Cell, Gene, and Immune Therapies

The emerging therapeutic landscape has a new cast of characters' engineered cells, programmable nucleic acids, and multi-valent antibodies' to name a few. This lecture-based course will provide an overview of these new therapeutic modalities, the basic science guiding their development, and a discussion of new regulatory and safety challenges that emerge in these modalities. As a final project, students will produce a report spanning the preclinical and clinical development of a new therapy. Examples include CRISPR-edited cell therapies, bispecific T cell engagers, in vivo CRISPR base editors, and antisense oligo therapies.