ASGCT NIH Gene Therapy Symposium Program Information

(Program subject to change)

Day 1: September 26, 2011
Time	Topic	Speaker
8:30 am - 8:40 am	Introduction to the Symposium and Keynote	R. Jude Samulski, PhD
8:45 am - 9:15 am	Keynote Address Gene Transfer Enters the Mouth	Lawrence A. Tabak, DDS, PhD
Summary of Positive Results and Successes
9:20 am - 10:20 am	Hematopoietic Stem Cells
	ADA-SCID and Wiskott-Aldrich	Donald B. Kohn, MD
	Hemoglobinopathies	Michel Sadelain, MD, PhD
	Adrenoleukodystrophy (ALD)	Nathalie Cartier-Lacave, MD
10:20 am - 10:25 am	Moderator Discussion and Review - Donald B. Kohn, MD
10:25 am - 10:45 am	Coffee Break
10:45 am - 11:05 am	Leber Congenital Amaurosis (LCA)	William Hauswirth, PhD
11:05 am - 11:25 am	Hemophilia	Ulrike M. Reiss, MD
11:25 am - 11:45 pm	Cardio/Pulmonary	Barry J. Byrne, MD, PhD
11:45 am - 11:50 am	Moderator Discussion and Review - Barry J. Byrne, MD, PhD
11:50 am - 12:50 pm	Lunch (Not Provided)
12:50 pm - 1:10 pm	Cancer	Malcolm K. Brenner, MD, PhD
1:10 pm - 1:30 pm	Neurological	Beverly L. Davidson, PhD
1:30 pm - 1:35 pm	Moderator Discussion and Review - Beverly L. Davidson, PhD
Summary of Morning Session and Review of Accomplishments and Future Challenges
1:35 pm - 2:05 pm	Summary of Morning Session and Review of Accomplishments and Future Challenges	Donald B. Kohn, MD, Barry J. Byrne, MD, PhD, and Beverly L. Davidson, PhD
2:05 pm - 2:45 pm	Comparison of Clinical Trials Outside of the United States: What has made the clinical trials successful? What are the funding and regulatory issues in both regions?	David Williams, MD Natalie Cartier, MD Maria-Grazia Roncarolo, MD, PhD Wuh-Liang Hwu, MD, PhD
2:45 pm - 2:55 pm	Discussion Moderated by Donald B. Kohn, MD and Barry J. Byrne, MD, PhD
2:55 pm - 3:15 pm	Coffee Break
Breakout Discussion Sessions
	Discussion Topic	Discussion Lead
3:15 pm - 4:45 pm	1. Immunodeficiency 2. Hemoglobinopathies 3. Cancer 4. Coagulation Disorders 5. Neurologic 6. Cardiovascular 7. Infectious Disease 8. Pulmonary 9. Eye 10. Metabolic	Donald B. Kohn, MD Michel Sadelain, MD, PhD Malcolm K. Brenner, MD, PhD Katherine A. High, MD Beverly L. Davidson, PhD Barry J. Byrne, MD, PhD Boro Dropulic, PhD Terence R. Flotte, MD William Hauswirth, PhD Nathalie Cartier-Lacave, MD
Day 2 of Program: September 27, 2011
8:30 am - 10:00 am	Review of the Results of the Breakout Session Barrie J. Carter, PhD & Malcolm K. Brenner, MD, PhD
10:00 am - 10:20 am	Coffee Break
How to move gene therapy into the clinic?
10:20 am - 10:50 am	How to Make Funding More Efficient: "The California Institute for Regenerative Medicine (CIRM): Model for Supporting Clinical Translation of Stem Cell Therapies"	Ellen G. Feigal, MD
10:50 am - 11:20 am	NIH, Office of Rare Diseases Research	Stephen Groft, PharmD
Regulatory hurdles and resources available to investigators to help address those hurdles
11:20 am - 11:50 am	Overview of NIH Resources Available to Investigators	Traci Mondoro, PhD (PACT) Sonia Skarlatos, PhD (GTRP)
11:50 am - 12:00 pm	Scale of Vector Production Issues	Robert M. Kotin, PhD
12:00 pm - 12:20 pm	Regulatory Realities Versus Resources	Andra Miller, PhD
12:20 pm - 12:50 pm	Needed Resources to Move the Field Forward	Barrie J. Carter, PhD