PackGene Supports NeueXcell Therapeutics’ NXL-001 Alzheimer’s Gene Therapy Clinical Milestone

HEFEI, China – June 12, 2025 –

PackGene congratulates NeueXcell Therapeutics and investigators at the First Affiliated Hospital of the University of Science and Technology of China (Anhui Provincial Hospital) on a significant clinical milestone: the successful completion of dosing and one-month safety follow-up for the first participant in an investigator-initiated clinical trial (IIT) evaluating NXL-001, an investigational AAV gene therapy for severe Alzheimer’s disease (AD).

This achievement represents a historic first: the world’s initial clinical application of in vivo neuronal regeneration therapy in patients with severe Alzheimer’s disease, potentially opening a new therapeutic avenue for patients facing one of medicine’s most challenging neurodegenerative disorders.

Advancing Hope for Patients with Severe Alzheimer’s Disease

Alzheimer’s disease accounts for approximately 60–80% of dementia cases worldwide and remains one of the leading causes of disability and mortality among older adults. Progressive neuronal loss leads to memory decline, cognitive impairment, and loss of independence. Despite recent therapeutic advances, current treatments have limited ability to restore damaged neural networks or reverse disease progression.

For patients with advanced disease, treatment options remain particularly limited, underscoring the urgent need for innovative therapeutic strategies.

The NXL-001 clinical study is led by Professor Jiong Shi from the Department of Neurology at the First Affiliated Hospital of USTC, with administration procedures performed by neurosurgical teams led by Professor Ruobing Qian and Professor Yinbao Qi. Through extensive multidisciplinary coordination and detailed preoperative planning, investigators successfully completed dosing of the first participant.

Encouragingly, during the first month following treatment, the participant recovered well, with investigators reporting no drug-related serious adverse events or dose-limiting toxicities observed during the safety follow-up period.

The successful completion of first-patient dosing and follow-up establishes an important foundation for this globally pioneering study and marks a meaningful step forward for neuronal regeneration technologies entering clinical evaluation.

A Novel Regenerative Approach to Alzheimer’s Disease

Unlike conventional therapies that primarily target symptoms or disease-related protein accumulation, NXL-001 was developed using Professor Gong Chen’s pioneering in vivo neuronal regeneration platform, designed to directly regenerate functional neurons within the brain.

NXL-001 uses an AAV vector to deliver the neural transcription factor NeuroD1 into brain astrocytes, inducing their conversion into functional neurons. By transforming resident support cells into neurons, the therapy aims to regenerate damaged neural circuitry and restore neurological function.

Preclinical studies demonstrated that NeuroD1-mediated therapy successfully regenerated neurons within Alzheimer’s disease models, repaired neural networks, and improved learning and memory performance.

PackGene Supports Translation from Research to Clinical Development

PackGene is honored to support NeueXcell’s NXL-001 program through its integrated end-to-end CDMO platform, providing services spanning plasmid development through AAV manufacturing support to help accelerate clinical advancement.

As innovative gene therapies increasingly transition from scientific concept to patient treatment, manufacturing quality and translational readiness remain essential to successful clinical progress.

PackGene remains committed to enabling partners with scalable gene therapy solutions and supporting the advancement of transformative therapies that may one day provide new hope for patients and families affected by devastating diseases.

Behind every first-in-human milestone is not only scientific innovation—but also patients and families waiting for meaningful change.