NeuExcell Therapeutics Announce First Patient Dosed in Clinical Trial for Alzheimer’s Gene Therapy

HEFEI, China – June 12, 2025 – NeuExcell Therapeutics, in collaboration with The First Affiliated Hospital of the University of Science and Technology of China (Anhui Provincial Hospital), has successfully dosed the first patient in an IIT (Investigator-Initiated Trial) clinical study for NXL-001, an innovative gene therapy for Alzheimer’s disease (AD). The patient has completed a one-month safety follow-up visit. This marks the world’s first application of an in situ neural regeneration therapy for severe Alzheimer’s disease, offering new hope for patients.

The study is led by Professor Shi Jiong of the hospital’s Neurology Department, with the precise administration of the therapy performed by the surgical team of Professors Qian Ruobing and Qi Yinbao from the Neurosurgery Department. The research team conducted thorough pre-operative preparations and successfully completed the first dosing with excellent surgical skill. Following the one-month safety visit, the patient is recovering well, with no drug-related serious adverse events or dose-limiting toxicities observed.

This successful first patient dosing lays a strong foundation for this groundbreaking clinical study and marks a significant step in the clinical advancement of in situ neural regeneration technology.

About NXL-001

NXL-001 is an innovative therapeutic developed from Professor Gong Chen’s pioneering in situ neural regeneration technology platform. NXL-001 uses an AAV vector to deliver the neural transcription factor NeuroD1 to astrocytes in the brain, inducing their trans-differentiation into functional neurons. In preclinical studies, NeuroD1 was shown to regenerate neurons in situ, repair damaged neural circuits, and improve learning and memory in AD animal models.

As a partner to NeuExcell Therapeutics, PackGene Biotech extends its warm congratulations on this important progress. PackGene Biotech is honored to have assisted in advancing NeuExcell Therapeutics’ AAV gene therapy drug by providing one-stop CDMO services, from plasmid to AAV manufacturing, for the NXL-001 clinical study.