Emily’s Entourage Awards 2025 Translational Research Grant to Develop New, Highly-Targeted Therapy for Cystic Fibrosis Nonsense Mutations

The newly-funded project seeks to develop a highly targeted treatment to bypass cystic fibrosis nonsense mutations, offering new hope for those still waiting for mutation-targeted therapies.

Lower Merion, PA—July 17, 2025—Emily’s Entourage (EE), an innovative 501(c)(3) organization accelerating research for individuals with cystic fibrosis (CF) who do not benefit from existing CFTR modulator therapies, is proud to announce a 2025 Translational Research Grant awarded to Andrei Korostelev, PhD, professor at the RNA Therapeutics Institute, University of Massachusetts Chan Medical School. Dr. Korostelev’s project aims to develop a new, highly targeted therapy for CF caused by nonsense mutations—a type of genetic change that affects nearly 10% of the CF community who are unable to benefit from current mutation-targeted therapies. 

Nonsense mutations act like premature “stop signs” in the genetic instructions for making the CFTR protein, which is essential for healthy lung and organ function. These faulty stop signals halt protein production prematurely, resulting in an incomplete, nonfunctional protein. Although various approaches have tried to bypass these stop signals, they have not shown meaningful clinical benefits. 

Building on years of pioneering work in RNA biology and protein synthesis, Dr. Korostelev’s team seeks to develop a first-in-class therapeutic approach targeting CF-causing nonsense mutations, such as G542X, using readthrough antisense oligonucleotides (R-ASOs). These short strands of genetic material are designed to bind near the premature stop signals in CFTR’s instructions, encouraging the cell’s machinery to “read through” the mutation and continue building the full-length CFTR protein.

What makes this approach especially promising is its precision. Unlike earlier readthrough therapies, R-ASOs are designed to target the faulty stop codons in CFTR specifically—dramatically reducing the risk of interfering with normal stop codons elsewhere.

The grant from Emily’s Entourage will support Dr. Korostelev’s efforts to refine this approach for several of the most common CF nonsense mutations, including Y122X, R553X, R1162X and W1282X. Early lab results suggest that placing an R-ASO in just the right position downstream of a nonsense mutation can boost readthrough activity by up to 20-fold.

In parallel, Dr. Korostelev is working with Anastasia Khvorova, PhD, a global leader in RNA-based drug development, to improve how R-ASOs are delivered and stabilized inside the body. Together, they aim to enhance the therapeutic impact of R-ASOs by testing chemical modifications and combinations with other low-dose agents. This work will be carried out in cell-based models of CF, with the long-term goal of preparing the most promising candidates for studies in animal models—and eventually, in humans.

“People with nonsense mutations have waited far too long for a breakthrough,” said Korostelev. “With this grant, we aim to develop safer, more effective treatments that could transform care for the subset of CF patients who currently have no mutation-targeted therapeutic options.”

Approximately 10% of people with cystic fibrosis have genetic mutations that are ineligible for existing CFTR modulators. Those with nonsense mutations of CF are among the genetically ineligible mutations. Emily’s Entourage is committed to advancing research and therapeutic development for all individuals that do not benefit from existing CFTR modulators, including those with nonsense mutations, to fill this critical gap.

“This work represents a critical step forward in our mission to accelerate the development of lifesaving treatments for the final 10% of the CF community, a group that has been left behind and is in urgent need of advances” said Chandra Ghose, PhD, Chief Scientific Officer at Emily’s Entourage. “It’s a rare opportunity to support research that is personalized AND can be optimized to include others that carry a different nonsense mutation..”

About Emily’s Entourage’s (EE’s) Grant Program 

Emily’s Entourage’s (EE’s) Grant Program provides grant funding to accelerate research and therapeutic development for people with cystic fibrosis (CF) who do not benefit from existing mutation-targeted therapies. EE provides funding through several grant funding mechanisms, including translational grants, collaborative grants, and preclinical exploratory grants, as well as through venture philanthropy investments.

To date, EE has awarded millions of dollars to multi-disciplinary teams around the world and helped to secure millions of dollars 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 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, Emily’s Entourage has awarded millions of dollars in research grants, launched a now clinical-stage CF gene therapy company, developed the Clinical Trial Connect (CTC)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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