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InC01 is a protein-engineered anti-cancer cytokine therapy developed by InCephalo for the treatment of brain cancer. It utilizes a modified form of interleukin-12 (IL-12), a potent anti-cancer cytokine, and is designed to be administered locally into brain tumors. The drug leverages InCephalo's proprietary Compartment Lock (C-Lock) technology, which enables the molecule to remain at the tumor site long enough to exert its therapeutic effect while ensuring rapid degradation if it diffuses away from the target area. This approach aims to maximize local efficacy and minimize systemic toxicity—a significant challenge in treating brain cancers due to the blood-brain barrier and potential side effects of cytokines like IL-12. In preclinical studies, including tests on patient-derived brain tumor samples, InC01 demonstrated proof-of-concept efficacy. Based on these results, it received orphan drug designation from the European Medicines Agency (EMA) for use against rare life-threatening or chronically debilitating conditions such as certain types of brain cancer[2].
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