On August 15, 2025, Shanghai Vitalgen BioPharma Co., Ltd. (hereinafter referred to as "Vitalgen") announced that its independently developed innovative gene therapy VGN-R13 for the treatment of amyotrophic lateral sclerosis (ALS) has achieved significant progress in an early exploratory clinical study conducted at Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology. The study has completed enrollment of all subjects in the first dose cohort, demonstrating encouraging clinical results.
This investigator-initiated trial (IIT) is led by Professor Chen Hong, Director of the Department of Rehabilitation Medicine at Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology, President of Tongji Rehabilitation Hospital, Director of the Stem Cell Research Center, and Director of the Rehabilitation Branch of Hubei Rare Disease Center, in collaboration with Professor Zhang Min, a leading expert in the ALS field. The study aims to evaluate the safety and preliminary efficacy of VGN-R13 in patients with ALS. Study data indicates:
Favorable Safety Profile: All three subjects in the first dose cohort have completed at least four weeks of follow-up, with the first subject having completed 26 weeks of long-term follow-up. No dose-limiting toxicity (DLT) events have been observed, and no adverse drug reactions related to VGN-R13 have been reported, preliminarily confirming the favorable safety and tolerability profile of the investigational product.
Clear Efficacy Signals: Potential clinical benefits associated with VGN-R13 have been observed across analyses of both subjective and objective measures, including clinical functional rating scales, pulmonary function parameters (vital capacity), and key biomarker assessments. These findings are consistent with preclinical data, which demonstrated that intrathecal administration of VGN-R13 significantly improved motor function and extended median survival in an ALS mouse model.
Dr. Zhao Xiaoping, Chairman of Vitalgen, stated, "The results from this IIT study mark a significant milestone as the first-in-human study of VGN-R13 in patients with sporadic ALS. We will accelerate the R&D process, striving to deliver a breakthrough treatment option for patients with ALS as soon as possible."
About VGN-R13
VGN-R13 is a gene therapy product independently developed by Vitalgen. It is a single-dose, adeno-associated virus (AAV)-based gene replacement therapy for the treatment of amyotrophic lateral sclerosis (ALS). Administered via intrathecal injection, VGN-R13 transduces neurons in the spinal cord and brain, degrading pathological aggregates within the neurons of patients with ALS and preventing neuronal apoptosis induced by these aggregates. Additionally, the product plays a crucial role in axonal growth and structural repair of neurons, thereby reducing neuronal apoptosis while repairing neuronal damage. This dual mechanism is intended to slow disease progression and restore impaired motor function in patients with ALS.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a rare, progressive, and fatal neurodegenerative disorder characterized by the gradual loss of motor neurons in the brain and spinal cord that control voluntary muscle movement. The disease primarily manifests clinically as progressive skeletal muscle weakness, atrophy, fasciculations, bulbar palsy, and signs of upper motor neuron involvement. The condition progresses relentlessly, ultimately leading to death due to respiratory muscle paralysis or complicated by respiratory infections. The average life expectancy for patients with ALS from symptom onset is three to five years. Recognized by the World Health Organization as one of the five most intractable diseases, there remains no curative treatment available worldwide.