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AAV6-NeuroD1 (NXL-004) is an investigational gene therapy developed by NeuExcell Therapeutics for the treatment of recurrent malignant glioma, including glioblastoma. It utilizes a self-complementary adeno-associated virus serotype 6 (scAAV6) vector to deliver the NeuroD1 gene, which encodes a neural transcription factor. The therapy's mechanism of action involves in vivo trans-differentiation, where it reprograms both reactive glial cells and glioma cells into non-proliferative, functional neuron-like cells. This approach aims to suppress tumor proliferation and induce cell death while potentially restoring neural function. It is currently being evaluated in first-in-human clinical trials via intracavitary or intratumoral injection.
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