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INO-1001 is a small molecule isoindolinone derivative that acts as a potent inhibitor of the nuclear enzyme poly(ADP-ribose) polymerase (PARP), specifically targeting PARP-1 and PARP-2. By inhibiting PARP activity, INO-1001 interferes with DNA repair mechanisms in tumor cells, leading to chemosensitization and radiosensitization—making cancer cells more susceptible to chemotherapy and radiation therapy. It has been investigated for use in brain cancer, cardiovascular disorders (including myocardial infarction), abdominal aortic aneurysm, acute lung injury, erectile dysfunction, glioblastoma, malignant melanoma, and reperfusion injury[2][4][5][8]. The drug was originally developed by Inotek Pharmaceuticals and later associated with Genentech following company mergers[5].
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