Dr. Le Cong is Associate Professor of Pathology and Genetics at Stanford University, where his lab pioneers the intersection of genome engineering and artificial intelligence for biomedical discovery. A co-inventor of foundational CRISPR/Cas9 gene-editing tools for in vivo gene therapy, his group develops advanced technologies for large-scale genome editing and cell therapу. Recent innovations include RNAGenesis, a foundation model for generative RNA design; CRISPR-GPT, an Al agent that automates complex gene-editing workflows; and LabOS, an AI-XR co-scientist platform bridging computation and physical experimentation. Dr. Cong is a recipient of the NHGRI Genomic Innovator Award and a Baxter Foundation Faculty Scholar.

This session explores how artificial intelligence (AI) and machine learning (ML) are transforming preclinical development of gene and cell therapies. Topics include Al-driven CRISPR optimization and guide RNA design, computational AAV vector and construct engineering, ML-based safety and toxicology risk prediction, and Al-powered target identification using multi-omics data. A special focus will be given to CRISPR-GPT, an Al agent system automating gene-editing workflows, and practical guidance on how academic labs can integrate accessible Al tools into their programs. The session will also address how Al can shorten development timelines and reduce attrition before clinical translation, including a look at the evolving FDA and EMA frameworks for Al-generated preclinical evidence and what this means for your IND package.