Grant: Planning and Dissemination Grant - Institute Community Support

Although an overwhelming majority of cystic fibrosis (CF) patients are able to benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs, there remain few options for the small population of patients whose genotypes render them either ineligible or unable to benefit from this type of treatment. For this population, gene therapies (nucleic acid-based therapies remain their most promising avenue of treatment. Unfortunately, the CF population is experiencing highly disparate treatment outcomes due to the availability of CFTR modulators for individuals with common mutations, while individuals with less common mutations continue to suffer the full brunt of undertreated disease. Our health equity initiative aims to provide comprehensive and accessible education regarding genetic therapies for the subset of the cystic fibrosis patient population who may benefit from non-CFTR modulator therapies. Given the novelty and complexity of these treatments, providing patients and caregivers with thorough, accurate, and accessible information about these treatments is urgently needed. With the resources provided by this grant, Cystic Fibrosis Canada will be able to leverage our trusted reputation among the CF community along with our partnerships with local clinics to provide this essential education to this population. The educational materials developed through this initiative will have relevance to many other populations suffering from genetic diseases that may be treated through gene therapy. Providing education to these patients is essential in encouraging the uptake of these potentially life-saving treatments.