Emily’s Entourage Awards $220,000 Research Grant to Dr. Alexandra Pitrowski-Daspit of the University of Michigan to Support Non-viral Nucleic Acid Delivery Research for CF Nonsense Mutations

The research is focused on developing polymeric vehicles to improve the delivery of  nucleic acid-based therapeutics to target tissues and cells in the body for the treatment of CF.  

Lower Merion, PA – November 20, 2023 — Emily’s Entourage (EE), an innovative 501(c)3 that accelerates research for new treatments and a cure for the final 10% of people with cystic fibrosis (CF), today announced it has awarded $220,000 in funding to Dr. Alexandra S. Piotrowski-Daspit, PhD, Assistant Professor of Biomedical Engineering and Internal Medicine, Pulmonary and Critical Care Medicine Division at the University of Michigan. The grant supports research focused on non-viral nucleic acid delivery to provide more effective treatment options for people with nonsense mutations of CF.

Alexandra Piotrowski-Daspit, PhD
University of Michigan

CF is a progressive genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There are over 1,700 CF-causing mutations, which are classified according to resulting functional alterations in CFTR. Nonsense mutations such as W1282X and G542X result in premature stop codons, thereby producing truncated CFTR protein and loss of function. Nucleic acid-based therapies, including gene editing, gene therapy and mRNA therapy, offer the potential to replace or correct dysfunctional CFTR. However, delivery of these therapies to target organs remains a primary barrier to clinical translation. 

The project, titled, “Multi-organ targeted non-viral nucleic acid delivery for CF nonsense mutations,” aims to explore the development of polymeric vehicles to improve the delivery of nucleic acid-based therapeutics to target tissues and cells in the body, primarily the lung and the gastrointestinal system. 

“Nucleic acid-based therapeutics are especially promising because of the way they work to replace or correct dysfunctional CFTR. These treatment approaches could lead to treatment options for people with CF, but we need to make sure they can be delivered effectively,” said Chandra Ghose, PhD, Chief Scientific Officer of EE. “Research and development efforts in this area will be critical as we continue to add to our toolbox and innovate in order to develop treatments for all people with CF, including those with rare and nonsense mutations.” 

The grant will support the research team’s efforts to explore nucleic acid-based therapeutics, with a focus on genome editing agents to replace or correct dysfunctional CFTR. The ultimate goal is to develop effective vehicles for targeted in vivo therapeutic nucleic acid delivery to epithelia in the lungs and gastrointestinal tract, addressing the unique challenges posed by the W1282X CF nonsense mutation.

“We are incredibly grateful to Emily’s Entourage for this generous grant, which will enable us to advance our research on polymeric vehicles for targeted delivery of nucleic acid-based therapeutics,” said Dr. Piotrowski-Daspit. “The long-term goal of our work is to develop a treatment applicable to all people with CF, providing them with the opportunity to lead full, productive lives.”

The proposed interdisciplinary research is highly clinically relevant, furthering the translation of promising nucleic acid therapeutics for people with CF, including the final 10% of the CF patient population with rare and nonsense mutations that do not benefit from currently available CFTR modulator therapies. 

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 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-acquired CF gene therapy company, developed a patient registry 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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