HDAd-ABE8e-R553X

HDAd-ABE8e-R553X is an experimental gene therapy candidate designed for the treatment of cystic fibrosis (CF) caused by the R553X nonsense mutation in the CFTR gene. The therapeutic utilizes a helper-dependent adenoviral (HDAd) vector to deliver an adenine base editor (ABE8e), which is engineered to perform a precise A-to-G conversion at the mutation site. This base editing approach aims to correct the premature stop codon, thereby restoring the expression of full-length, functional CFTR protein. Preclinical studies conducted by researchers at the University of Iowa, Harvard University, and the University of Rochester have demonstrated significant editing efficiency in humanized mouse models and primary human airway epithelial cells, suggesting its potential as a corrective treatment for patients with nonsense CFTR mutations.