Reforgene presents at the 4th China Gene Therapy Industry Development and Product Quality Control Forum

On October 18-19, 2019, the "2019 China Gene Therapy Industry Development and Product Quality Control Forum", jointly hosted by the Management Committee of the Daxing Biomedical Industry Base of Zhongguancun Science Park, the National Institutes for Food and Drug Control and the Zhongguancun Pharma Valley Biotech Industry Research Institute, was held at the National Institutes for Food and Drug Control in Beijing. The forum focused on the whole process of gene therapy drug R&D, adhered to problem-oriented and demand-oriented principles, and invited experts from enterprises and institutions in the gene therapy and cell therapy fields to share AAV vector-based in vivo gene therapy, LV vector-based ex vivo gene-modified cell therapy, and oncolytic virus product development based on modified viruses such as HSV. It paid attention to the problems and guiding principles faced internationally in CMC, non-clinical research and clinical trials of gene therapy products, and to multiple topics including product principle design, molecular design, manufacturing processes, quality methods and standards, quality control, as well as professional technologies and specifications in animal model efficacy evaluation, safety evaluation, clinical protocols and registration applications. The forum set up a main report session and several sub-forums.

The gene therapy industry is currently ushering in good development opportunities, but still faces challenges on multiple fronts, including: the production technology of gene therapy vectors urgently needs improvement, requiring more efficient and standardized manufacturing processes and quality control technologies; the production of GMP-grade viral vectors has become a bottleneck in moving gene therapy from basic research to clinical application. Most gene therapies currently in the clinical stage use viral vectors for gene delivery, and viral vector gene drugs, due to their complex drug structure, pose higher challenges to production, especially large-scale production and quality control. The insufficient large-scale preparation capacity of gene therapy vectors seriously restricts the process of gene therapies transitioning from the clinical trial stage to commercial production.

The number of participants in this meeting exceeded expectations, reflecting the widespread attention the gene therapy industry is receiving and its good development opportunities. The meeting was addressed by Li Bo, Director of the National Institutes for Food and Drug Control, and He Rui, Deputy District Mayor of Daxing District, Beijing, and invited speakers including Rao Chunming, Director of the Recombinant Drug Division of the Institute for Biological Product Control of the National Institutes for Food and Drug Control, Professor Xiao Xiao of the University of North Carolina, Professor Wang Wenya of Peking University and Professor Wu Xiaobing of the Beijing Ruixi Rare Disease Gene Therapy Research Institute. Dr. Liang Junbin of Reforgene was invited to participate and delivered a report titled "Efficient Gene Editing Treatment of Beta-Thalassemia". The report introduced the current status and hazards of thalassemia in China, global drug development progress, and related technical strategies, while focusing on introducing Reforgene's R&D progress on the disease to the participants, with the hope that the therapy can enter the clinical stage as soon as possible to serve patients.