NeuExcell Therapeutics, a Shanghai-based developer of in vivo neural regenerative therapies, has announced the first patient dosing in a clinical trial for its first-in-class (FIC) NeuroD1 gene therapy product, NXL-004, targeting malignant glioma. This marks NXL-004 as the world’s first gene therapy of its kind to enter the clinical setting.
NXL-004 leverages NeuExcell’s innovative in vivo trans-differentiation technology platform, designed to inhibit tumor cell proliferation while promoting their transformation into neural cells through the overexpression of the NeuroD1 transcription factor. This approach offers a significant advantage by minimizing side effects on healthy brain cells, in contrast to conventional treatments such as radiation or chemotherapy, which often cause collateral damage. The investigator-initiated trial (IIT) aims to assess the safety and tolerability of the AAV-NeuroD1 gene therapy in patients diagnosed with glioblastoma.- Flcube.com