This UCSF-Stanford CERSI mini-course introduces key concepts in therapeutic drug discovery, focusing on large molecule discovery. The first lecture provides an overview of therapeutic target considerations, including what makes a large vs. small molecule target. The role of human genetic evidence as well as in vitro and in vivo biology studies in target validation will be discussed. The second lecture addresses large molecule therapeutic development. Large molecule modalities presented will include native proteins, peptides, muteins, antibodies, antibody drug conjugates and alternative scaffolds. There will also be a focus on antibody structure and function. The third lecture will discuss manufacturing considerations for large molecules, including common manufacturing platforms utilized in the biopharma industry. The case study will present an actual pre-clinical therapeutic antibody discovery and development program, and discuss decision points and potential pitfalls along the pathway to developing a therapeutic.
- Describe differences between target classes and tractability for either large or small molecule development
- Describe the types of tool molecules required for target validation
- List the types of information and data required to pursue a potential therapeutic target
- Describe different large molecule modalities and when they are used
- Describe properties of antibodies that enable therapeutic development (i.e. why are antibodies such good drugs (PK, effector function, etc))
- Explain why manufacturability matters to the overall drug development process
- Discuss what makes a good target product profile
- List some common concerns regarding large molecule production
John Delaney, PhD, Executive Director, Biologics Discovery, Amgen Inc.
Ai Ching Lim, PhD, Director, Biologics, Therapeutic Discovery, Amgen Inc.