Agenda Details

Learn more about sessions and presentations at the 26th Annual Meeting

Detailed Program

How to Become a Site for AAV Gene Therapy Trials

This workshop is designed to outline the steps needed to become a site for AAV gene therapy trials and discuss what is needed to treat and monitor patients before, during and after therapy treatments.

Session Details

Co-Chair

Charles P.. Venditti, PhD, MD. National Human Genome Research Institute, National Institutes of Heal...

Co-Chair

Nicolas J. Abreu, MD. NYU Langone, New York, NY

Preclinical/IND-enabling Basics of AAV Biology and GT Vectors Preclinical Enablement and the IB

Mark A.. Kay, MD, PhD. Stanford University School of Medicine, Stanford, CA

Regulatory & Site Initiation IBC and IRB Site Activation and Pharmacy

Eric Crombez, MD. Ultragenyx, Cambridge, MA

Clinical Protocol Implementation: Who sees the Patient and What is Needed?Clinical Infrastructure Treating the Patient/Acute Monitoring Phase I/II vs III vs IV

Kimberly Goodspeed, MD. University of Texas Southwestern Medical Center, TX

Follow up and Long-term Toxicity: What needs to be followed for follow up?

Denise E.. Sabatino, PhD. The Children's Hospital of Philadelphia, Philadelphia, PA

Panelist

Eric Crombez, MD. Ultragenyx, Cambridge, MA

Panelist

Denise E.. Sabatino, PhD. The Children's Hospital of Philadelphia, Philadelphia, PA

Panelist

Mark A.. Kay, MD, PhD. Stanford University School of Medicine, Stanford, CA

Panelist

Kimberly Goodspeed, MD, MSCS. University of Texas Southwestern Medical Center, Dallas, TX

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