Source: Center for Drug Administration (CDE)

On September 12, 2023, the Center for Drug Evaluation (CDE) of the State Drug Administration publicly solicited opinions on the Technical Guidelines for Clinical Trials of Gene Therapy Products for Rare Diseases (Draft for Comment).

On January 18, 2024, the Center for Drug Evaluation (CDE) of the State Food and Drug Administration officially issued the "Technical Guidelines for Clinical Trials of Gene Therapy Products for Rare Diseases", which will take effect from the date of release. The guideline aims to put forward suggestions for the clinical research and development of rare disease gene therapy products in combination with the characteristics of rare diseases and gene therapy products, and provide references for the clinical trials of rare disease gene therapy products.

The "China Rare Disease Definition Study Report 2021" defines rare diseases as "the incidence of newborns is less than 1/10,000, the prevalence is less than 1/10,000, and the number of patients is less than 140,000; A disease that meets one of these criteria is a rare disease." About 80% of rare diseases are caused by single gene defects, and gene therapy is one of the important means to treat rare diseases. As of January 2024, 176 types of diseases have been registered in China's National Military Disease Registration system.

Gene therapy products refer to products that achieve therapeutic purposes by modifying or manipulating the expression of genes or changing the biological characteristics of living cells. The main mechanism of action includes the replacement of disease-causing genes by normal genes, the inactivation of genes that do not work normally or the introduction of new or modified genes. The use of gene therapy products for the treatment of rare diseases is mainly considered to treat the cause of the disease, with a view to achieving the purpose of permanent or semi-permanent treatment of the disease. At present, the gene therapy products in China are mainly concentrated in the fields of nervous system diseases, metabolic diseases, blood diseases, eye diseases and so on.