A Focus on Gene and Cell Therapy for Clinicians

All times below listed in ET

Planned by the Clinician Education Working Group. Held in collaboration with the National Organization for Rare Disorders (NORD).

8:00 – 8:20 a.m.

A Focus on Gene and Cell Therapy for Clinicians - Introduction: What Therapies Are Available Today?
Terence R. Flotte, MD, UMass Chan Medical School

8:20 – 8:45 a.m.

Types of Therapies – Lentiviral/In-vivo HSC
Matthew Porteus, MD, PhD, Stanford University

8:45 – 9:10 a.m.

Types of Therapies – AAV
Jerry R. Mendell, MD, PhD, The Research Institute at Nationwide Children's Hospital

9:10 – 9:35 a.m.

Types of Therapies – CAR-T Therapies
David L. Porter, MD, Hospital of the University of Pennsylvania

9:35 – 10:00 a.m.

Clinical Trials and My Role - How Do I Find a Clinical Trial?
Florian Eichler, MD, Massachusetts General Hospital

10:00 – 10:25 a.m.

Clinical Trials and the Role of FDA - Identifying Definitive Clinical Outcomes
Peter Marks, FDA

10:25 – 11:05 a.m.

Running a Clinical Trial
Kathryn Wagner, MD, PhD

11:05 – 11:30 a.m.

Ethics of Recommending a Clinical Trial
Alison Bateman-House, PhD, MPH

11:30 – 11:55 a.m.

Hospital Pharmacy Role in Managing Insurance Issues
Michael Storey, PharmD, Nationwide Children’s Hospital