CSF2RA gene-corrected autologous macrophages

CSF2RA gene-corrected autologous macrophages is an investigational gene-modified cell therapy being developed for the treatment of hereditary pulmonary alveolar proteinosis (hPAP). The therapy, often referred to as Pulmonary Macrophage Transplantation (PMT), involves the ex vivo genetic correction of autologous CD34+ hematopoietic stem and progenitor cells using a lentiviral vector to deliver a functional CSF2RA gene, which encodes the alpha subunit of the GM-CSF receptor. These corrected cells are then differentiated into macrophages and delivered directly into the lungs via bronchoscopic instillation. This approach aims to restore functional GM-CSF signaling in alveolar macrophages, enabling them to clear the surfactant accumulation that characterizes hPAP. The program is currently being evaluated in Phase 1 clinical trials led by Cincinnati Children's Hospital Medical Center.

Other names

Pulmonary Macrophage TransplantationPMT