College of Medicine

KEYNOTE SESSION

• 0:00 - 3:09  Welcome (FSU College of Medicine Dean Alma B. Littles, M.D.)
  
• 3:09  - 9:35 Opening Remarks (State Representative Adam Anderson (R-Palm Harbor))
  
• 9:35  - 20:08 Introductory Remarks (FSU President Richard D. McCullough, Ph.D.)
  
• 20:08  - 1:04:18 KEYNOTE: Lessons Learned from 30 Years of Clinical Trials with AAV vectors (UMass Chan Medical School Executive Deputy Chancellor and Provost Terence R. Flotte, M.D., University of Massachusetts, Worcester)
  
• 1:04:18  - 1:17:40 Discussion (President Richard D. McCullough & Terence R. Flotte)

CHECK PRESENTATION CEREMONY

• 1:17:40  - 1:27:53 Special ceremony featuring Representative Anderson presenting President McCullough with a $5 million check on behalf of State of Florida in support of the IPRD.

SYMPOSIUM 1: RARE DISEASE CENTERS AND PROGRAMS

• 1:27:53  - 1:29:36 Introduction (FSU IPRD Director Pradeep G. Bhide, Ph.D.)
  
• 1:29:36 - 1:50:08 Whole genome sequencing and pharmacogenomics in a clinical setting: Lessons from the Baby Manatee Project (David Seo, M.D., Nicklaus Children's Health System, Miami)
  
• 1:50:08  - 2:15:46 Genetic studies in patients with rare and undiagnosed diseases in Florida (Mustafa Tekin, M.D., University of Miami)
  
• 2:15:46  - 2:40:33 Establishing a rare disease center: The Collaborative Center for X-Linked Dystonia Parkinsonism (Nutan Sharma, M.D., Ph.D., Massachusetts General Hospital and Harvard Medical School, Boston)
  
• 2:40:33  - 2:55:20  Discussion and Closing Remarks (IPRD Director Pradeep G. Bhide)

SYMPOSIUM 2: GENOMICS AND GENE THERAPY

• 2:55:20  - 2:58:12  Introduction (FSU IPRD Associate Director Michelle Arbeitman, Ph.D.)
  
• 2:58:12  - 3:28:23  Gene therapy and molecular medicine in rare disease: Future Vision (Barry Bryne, M.D., Ph.D., University of Florida)
  
• 3:28:23  - 3:52:06 Getting beyond one disease at a time: Platform Approaches to Genetic Therapy Clinical Trials (Philip J. (P.J.) Brookes, Ph.D., National Institutes of Health, Rockville, Maryland)
  
• 3:52:06  - 4:19:18 Genome sequencing and population-based analysis for Mendelian conditions (Greg Barsh, M.D., Ph.D., Stanford University)
  
• 4:19:18  - 4:25:14 Discussion and Closing Remarks (IPRD Associate Director Michelle Arbeitman)

GENEDX INDUSTRY ROUND TABLE: BIOPHARMA AND RARE DISEASES

• 4:25:14  - 4:29:23  Introduction (FSU Vice President for Research Stacey Patterson, Ph.D.
  
• 4:29:23  - 4:42:23  Roundtable Presentation: Paul Kruszka, M.D., MPH, FACMG (GeneDx)
  
• 4:42:23  - 4:54:36 Roundtable Presentation: Jonathan Hickman, PharmD. (Genentech)
  
• 4:54:36  - 5:12:12 Roundtable Presentation: Heather Lau, M.D. (Ultragenyx)
  
• 5:12:12  - 5:32:22 Roundtable Discussion (Paul Kruszka, Jonathan Hickman, & Heather Lau)