Note: Following are links to the presentation slides from faculty members who gave ASGCT authorization to post them on this web page.

May 17, 2010

Welcome
Co-Chairs: Katherine High, MD & Helen Heslop, MD

Session I: Planning for a Clinical Trial
Moderator: Andra Miller, PhD
Speakers:

• What is an IND? - Kimberly Benton, PhD
• CMC Requirements for Early Phase Gene and Cell Therapy Clinical Trials - Ramjay Vatsan, PhD
• What Vector and Cell Resources Are Available?
• Production Assistance for Cellular Therapies (PACT) - Traci Mondoro, PhD
• NHLBI Gene Therapy Resources - Sonia Skarlatos, PhD
• Resources Offered through the Association of Academic Biologics Manufacturers (AABM) - Johannes C.M. van der Loo, PhD
• Contracted Manufacturing for Cell Therapy - Minako Koga

Session II: Preclinical Development
Moderator: Stephanie Simek, PhD
Speakers:

• Preclinical Considerations for Cell and Gene Therapy Products: CBER Perspective - Mercedes Serabian, MS
• Preclinical Biodistribution of Cell and Gene Transfer Vectors - Edwin Horwitz, MD, PhD
• Preclinical Toxicology: Combining Novel Designs with Traditional Principles
• Case Study: How Do You Know When Your Preclinical Work in Cell Therapy is Ready for the Clinic? – Jeffrey Miller, MD
• A Phase I Pilot Study of Safety and Feasibility of Stem Cell Therapy for AIDS Lymphoma Using Stem Cells Treated with a Lentivirus Vector Encoding Multiple Anti-HIV RNAs – John J. Rossi, PhD
• Preclinical Studies in Gene Delivery Biodistribution & Host Response Examples from AAV – Barrie J. Carter, PhD & Katherine A. High, MD

May 18, 2010

Session III: Clinical Trial Design, Approval Process and Trial Conduct
Moderator: Jacqueline Corrigan-Curay, MD, JD
Speakers:

• General Considerations for Clinical Trial Design - Steven Winitsky MD
• Special Considerations in Gene Transfer: Small Populations – Orphan Diseases - Zi-Fan Yu, ScD
• Special Considerations in Gene Transfer: Surrogate Endpoints - Michael Kalos, PhD
• Updates to the NIH Guidelines for Research Involving Recombinant DNA Molecules (NIH Guideleines) - Jacqueline Corrigan-Curay, MD, JD
• Federal and Institutional Approval Process (IRB, IBC, NIH/RAC, FDA) - John Zaia, MD
• Good Clinical Practices (GCP) Overview - Martha Reitman, MD (Materials Unavailable)

Session IV: Clinical Trial Compliance, Monitoring and Oversight
Moderator: John Rossi, PhD
Speakers:

• Adverse Event Reporting in IND Studies - Helen Heslop, MD
• Data Safety Monitoring: Why Is It Done? How Do You Do It? - Bambi Grilley, RPh
• Site Monitoring Visits: What To Expect and How To Prepare - Colleen Allen
• Preparing for an FDA Inspection - Patricia A. Holobaugh

Session V: Bioethics, Research Integrity and Conflicts of Interest
Moderator: Traci Mondoro, PhD
Speakers:

• Bioethics in Clinical Research - Robyn S. Shapiro, JD
• Conflicts of Interest in Clinical Studies - Steven Joffe, MD, MPH

Session VI: Special Considerations for Follow Up in Gene and Cell Therapy Clinical Trials
Moderator: Barrie Carter, PhD
Speakers:

• Genotoxicity Considerations in Clinical Trials Utilizing Integrating Vectors - Cynthia Dunbar, MD
• The Letter! - Dear Gene Therapy IND or Somatic Cell Therapy Letter - Adrian Gee, PhD
• Monitoring the Safety of Stem Cell Based Therapeutics in Clinical Trials - Jane Lebkowski, PhD
• Phase 3 Labeling Issues - Wilson W. Bryan, MD