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NAI001 is a preclinical small molecule inhibitor developed by Hangzhou Nain Biotechnology that targets the transcription factor OCT4 (also known as POU domain, class 5, transcription factor 1 or POU5F1). OCT4 is a master regulator of pluripotency and self-renewal in embryonic stem cells and is frequently overexpressed in cancer stem cells (CSCs), where it drives tumor progression, metastasis, and resistance to conventional therapies. By specifically inhibiting OCT4, NAI001 is designed to deplete the cancer stem cell population and sensitize solid tumors to treatment, potentially offering a novel therapeutic approach for advanced malignancies.
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