Translational Science Training Course: Best Practices and Lessons Learned
Tuesday, May 15

Introduction
8:30 AM - 8:45 AM
Room: 201 AB

Speakers

Larry A. Couture, PhD

Course Part I
8:45 AM - 11:30 AM
Room: 201 AB
Clinical Trials, Regulatory Affairs and Biostatistics

Running clinical trials is a multidisciplinary endeavor, so how are clinical trials designed and justified? Although in gene and cell therapy, the initial feasibility is usually suggested by preclinical efficacy experiments, translating this into meaningful and feasible clinical trials is never straightforward. The design of the trial will drive the other support activities. This session explores how this process works in practice, with some emphasis on areas, Regulatory and Biostatistics, that tend to be unfamiliar to new trial practitioners in translational medicine.

Moderator
Douglas J. Jolly, PhD

Speakers

Shelly Heimfeld, PhD
Challenging Issues in Clinical Trial Development within an Academic Setting

Andra E. Miller, PhD
Regulatory Strategies for Cell and Gene Therapies

John Scott, PhD
Biostatistical Considerations in Translational Science

Panel
Shelly Heimfeld, PhD
Andra E. Miller, PhD
John Scott, PhD
John A. Zaia, MD

Lunch
11:30 AM - 12:30 PM

Course Part II
12:30 PM - 3:00 PM
Room: 201 AB
Manufacturing, Preclinical Studies and Assays

This session will cover topics relating to the manufacture of material for clinical trials and pre-clinical animal studies, considerations for the design and conduct of pre-clinical animal studies and considerations in choosing methodologies and the development of assays suitable for in-process testing and release of manufactured cell and gene therapy products. Topics to be covered through the presentations or the panel discussion will include: GMP requirements for early phase manufacturing, process development objective, raw material sourcing and vendor qualification, animal models, GLP requirements for animal studies and assay qualification considerations.

Moderator
Larry A. Couture, PhD

Speakers

Richard O. Snyder, PhD
cGMP Manufacturing of Viral Vectors

Ying Huang, PhD
Preclinical Considerations in Cell and Gene Therapy Product Development - CBER Perspective

Kenneth Cornetta, MD
Production is Half the Battle: Getting Gene Therapy Products Certified for Clinical Trials

Panel
Richard O. Snyder, PhD
Ying Huang, PhD
Kenneth Cornetta, MD
Joy Cavagnaro, PhD, DABT, ATS

Course Part III
3:00 PM - 5:00 PM
Room: 201 AB
Case Studies (Clinical Trials)

This session will hear from investigators in the field who have successfully surmounted problems related to study design and regulatory concerns. This will cover issues relating to cell-based therapy, to Hemophilia B and the audience will be provided an opportunity to ask specific questions related to their projects.

Moderator
John A. Zaia, MD

Speakers

Andrew M. Davidoff, MD
AAV-mediated FIX Gene Transfer for Hemophilia B: Bench to Bedside

Edmund V. Mickunas
Human Embryonic Stem Cell Derived Retinal Pigment Epithelium for Treatment of Macular Degeneration-Regulatory and Clinical Considerations

Carl H. June, MD
Development of Chimeric Receptors for Leukemia