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PQ001 is an investigational immunotoxin consisting of a tumor-targeting monoclonal antibody conjugated to the ribosome-inactivating protein saporin. Developed by PhotoQ3, PQ001 is designed for use with the company's proprietary intelligent Targeted Antibody Phototherapy (iTAP) platform to treat various solid tumors, including lung cancer, malignant brain tumors (glioblastoma), ovarian cancer, and pancreatic cancer. Saporin, a Type I ribosome-inactivating protein derived from *Saponaria officinalis*, lacks a cell-binding domain and is only active upon entering the cytosol, where it depurinates 28S ribosomal RNA to arrest protein synthesis. To overcome the poor endosomal escape of conventional immunotoxins, the iTAP method co-administers PQ001 with a photosensitizer (such as talaporfin sodium/NPe6) followed by 664 nm laser irradiation. This light activation triggers localized endosomal membrane disruption, facilitating the efficient release of saporin into the cytosol of target cells and enabling potent antitumor activity at low doses with minimal systemic toxicity.
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