We are thrilled to share that Emily’s Entourage (EE) has awarded $440,000 in funding for two new grants to advance cystic fibrosis (CF) research and drug development. The grants support the development of a new nonsense mutation ferret model and a novel form of gene therapy using lipid nanoparticles.
“These grants reflect Emily’s Entourage’s unwavering commitment to advance lifesaving research to fill critical unmet needs and expeditiously pursue promising therapeutic opportunities for the CF community, especially those that do not benefit from existing mutation-targeted therapies.“
— Chandra Ghose, PhD, Chief Scientific Officer, Emily’s Entourage
Generating W1286X cystic fibrosis ferret model for examining nonsense mutation-directed therapies
Xingshen Sun, PhD
University of Iowa
Before a drug or treatment approach can be used in human clinical trials, it first needs to be tested in an animal model to assess safety and efficacy. However, no appropriate animal models with a cystic fibrosis transmembrane conductance regulator (CFTR) nonsense mutation are available to date. Previous studies demonstrate that the ferret is an ideal species for a CF model because it develops a lung disease that leads to respiratory failure and develops pancreatic diseases that lead to CF-related diabetes. This project will aim to generate a CF ferret model containing the W1286X mutation in the CFTR gene, similar to the human W1282X mutation. The ferret model will be used to test whether treatment using multiple drugs can reduce disease symptoms in the ferret. This new W1286X ferret model provides a new opportunity to accelerate the identification of effective and safe therapeutic approaches, significantly shortening the course of identifying new drugs or new therapeutic strategies for patients with CFTR-W1282X mutation, and potentially other nonsense mutations, in ways that cell-based systems or rodent models will not allow.
Pulmonary non-viral delivery of base editors targeting W1282X
Debadyuti (Rana) Ghosh, PhD (pictured) and Hugh Smyth, PhD
The University of Texas at Austin
Gene-editing therapies that may be able to repair the defective gene associated with CF have the potential to achieve a permanent cure for the disease. There is currently a technology that can efficiently correct the mutation and restore the key protein that is involved with normal cell and lung function. However, it is difficult to deliver this therapy to the patient without it degrading in the body. This project will screen and identify pharmaceutical carriers — or materials for delivering the gene therapy cargo — that can protect it from degradation and, ultimately, be delivered to the lungs as inhaled medicines.
Since 2011, EE has awarded more than $5.2 million to 24 research projects across the globe. These latest grants comprise the sixth round of grant funding awarded by EE to accelerate therapeutic development for people with CF that do not benefit from existing mutation-targeted therapies, including those with nonsense mutations of CF.
We are grateful to the grant recipients and all of the dedicated scientists who are leading the charge to fill critical unmet needs for all people with CF fast — nobody left behind! View a comprehensive listing of all awarded research grants here.