CB 309qc

This year, our biotech/drug development course will focus on how a novel therapeutic modality has grown from a scientific curiosity to a promising and indeed proven therapeutic approach.  Antisense nucleotides (ASOs) define along with small molecule drugs and biologicals (antibodies) a new therapeutic modality.  The efficacy of this modality, the cell biology and chemistry of ASOs as new drug, will be discussed in the context of an ASO that has been shown to be efficacious in addressing two devastating diseases: Spinal Muscular Dystrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS). We will also address the critical issue of clinical trials, including their design, the criteria of success and, using as a paradigm an antibody that in spite of early promise has yet to fulfill the criteria necessary to address Alzheimer’s , a disease that unlike SMA where patient numbers are relatively small, it affects hundreds of thousands if not millions of patients worldwide. This Course Begins in mid-March, and highlights different topics each week that will illustrate how investigation of basic principles and phenomena in cell and molecular biology open important doorways to understanding of disease mechanisms and how such knowledge can be translated into drug development and avenues to commercialization. A lively in depth discussion is the core objective for students in the course; thus the assessment will be entirely based on in-class participation.