Gene Therapy Platform Technologies

Overview

Modular approach to gene therapy development using shared manufacturing infrastructure, analytical methods, and regulatory packages across multiple disease programs, typically changing only the transgene while keeping the vector backbone and process constant. Gene therapy platform technologies enable reuse of data and manufacturing modules across different diseases: starting from a common vector backbone (lentiviral or AAV), only the transgene is changed while the rest of the manufacturing process remains the same. Platform technologies in gene therapy allow shared analytical testing methods across multiple products and diseases, reducing development time and costs by leveraging common knowledge accumulated across programs. (Segment 5 (20:00-25:00))