SP-101

SP-101 is an investigational gene therapy developed for the treatment of cystic fibrosis (CF). It is a recombinant adeno-associated virus (AAV) vector-based therapy designed for inhalation delivery. The vector carries a functional and regulated shortened human CFTR minigene (hCFTRΔR), aiming to restore expression of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in airway epithelial cells. This approach targets the underlying genetic defect in CF and is mutation agnostic, making it suitable for patients who are ineligible for or do not benefit from current small molecule CFTR modulators—including those with nonsense or Class I mutations. In clinical trials, SP-101 is administered together with doxorubicin as an augmenter to enhance AAV-mediated gene transfer and transgene expression in the lungs. The therapy has received orphan drug designation for cystic fibrosis and is currently being evaluated in Phase 1/2 clinical studies as an inhaled single-dose treatment[1][2][3][4][5][6][7][8].