Wednesday, August 21:

Preparation of a Successful Initial IND Application

Bruce K Burnett, Ph.D., RAC (US, EU), Director, Regulatory Affairs, Duke University

This course will provide a broad overview of the entire IND process and is designed for individuals involved in any aspect of the drug development process including R&D, manufacturing, clinical and regulatory. Attendees will review all of the filing components that are required for a successful initial IND application, with specific emphasis on:

• IND-enabling preclinical safety studies
• Manufacturing information for a Phase 1 investigational drug or biologic
• Clinical protocol, Informed Consent, and Investigator Brochure
• Required FDA forms 1571, 1572, and 3674
• PDUFA requirements for electronic submissions using the CTD format
• Meetings with FDA including Pre-IND meeting and meetings to discuss Clinical Hold issues
• Registration with ClinicalTrials.gov

Dr. Burnett brings over 25 years of experience in the pharmaceutical industry involving research and development, scientific affairs, quality control/assurance, and regulatory affairs. He came to Duke from AlphaVax, where he last served as Vice President of Quality and Regulatory Affairs. He has also held both regulatory and quality positions at Biogen Genetics Institute, and Serono.  Dr. Burnett received his undergraduate degree in chemistry from the University of California, San Diego and his PhD in chemistry/biochemistry from MIT working in the laboratory of Nobel Laureate Dr. Har Gobind Khorana.  Dr. Burnett’s regulatory experience includes working on license applications that have resulted in the US approval of Tysabri (natalizumab), Amevive (alefacept), Neumega (oprevelkin, IL-11) and Benefix (coagulation Factor IX). He has also been responsible for preparing and submitting multiple initial INDs to CBER or CDER, preparing for many pre-IND and End of Phase 2 meetings, and leading numerous teleconferences with the Agency reviewers.

Thursday, August 22:

Introduction to Protein Characterization Technologies for Biologics Development

Christine P. Chan, Ph.D., Senior Manager, Technology Development, Genzyme Corporation

This course covers the fundamentals of protein structural analysis using modern biochemical and biophysical technologies. Analytical methods commonly used for CMC analytical characterization, release and stability studies of biotechnology products are reviewed with practical examples. Application considerations from early-phase development to commercialization are also discussed.

• Introduction to protein structure and post-translational modifications
• Protein Chemistry Techniques: capillary electrophoresis and HPLC, enzymatic methods and peptide mapping, mass spectrometry
• Biophysical Characterization: spectral methods, light scattering, calorimetry, analytical ultracentrifugation, plus aggregation, subvisible and visible particles analysis
• Binding and cell-based potency assays, activity assays, impurity analysis
• Bioprocess impact on product quality, conducting forced degradation studies
• ICH guidance documents, testing strategy through the product lifecycle, process control strategy considerations

Christine Chan is a protein biochemist with broad experience in the biopharmaceutical industry, including prior experience at Sandoz Pharmaceuticals and Merck & Co., Inc. She specializes in the analysis of recombinant products produced from mammalian cells for vaccines and biologics development. She has extensive hands-on experience with classical protein chemistry methods including Edman sequencing, amino acid analysis and protein purification, as well as capillary electrophoresis, mass spectrometry and biophysical methodologies. She is experienced in enzymatic assays, immunoassays as well as cell-based assays. Dr. Chan obtained her Ph.D. from the University of California-Davis and did postdoctoral work at the Howard Hughes Medical Institute at the University of Washington on growth factor signal transduction and protein phosphorylation.

Introduction to Biopharmaceutical Upstream and Downstream Separation, Clarification and Purification Processes

A. Mark Trotter, Development Manager, Life Sciences Purification Technology, 3M Purification, Inc.

This instructional program will review the major upstream and downstream technologies and applications used to produce biomolecular drug products, e.g., mAbs, vaccines, and genetic therapeutics.  From the bioreactor/fermenter off load to the final dosage formulations, each process step will be examined with regard to technology, equipment/instrumentation and typical applications. These processes include depth filtration, tangential flow filtration (TFF), chromatography steps (both up and downstream), and viral clearance processes. The class will provide insights into the basic unit operations with focus on equipment and application.

• Basic concepts of clarification and purification
• Upstream and downstream unit operations for clarification and purification
• Review of various process steps in these applications
• Technology and equipment used with each unit operation: depth, tangential flow and final filtration steps, basic chromatographic and viral clearance processes
• Relationship with other unit operations
• Scale-up considerations

Mark Trotter has twenty-five years experience in biopharmaceutical industries, from work in pharmacologic research as project leader to field sales, and technical service director. This extensive background is coupled with an in-depth regulatory knowledge that supports his expertise in process validation.  He completed his post-graduate studies at Long Island University, C.W. Post College, earning his MS in Medical Microbiology and continuing on for his MBA in Finance.  He is considered a subject matter expert in upstream to downstream processes. He has published numerous technical articles, book chapters and has contributed editorial comment on these subjects.