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SRN-101 is a recombinant adeno-associated viral (AAV) vector-based immuno-gene therapy developed for the treatment of high-grade gliomas, including pediatric-type diffuse high-grade gliomas. It works by delivering an engineered cytokine gene directly into tumor cells via AAV vectors. Once inside the tumor, the engineered cytokine is expressed and secreted, killing tumor cells from within and stimulating local immune responses to attack remaining cancer cells at the tumor margin. This dual mechanism leverages both direct cytotoxicity and immune-mediated anti-tumor effects. SRN-101 has received Orphan Drug and Rare Pediatric Disease designations from the FDA due to its potential to address significant unmet needs in aggressive brain cancers with limited therapeutic options[1][5][6][7].
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