Course Detail

Course Overview and Goals

Biologics are usually substances derived from living sources that are used for the prevention, treatment or cure of injuries or diseases.  Quite often these sources are eukaryotic cells in culture or micro-organisms that have been genetically modified to produce the desired recombinant product.  Examples of biologics are blood clotting factors, vaccines and therapeutic antibodies.  It has been estimated that there are over 500 different biologics currently on the market or in clinical trials and a rapidly growing bioprocessing industry to produce high quality biologics in large quantities has been developing over the last couple of years.  This course will introduce students to different types of biologics, their production, purification, analysis and regulation.  Case studies in purification and characterization of biologics as well as discovery of vaccine targets and vaccine clinical trials will be presented.

Textbooks

Biopharmaceuticals: Biochemistry and Biotechnology, by Gary Walsh, Second Edition, 2003, John Wiley & Sons. (Please check with instructor before purchasing this book.)

How the Immune System Works, by Lauren M. Sompayrac, Second Edition, 2002, Blackwell Publishing.
(For students who have not previously studied immunology, this is an excellent introduction to a very complex topic .)

• Eradication of smallpox by vaccination: From the “first” biologic through operations management of the eradication program to preparedness against bioterrorism.
• Introduction to biologics, industrial proteins and polynucleotide products.
• Downstream processing of biologics: Techniques for purification of proteins and peptides.
• Approaches to stabilization of protein based biologics. 
• Case study: Commercial production of recombinant coagulation factor IX.
• High performance liquid chromatography (HPLC) as a preparative and analytical tool.
• Reverse phase HPLC and micro parallel liquid chromatography.
• Mass spectrometry and tandem mass spectrometry.Analysis of biologics and case studies.
• International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH):  Test procedures and acceptance criteria for biotechnological/biological products. 
• Regulation of biologics: The horse named Jim and the birth of the Center for Biologics Evaluation and Research (FDA), US Code of Federal Regulations (CFR) and the Biological License Application (BLA) process, ICH Common Technical Document, etc. 
• Basic immunology of vaccination: Innate and adaptive immune responses, humoral and cell-mediated immunity, antigen presentation and antibody production, structure of antibodies, monoclonal antibodies, etc.
• Vaccines: Different types of vaccines, the changing vaccine industry and clinical trials of the blockbuster pneumococcal conjugate vaccines.
• Commercial vaccine case studies: Chiron’s flu vaccine manufacturing problems.
• Ethical issues with vaccination and the Vaccine Adverse Event Reporting System (VAERS).

At the conclusion of the course students will:

• Appreciate the importance of biologics as a class of therapeutic agents and as a market force in the pharmaceutical industry.
• Understand how biologics are designed, produced, purified and regulated.Learn some key regulatory issues associated with biologics.
• Gain knowledge about vaccines, the vaccine industry and basic immunology related to vaccination.

Assignments  60%
Biologics Group Project  40%