Successful First Patient Dosing of AAV-NeuroD1 Gene Therapy for Glioblastoma by NeuExcell Therapeutics

NXL-004 is an innovative treatment approach developed based on Professor Chen Gong’s pioneering in situ differentiation technology platform. It is notably the first AAV gene therapy product for malignant glioblastoma to receive FDA orphan drug designation. The therapy targets glioblastoma cells by overexpressing the neurogenic transcription factor NeuroD1, inhibiting tumor cell proliferation while promoting their differentiation into neurons. This mechanism offers potential advantages over conventional treatments such as radiotherapy and chemotherapy, with minimal side effects on surrounding tissues.

The initial patient, suffering from recurrent glioblastoma, underwent surgery and dosing without complications. Postoperative observations revealed a stable condition with no significant adverse reactions, culminating in a smooth recovery and discharge within a week. This successful administration of AAV-NeuroD1 gene therapy represents a groundbreaking leap from preclinical research to clinical application, offering hope for improved treatment outcomes in glioblastoma patients.

Professor Huang Yulun expressed optimism regarding the therapy’s safety and efficacy, citing promising preclinical data and the favorable safety profile observed in the first clinical trial subject. Likewise, Professor Chen Gong, the founder of NeuExcell Therapeutics, commended Professor Huang Yulun’s team for their collaboration and surgical expertise, underscoring the significance of this milestone in advancing neurological disease treatments.

The successful dosing of NXL-004 at Suzhou Dushu Lake Hospital underscores the potential of gene therapy in revolutionizing the management of malignant glioblastoma, addressing the urgent need for more effective treatment options in neuro-oncology. This achievement serves as a testament to the collaborative efforts of researchers and medical professionals in pushing the boundaries of medical science for the benefit of patients worldwide.

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