Note: Following are links to the presentation slides from faculty members who gave ASGT authorization to post them on this website. Certain faculty members were unable to authorize the posting of their slides due to publication issues.

Program Co-Chairs: Katherine A. High, MD (The Children's Hospital of Philadelphia) and Daniel R. Salomon, MD (The Scripps Research Institute)

Dates: April 7-8, 2005
Location: Marriott Crystal Gateway, Arlington , VA

April 7, 2005

Welcome/Overview of Meeting
Katherine A. High, MD

Session I: Vectorology
Co-Chairs: Arthur Nienhuis, MD and Barrie Carter, PhD

Speakers:

• David Williams, MD – Stem Cell Therapy in Fanconi Anemia (Materials Unavailable)
• Cynthia Dunbar, MD – Pathophysiology of adverse events of the X-linked SCID trail. Modeling insertational mutagenesis in non-human primates (Materials Unavailable)
• Philip Gregory, PhD – Gene Correction Work as Alternative Therapeutic Modality

Session I: Vectorology Continues

• Terence R. Flotte, MD – AAV
• Arthur Beaudet, MD – Adenovirus
• Joseph Glorioso, PhD– Herpesvirus

Session II: Immunology of Gene Therapy
Co-Chairs: Savio L.C. Woo, PhD and Michel Sadelain, MD, PhD

Speakers:

• Daniel Salomon, PhD – Innate Immunity and the Paradox of Using Viral Pathogen-Based Vectors
• Hildegund Ertl, MD – Adaptive Immunity to Payload Gene Products (Materials Unavailable)
• Philip Johnson, MD – HIV Vaccine Trial

Session III: Vaccines – Cancer and Other Diseases
Co-Chairs: Hildegund Ertl, MD and Dale Ando, MD

Speakers:

• Malcolm Brenner, MD, PhD – Retinoblastoma or Prostate or Both
• Karin Jooss, PhD – GVAX Experience (Materials Unavailable)
• John Nemunaitis, MD – Trials with Adenovirus as a Vaccine
• Stephen Russell, MD, PhD – Oncolytic RNA Viruses (Materials Unavailable)

Session IV: Real Stories
Co-Chairs: Samuel C. Wadsworth, PhD and Glenn F. Pierce, MD, PhD

• Jean Bennett, MD, PhD: Pre-clinical studies for Lebers congenital amaurosis. Timeline from compelling pre-clinical studies to initiation of clinical trials
• Katherine A. High, MD: Hemophilia (Materials Unavailable)
• Mark H. Tuszynski, MD, PhD: Neural cell therapy

Session V: Real Stories – The Role of Disease Foundations in Moving Gene Transfer Forward
Co-Chairs: Alan Kinniburgh, PhD and Sharon Hesterlee, PhD

• Rachel Salzman, DVM - The Stop ALD Foundation
• Mark Skinner – World Federation of Hemophilia  

April 8, 2005

Session VI: Real Stories
Co-Chairs: Richard C. Mulligan, PhD and Cynthia Dunbar, MD

• Kenneth H. Fischbeck, MD: Muscular Dystrophy
• Carl H. June, MD: HIV and Lentivirus (Materials Unavailable)
• Douglas J. Jolly, PhD: Retroviral Factor VIII
• Ronald G. Crystal, MD - Gene Therapy of Batten's Disease (Materials Unavailable)

Session VII: FDA/NIH Perspective
Co-Chairs: Stephanie Simek, PhD and Kenneth Cornetta, MD

• Daniel Rosenblum, MD – Gene Transfer Studies: Clinical and Preclinical Issues of Regulatory Concern
• Andrew Byrnes, PhD – Common Challenges in the Development of Gene Therapy Products
• Kenneth Cornetta, MD – Challenges to Gene Therapy: The IU NGVL Experience
• Additional Panelists for Discussion:
  Maritza McIntyre, PhD - Product Expert
  Amy Patterson, MD, Office of Biologic Activities, NIH
  Mercedes Serabian, MS, DABT - Pharm/Tox Expert

Session VIII: Final Discussions – Initiating the process of "consensus thinking": What are the directions forward?”
Co-Chairs: Katherine A. High, MD and Daniel Salomon, PhD