The funds raised by Emily’s Entourage drive high-impact research, information exchange, and collaboration to accelerate breakthroughs for people with nonsense mutations of Cystic Fibrosis. These individuals make up the roughly 10% of the CF population without a targeted therapy available or in development.
The work funded by Emily’s Entourage is particularly significant because although nonsense mutations are relatively uncommon in the CF population, they are the cause of approximately 12% of all genetic diseases, affecting roughly 30 million people worldwide. Progress on nonsense mutations of Cystic Fibrosis can have far-reaching implications for patients suffering from many different genetic diseases, including certain types of muscular dystrophy, inherited blood disorders, and cancers.
In partnership with its Scientific Advisory Board, Emily’s Entourage uses strategic research priorities to guide its targeted financial investments in research projects with the greatest potential for impact within 5 years. This results-oriented approach to expedite research is paradigm shifting and a sharp departure from the norm, where the scientific community drives research direction and progress can be fragmented and inefficient.
Emily’s Entourage provides grant funding for research on nonsense mutations of Cystic Fibrosis with a particular focus on the W1282X mutation. The organization funds work with the greatest potential to reach patients within 5 years. Comprised of leading researchers and clinicians, the Emily’s Entourage Scientific Advisory Board helps identify and prioritize key research areas to expedite therapeutic development for nonsense mutations of CF in the short to mid term. Grant recipients are determined by the organization’s Leadership Team. Award amounts vary depending on the scope and duration of the selected projects.
Emily’s Entourage hosts scientific symposia that bring together the world’s top CF minds from academia, clinical care, and the biopharmaceutical industry. The goal of these symposia is to generate multi-disciplinary discussions, exchange ideas, share data and updates, and foster partnerships to expedite research and drug development for nonsense mutations using the W1282X mutation as a prototype.
Emily is one of only a few hundred people worldwide with two copies of the rare W1282X nonsense mutation, making her “pure” cells a highly valuable research asset. One of the most vital resources that Emily’s Entourage offers the scientific community is cells, which are developed into critically important cell models, including immortalized cell lines, induced pluripotent stem cells, airway epithelial cells, and organoids, and shared with investigators and companies around the world to spur research and drug development.