Notification of Public Comment Soliciting on “Technical Guideline for the Pharmaceutical Study and Evaluation of Gene Therapy Products (Draft for Comments)”

Technical Guidelines for Pharmaceutical Research and Evaluation of Gene Therapy Products

(Draft for Comments)

Foreword

In recent years, with the continuous advancement of gene delivery systems and editing technologies, the field of gene therapy has developed rapidly, providing new treatment ideas and methods for the treatment of some intractable diseases, especially rare genetic diseases. These guidelines are formulated to regulate and guide the research, development and evaluation of gene therapy products in accordance with drug management regulations. Due to the rapid development of biotechnology, the types of gene therapy products are changing with each passing day, and there are large differences in the technical characteristics of various products. These guidelines are only based on current technological development and scientific cognition, and put forward general technical requirements for the particularity of pharmaceutical research of gene therapy products to ensure the quality, safety and effectiveness of research and therapeutic drugs. In the future, with the development of technology, deepening of cognition and accumulation of experience, these technical guidelines will be gradually supplemented and improved. At the same time, combined with the development trend of the industry, corresponding technical guidelines will be formulated for specific varieties. These guidelines are only general technical requirements for gene therapy products, providing guidance for the research and development and application of companies related to gene therapy products, and at the same time, they are also an important reference for regulatory agencies to supervise and evaluate gene therapy products. The research and development of gene therapy products should comply with the relevant laws and regulations such as the Drug Administration Law of the People's Republic of China, the Drug Registration Management Measures of the People's Republic of China, and the Pharmacopoeia of the People's Republic of China, and follow the relevant technical requirements of this guideline. If necessary, other relevant guidelines can be comprehensively referenced. Due to the rapid development and diverse categories of gene therapy products, the wide range of clinical indications covered, and the different pharmaceutical research of products at different stages of research and development and with different clinical indications, the applicability of the technical requirements of this guideline to specific varieties or research should adopt the principle of specific analysis of specific issues.

Specific news link: Notice on Soliciting Comments on the "Technical Guidelines for Pharmaceutical Research and Evaluation of Gene Therapy Products (Draft for Comments)"