News
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.