Time：July 08-10, 2024 

Venue：Boston, MA, USA

Sessions：Gene Therapy: Methods, Strtegies & Clinical Applications

Poster Presentation Title：

Spinal Muscular Atrophy and Pompe Disease Gene Therapy Clinical Trials in China

Presenting Author： Xiaodong Wang, Ph.D

Trained in human genetics and serving as the vice president of medical affairs at GeneCradle Therapeutics, Dr. Wang is actively involved in clinical trial protocol designs, data analysis, and results discussion with investigators.

Abstract：

Spinal muscular atrophy (SMA) and Pompe disease (PD) are both rare diseases that can be life-threatening or significantly impact the quality of life. GC101 and GC301 are AAV-based gene therapy drugs specifically designed to treat SMA and PD, respectively. Intrathecal administration of GC101 and intravenous administration of GC301 have demonstrated safety and provided clinically significant improvement in  survival, motor function, and achievement of motor milestones, which were not previously expected in  patients with SMA and PD.

ABOUT SUMMIT

Cell and gene therapy represent intersecting fields of biomedical research with the shared goal of treating, preventing, or curing diseases. These approaches are aimed at understanding the underlying causes of various diseases, ranging from rare and complex conditions to those with genetic or acquired origins. This rapidly expanding treatment approach is poised to address significant unmet medical needs across a wide range of conditions. However, the conceptual framework of cell and gene therapy is both expensive and requires precision, presenting challenges for industrial implementation and patient accessibility.

Emerging tools and techniques in the market have facilitated the adaptability and utilization of cell and gene therapy. These advancements have made it easier to employ CGT as a method of treating diseases, including rare and complex disorders, single gene disorders, and various types of cancer. With these advancements in mind, the International Summit on Cell and Gene Therapy serves as a platform to explore the fundamental concepts of these transformative therapies. The summit brings together experts from academia and industry to discuss a range of topics related to cell and gene therapy, including ethical considerations, social implications, tools, techniques, marketing strategies, and the commercialization of cell and gene therapy products. It also showcases the latest research progress in CGT and explores opportunities for further enhancements in availability and productivity of these therapies. By fostering collaboration and knowledge exchange, the summit aims to advance the field of cell and gene therapy, benefiting patients worldwide.