Cell and Gene Therapy Clinical Trials and Patients at a New Frontier in Drug Development

June 11, 2021

This panel discussion on Cell and Gene Therapy has something for all stakeholders in clinical research. It provides an introduction to and education about clinical trials in this emerging area of drug development. Moderator Steve Smith is a patient advocate whose son and granddaughter have been diagnosed with unrelated rare disorders. He explains how genetic testing and biotech innovation has advanced since the time his son was diagnosed to benefit his granddaughter who was diagnosed very soon after birth. Dominique Pichard, MD, is the Chief Science officer of the International Rett Syndrome Foundation and mother of a daughter with Rett Syndrome. She addresses the importance of recent innovations, the availability of clinical trials as an option for Rett Syndrome families, and the approach her advocacy group uses to educate patients about clinical research options. Daniel Kavanaugh, PhD, an expert on safety and ethics in clinical research, defines cell and gene therapy, and describes how safety and ethics are addressed by oversight from independent biosafety review committees, independent data monitoring committees, and institutional or independent review boards. Karmen Trzupek, MS, CGC, is the Director, of Clinical Trial Services, and the Director, of Rare Disease Genetics Services for InformedDNA, a clinical genetic counseling practice. She works with clinical trial sponsors, sites, and patients to match patients with the appropriate therapeutic approach and educate all stakeholders about appropriate uses and interpretations of new genetic testing options.

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