Hefei, China, March 5, 2025 — NeuExcell Therapeutics, a biotechnology company dedicated to pioneering neuroregenerative gene therapies, announced the initiation of a first-in-human clinical trial evaluating its investigational gene therapy, NXL-001, to treat Alzheimer’s disease (AD). The study is being conducted at the First Affiliated Hospital of the University of Science and Technology of China (Anhui Provincial Hospital) under the leadership of Prof. Jiong Shi (Department of Neurology, Principal Investigator), with strong support from Prof. Ruobing Qian and Prof. Yinbao Qi (Department of Neurosurgery).
This investigator-initiated trial (IIT) represents a major milestone as the world’s first clinical study of a gene therapy leveraging in situ neural regeneration technology to address neurodegeneration in Alzheimer’s disease. The trial aims to assess the safety, tolerability, and preliminary efficacy of NXL-001 in AD patients, offering a potential paradigm shift in the treatment of this debilitating disorder.
A Novel Approach to Alzheimer’s Disease Treatment
Alzheimer’s disease is one of the most prevalent neurodegenerative disorders, accounting for 60-80% of dementia cases worldwide, and remains a leading cause of disability and mortality among the elderly. Current treatment options primarily provide symptomatic relief without addressing the progressive neuronal loss that drives cognitive decline.
NXL-001, a proprietary gene therapy candidate independently developed by NeuExcell Therapeutics, is designed to directly reprogram astrocytes into functional neurons within the brain. This approach aims to restore lost neuronal function and potentially halt or reverse disease progression. “The irreversible loss of neurons is the fundamental pathology of Alzheimer’s disease, yet no existing therapies directly address this issue,” said Prof. Jiong Shi, Principal Investigator. “NXL-001 represents a transformative approach by regenerating functional neurons directly within the brain, providing a novel therapeutic strategy with the potential to redefine AD treatment.”
A Decade of Scientific Innovation Leading to Clinical Translation
The clinical trial launch marks a critical step in the translation of cutting-edge gene therapy research into clinical applications.“Since our 2013 breakthrough study published in Cell Stem Cell, which first demonstrated astrocyte-to-neuron reprogramming in Alzheimer’s disease models, our team has continued to advance this technology to achieve widespread neuronal regeneration and functional recovery,” said Prof. Gong Chen, Co-founder and Chairman of NeuExcell Therapeutics. “The initiation of this first-in-human trial represents a significant milestone in our mission to develop innovative therapies for neurodegenerative diseases.”
About NeuExcell Therapeutics
NeuExcell Therapeutics is a clinical-stage biotechnology company dedicated to developing next-generation gene therapies for neurodegenerative diseases through its proprietary in situ neural regeneration platform. By leveraging cutting-edge astrocyte-to-neuron reprogramming technology, NeuExcell aims to provide transformative solutions for conditions such as Alzheimer’s disease, stroke, and Huntington’s disease.
For more information, please visit www.neuexcell.com.