Gene Therapy for Rare Diseases

Overview

Use of viral vectors to deliver corrective genes into patient cells (especially CD34 hematopoietic stem cells) to treat genetic rare diseases, requiring translational expertise from academic discovery through manufacturing, regulation, and commercialization. Cell and gene therapies are inherently biologically different from conventional medicines because they use living cells with intrinsic batch-to-batch variability, making standardization and consistency a fundamental manufacturing challenge. The conventional pharma system was not designed for gene therapies because they are one-time curative treatments with high upfront costs, unlike traditional chronic medicines where cost is diluted over time. (Segment 2 (05:00-10:00))