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VGN-R09b, China's First Domestically Developed Gene Therapy Drug for the Treatment of Primary Parkinson's Disease, Approved for Clinical Trials in the United States

2024-07-26

VGN-R09b is the world's first AAV gene therapy product to be approved for Parkinson's disease clinical research in both China and the U.S. Its registered Phase 1/2 clinical trial for primary PD was successfully initiated earlier this month at Huashan Hospital, Fudan University. The approval of VGN-R09b for clinical trials in the U.S. represents an important milestone for the Company in realizing its global synchronous R&D strategy, bringing the development of gene therapy drugs in the field of non-rare diseases to the world.

 

Dr. Zhao Xiaoping, CEO of the Company, commented, “The FDA's consent to conduct clinical trials of VGN-R09b for primary Parkinson's disease in the U.S. represents full recognition of our product's early R&D strategy and research data, as well as our GMP manufacturing platform. We expect VGN-R09b to deliver benefits to more patients around the world with its favorable safety profile and superior efficacy. “

 

About Parkinson's Disease

 

Parkinson's disease is the second most common neurodegenerative disorder, and its prevalence is increasing more rapidly than many other neurodegenerative disorders. It is estimated that the global prevalence of Parkinson's disease will double in the future, with the number of patients rising from 6.9 million in 2015 to 14.2 million in 2040. Approximately 1% of the population over the age of 65 and 4% of the population over the age of 85 will be affected.

 

About VGN-R09b

 

VGN-R09b is a gene therapy product self-developed and manufactured by the Company. VGN-R09b uses recombinant AAV as a carrier to deliver dual genes simultaneously so as to enhance dopamine synthesis in the brain while promoting neuroprotection. It effectively improves the clinical symptoms of patients with primary Parkinson's disease while slowing down the progression of the disease, thus improving the long-term prognosis. By local striatal injection, the drug can be applied directly to the site of disease, reducing the dose administered systemically and correspondingly leading to a decrease in adverse effects such as immune reactions. Theoretically, gene therapy requires only one injection to have long-term therapeutic effects.

 

VGN-R09b received the “Clinical Trial Approval Notice” issued by the National Medical Products Administration on April 19, 2024. The first subject was dosed in the registered clinical study of this product for treatment of aromatic L-amino acid decarboxylase deficiency (AADCD) on July 2, 2024 at the Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine. The registered clinical study for primary Parkinson's disease (PD) was also initiated on July 5, 2024 at Huashan Hospital, Fudan University.