April 28 - Day 1Tuesday, 28 April 2026 - CT (China Time, GMT+08:00)

• Act Early, Transform Lives: The transformative, long-lasting impact of autologous HSC gene therapy and the importance of pre-symptomatic intervention enabled by Newborn Screening.
• From Rare to Prevalent: The evolution from single-gene rare diseases toward scalable therapeutic platforms with potential in higher-prevalence indications.
• Scaling the Vein-to-Vein Journey: The operational roadmap required to enable high-volume delivery and COGs reduction, including DP automation, vector scale-up, and expansion of specialized treatment centres.
• Navigating Financial and Regulatory Complexity: How the biotech industry can progress despite funding constraints, market volatility, regulatory complexity and global supply-chain challenges.

• The strategic value of IITs in China's evolving regulatory landscape - why foreign sponsors should consider this pathway for CGTs
• Key regulatory considerations: IND vs IIT pathways, institutional ethics committee requirements, and engagement strategies
• Selecting the right clinical sites: evaluating PI expertise, institutional capabilities, and patient access for rare disease indications
• Manufacturing and supply chain: importing investigational products, managing cold chain logistics, and meeting Chinese cGMP expectations
• Building effective partnerships: working with local CROs, navigating hospital administrative structures, and managing cross-cultural communication
• Common pitfalls and how to avoid them: regulatory missteps, timeline miscalculations, and data quality issues
• Case examples from AAV gene therapy programs

Details of the clinical development and regulatory communication for IM19, a novel CD19 CAR-T product under NMPA review. We will highlight the molecule design guided by clinical unmet needs and its demonstrated advantages, including a >70% ORR with low rates of severe CRS/ICANS.