s) Grant Program provides funding to accelerate research and therapeutic development for people with cystic fibrosis (CF) who do not benefit from existing mutation-targeted therapies. EE supports a range of funding mechanisms, including translational grants, collaborative grants, and preclinical exploratory grants, as well as venture philanthropy investments.
To date, EE has awarded millions of dollars to multi-disciplinary teams around the world and helped secure millions more in follow-on funding. To view awarded grants, visit https://www.emilysentourage.org/awarded-grants/. To learn more about EE’s funding opportunities, visit https://www.emilysentourage.org/funding-opportunities/.
About Emily’s Entourage
Emily’s Entourage (EE) 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 do not benefit from currently available mutation-targeted therapies. Since 2011, EE has awarded millions of dollars in research grants, launched a clinical-stage CF gene therapy company, developed a global 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.
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