Emily’s Entourage Awards $220,000 Research Grant to Dr. Andrew Berical of Boston University to Advance Research into Gene Delivery Methods

The research is focused on developing partially humanized mouse airways to be utilized for testing the efficiency of gene delivery methods, including AAV vectors and lipid nanoparticles.

Andrew Berical, MD
Boston University

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. Andrew Berical, MD, Physician Scientist and Assistant Professor of Medicine at Boston University Chobanian & Avedisian School of Medicine, Pulmonary and Critical Care Medicine. The grant supports research to develop an in-vivo model of human airway epithelial cells to test gene therapy.

Dr. Berical is currently a faculty member and scientist in the Center for Regenerative Medicine, where his research focuses on airway disease modeling as well as the role of cellular regeneration as a potential therapy for CF and primary ciliary dyskinesia. In this research project, titled “In vivo human airway epithelial cells for CFTR gene therapy,” he plans to study the barriers to various gene delivery methods using W1282X human airway epithelial cells and a novel humanized mouse model.

The research proposal includes assessing the ability of viral vectors and lipid nanoparticles (LNP) to deliver mRNA to human airway epithelial cells with the goal of understanding delivery agent preferences for certain cell types of the human airway and the longevity of these gene delivery technologies.

“We are proud to support Dr. Berical in this groundbreaking research,” said Dr. Chandra Ghose, PhD, Chief Scientific Officer of EE. “Dr. Berical’s pioneering research aims to create an in vivo model of human airway epithelial cells to facilitate testing of gene therapy delivery mechanisms, which currently represents a major barrier to progress and is critical to accelerating the development of new therapies for individuals with CF, including those in the final 10%.”

Leveraging expertise in pluripotent stem cell directed differentiations and recent progress in transplanting human induced pluripotent stem cell (iPSC)-derived basal cells into a mouse airway, the project aims to create a partially humanized mouse airway for testing the efficiency of gene delivery methods. The team will then use this system to assess the ability of two specific agents to deliver genes to human airway epithelial cells: an Adeno-Associated Virus (AAV) vector and LNPs. 

“This generous support from Emily’s Entourage will enable our lab to address a significant gap in CF research. By creating a model system that includes human CF airway epithelial cells, we can advance our understanding of gene delivery mechanisms and pave the way for mutation-agnostic therapies that can benefit all individuals with CF, regardless of their specific CFTR mutation,” said Dr. Berical.

Through this research, Dr. Berical and his research team hope to bridge a significant knowledge gap in the CF field, ultimately contributing to the development of efficacious therapies for all individuals with CF.

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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