Rapid advancements in gene therapy have contributed significantly to the treatments and cures for genetic disorders. In China, there are approximately 200,000 newborn babies each year with genetic disorders, 22% of which are caused by monogenic mutations. Birth defects, endocrine or metabolic diseases, nervous system diseases, and blood system diseases are the four most common genetic disorders. Additionally, gene therapies such as chimeric antigen receptor (CAR) T-cell immunotherapy have been increasingly reported as effective treatments for diseases such as cancer and HIV infection. Globally, China has registered the third most clinical trials for gene therapy.
“The first human gene therapy in China was reported in 1996. Qiu and colleagues conducted a trial in patients with haemophilia B using an ex-vivo approach, which involved autologous fibroblast cells transduced by a retrovirus that expressed the human Factor IX gene. This approach generated an observable but unsustained therapeutic effect. Since then, at least 12 ongoing gene therapy clinical trials for genetic diseases, and many more for cancers, have been approved by the Center for Drug Evaluation of China (CDE).”
“Gene therapy safety and efficacy evaluation and monitoring methods are crucial for the success of a trial. Quick, effective, and reliable methods should provide timely and useful guidance to clinicians. For example, gene therapy for haemophilia still lacks quick and reliable methods to detect the cellular immune response. The cellular immune response could lead to liver toxicity and decline of the target gene expression if left without timely intervention. Beyond efficacy, gene therapy safety remains an ongoing issue that requires improvement. New vectors with lower dose requirements and minimised immunogenicity are in demand. Accumulation of clinical findings with large-scale clinical trials and long-term follow-up should provide additional insightful guidance for future gene therapy drug development and successful clinical trial design and performance.”
Read the full editorial here.
For a more comprehensive review, you may wish to read this publication by Frontiers, or this one by Nature.
References
- Zhang, L. (2022). Gene therapy in China: past, present, and future. The Lancet Haematology, 9(12), e869–e871. https://doi.org/10.1016/s2352-3026(22)00356-8
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