Enhancement of CFTR nonsense mutation readthrough via combined small molecule and ACE-tRNA therapies (NACFC 2026) - Sep 9, 2026 - Abstract #272; "These approaches encompass small molecules that influence translation termination, including PTC124, aminoglycosides (e.g., G418), and release factor degraders such as SRI-41315 and CC-90009. These findings support our hypothesis that reducing the competition for ACE-tRNA binding to PTCs results in increased potency and/or efficacy. Consequently, the co-administration of ACE-tRNAs and eRF1/eRF3 degraders could mitigate the delivery burden of ACE-tRNAs necessary for CFTR restoration. Ongoing studies are focused on evaluating the functional rescue of CFTR following these combination treatments in comparison to monotherapy."
Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases
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K. Thrasher 1; N. Ward 1; J. Porter 1; D. Bedwell 2; J. Lueck 3
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