NXL-004

NXL-004 is a first-in-class investigational gene therapy developed by NeuExcell Therapeutics for the treatment of malignant glioma, including glioblastoma. It utilizes an adeno-associated virus (AAV) vector to deliver the Neurogenic differentiation factor 1 into brain tissue. The therapy is based on in vivo trans-differentiation technology, specifically converting astrocytes or tumor cells into functional neurons through overexpression of Neurogenic differentiation factor 1. This approach aims to inhibit tumor cell proliferation and promote their transformation into neural cells, potentially regenerating damaged neuronal tissue with minimal side effects on healthy brain cells—unlike traditional radiation or chemotherapy[1][2][5]. NXL-004 has received FDA Orphan Drug Designation and entered Phase 1 clinical trials as of early 2024[3][7]. Preclinical studies have shown promising efficacy and safety.

Other names

NeuExcell ATN therapyAAV-NeuroD1AAV-NeuroD-1AAV-NeuroD 1Astrocyte-to-Neuron therapy