HEFEI, China – June 2025 – NeuExcell Therapeutics announced successful dosing of the first patient, who has had Alzheimer's disease (AD) for ten years, to evaluate the safety and efficacy of its proprietary neuroregenerative gene therapy product NXL-001. This study is led by Principal Investigator (PI) Professor SHI Jiong in the Department of Neurology, First Affiliated Hospital of USTC (Anhui Provincial Hospital) in Hefei, China. The sophisticated surgical procedures were successfully executed by a highly coordinated, multidisciplinary neurosurgical team led by Professor QIAN Ruobing and QI Yinbao. Assessment at the one-month follow-up showed that the patient was well recovered, with no drug-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs). This study represents the world's first application of in situ neuroregenerative gene therapy in a patient with severe Alzheimer's disease, offering new hope for millions of AD patients worldwide.
About Alzheimer's Disease:
Alzheimer’s disease (AD) is one of the most common neurodegenerative disorders, accounting for approximately 60-80% of all dementia cases. It is a leading cause of disability and death in the elderly. AD is characterized by significant neuronal loss and progressive cognitive decline. Currently, there is no effective treatment capable of regenerating millions of new neurons in a patient’s brain, making the disease difficult to cure. This underscores the urgent need to develop novel and more effective therapeutic treatments.
About NXL-001:
NXL-001 is an investigational therapeutic agent developed based on the in situ neuroregeneration technology platform pioneered by Professor Gong Chen's team. Utilizing an AAV vector, NXL-001 delivers the neural transcription factor NeuroD1 to astrocytes in the brain, inducing their direct reprogramming into functional neurons. In preclinical studies, NeuroD1 demonstrated the ability to regenerate functional new neurons, repair damaged neural circuits, and improve cognitive functions in AD animal models.
About NeuExcell Therapeutics:
NeuExcell Therapeutics is a clinical-stage biotechnology company dedicated to developing next-generation therapies to treat neural injuries and neurodegenerative diseases through its proprietary in situ neural regeneration platform AtN ASTROCYTES-TO-NEURONSTM technology. By leveraging this cutting-edge technology, NeuExcell aims to provide transformative solutions for the treatment of neurological disorders such as Alzheimer’s disease, stroke, and glioma. For more information, please visit www.neuexcell.com .