Proclamation of the “General Instructions on Human Gene Therapy Products (Draft)”

General Introduction to Gene Therapy Products for Human Use (Draft for Public Notice)

Overview

Gene therapy products are usually composed of vectors or delivery systems containing engineered gene constructs. Their active ingredients can be DNA, RNA, genetically modified viruses, bacteria or cells. By introducing exogenous genes into target cells or tissues, specific genes are replaced, compensated, blocked or corrected to achieve the purpose of treating diseases.

Depending on the different vectors, gene therapy products can be divided into gene therapy products with viruses as vectors, gene therapy products with plasmid DNA as vectors, and gene therapy products with bacteria as vectors. Among them, gene therapy products with viruses and plasmid DNA as vectors are the most common.

This general introduction is a general technical requirement for the production and quality control of gene therapy products, focusing on gene therapy products with viruses and plasmid DNA as vectors. Vectors used for gene-modified cells (autologous or allogeneic somatic cells modified in vitro or in vitro with gene therapy vectors before being introduced into subjects or patients) are also applicable to this general introduction. Specific varieties should also formulate relevant requirements in combination with the characteristics of the products themselves.

Specific news link: Public announcement of the draft general principles for gene therapy products for human use