Global Regulatory Requirements and Special Considerations for Gene Therapy

With the progress of precision medicine and the development of biopharmaceutical technology, development and investment into gene therapy drugs is increasing. Gene therapy drugs are opening new doors for the treatment of many genetic diseases and are also offering new approaches to the treatment of chronic and infectious diseases. However, since these drugs are relatively new to the market, there are still many technical bottlenecks and clinical trial challenges for us to break through and overcome. For example, how to facilitate the clinical translation of gene therapy technology needs further exploration; in addition, understanding the regulatory process of gene therapy products and how to deal with the regulatory differences between different regions or countries is important to planning a successful trial. Through the discussion of early development, registration and clinical trial management, we can help sponsors to develop a more efficient product development plan, leading to more effective novel treatment options.

Dr. Gao Guang, CMC Head, Executive Director, Caidya

• 20 years of extensive CBER FDA drug review and regulatory experience, as well as regulatory experience in compliance/regulatory perspective inspections, compliance and GMP regulations, extensive FDA CMC review experience.
• Extensive experience in drug and device product development and regulatory approval.
• Understanding of FDA registration filing requirements for drug and medical device products, with extensive experience in filing preparation and FDA communication; preparation of US FDA communication meetings, and handling of various types of review related matters.
• Comprehensive understanding of the Chinese review and registration system and WHO pre-qualification process for vaccines, chemical drugs, biologics, biosimilars and medical devices.
• Conducting foreign drug investigations/inspections for Chinese companies, evaluating the regulatory and oversight procedures of the Chinese drug regulatory authorities, and ensuring that exported products meet US FDA standards.