AAV-CFTR gene therapy

**AAV-CFTR gene therapy** is a nonproprietary platform descriptor for investigational recombinant adeno-associated virus gene therapies designed to treat cystic fibrosis by delivering a functional CFTR transgene to airway epithelial cells. Individual programs differ materially in AAV capsid, promoter, CFTR transgene design, dose, and delivery approach; examples have included legacy rAAV2-CFTR vectors, Abeona's preclinical ABO-401, Spirovant's SPIRO-2101, and 4D Molecular Therapeutics' 4D-710. The intended effect is durable epithelial expression of functional cystic fibrosis transmembrane conductance regulator protein, restoring chloride and bicarbonate ion transport and thereby addressing the underlying molecular defect rather than correcting an individual CFTR mutation with a modulator. ([investors.abeonatherapeutics.com](https://investors.abeonatherapeutics.com/news-events/press-releases/detail/150/abeona-therapeutics-to-report-new-preclinical-data-demonstrating-therapeutic-potential-of-abo-401-for-treatment-of-cystic-fibrosis-at-american-society-of-gene-and-cell-therapy-annual-meeting))

Other names

AAV-CFTRadeno-associated virus CFTR gene therapy