SP-101 + doxorubicin

SP-101 + doxorubicin is an **investigational combination therapy** for the treatment of **cystic fibrosis** (CF). SP-101 is a recombinant adeno-associated virus (AAV) gene therapy vector that delivers a human CFTR minigene (hCFTRΔR), aiming to restore CFTR function in airway epithelial cells. Doxorubicin, a small molecule typically used as a chemotherapeutic agent, is repurposed here as an **AAV transduction augmenter**—it significantly enhances the gene transfer and expression of the delivered CFTR transgene. The combination is mutation agnostic, meaning it is designed to benefit CF patients who are ineligible for or not adequately helped by existing CFTR modulator therapies. Evidence from preclinical and early clinical studies demonstrates that co-administration via inhalation corrects CFTR function in airway models and results in durable, broad airway transgene expression. Clinical development is ongoing, using single dose, inhaled administration in CF patients[1][2][3][4][5][6][8].

Other names

SP-101 and doxorubicinSP101 and doxorubicinSP 101 and doxorubicin