Next-Gen Therapeutics: Advanced Tools & Technologies in Drug Development

Catalog description

The emergence of cell and gene therapies is closely linked to rapid developments in the biotechnology research environment. Following FDA approval of adeno-associated virus (AAV) for therapeutic gene delivery, efforts to apply it in CRISPR-based gene editing have quickly moved to clinical trials. The demand for expertise for these and related technologies has never been higher. This course uses case studies to explore how experimental theory and quantitative modeling drive the development of next-generation medicines. Participants will gain practical experience in the application of quantitative principles for massively scaling experimental models. Topics include discovery and engineering of viral vectors (AAV), high throughput experimental design of gene editing platforms (CRISPR), advanced molecular assays, and quantitative analysis of barcoded DNA libraries by next-generation sequencing.