UPDATE after this event: The videocast recordings of “The Growing Promise of Gene Therapy Approaches to Rare Diseases” workshop, held August 20–21, 2018 at NIH, are now available from the NIH Videocast Archives. To find the two separate recordings, please notice that below that web page’s title “Most Recent VideoCasts” is a small calendar-icon located to the right of the array of page numbers. Click on that calendar-icon and select either August 20 or 21, 2018. The 2-day event’s videocasts are listed under their respective calendar dates.
On August 20-21, 2018 in Bethesda, Maryland, the National Center for Advancing Translational Sciences (NCATS) and the Food and Drug Administration’s Center for Biologics Evaluation and Research co-sponsored a workshop entitled “The Growing Promise of Gene Therapy Approaches to Rare Diseases.” The workshop reviewed the state of current gene therapy approaches; identified challenges and strategies to overcome those challenges; and discussed how to collaboratively scale and accelerate gene therapy development to benefit patients with rare diseases for which there are yet no effective treatments. Lysosomal Disease Network’s Principal Investigator, Dr. Chester B. Whitley, was among the speakers on a panel, and spoke about gene therapy for lysosomal diseases. This joint meeting facilitated discussion among stakeholders including NIH and FDA staff, academics, researchers, biotech- and pharma-industry, and patient group representatives, on overcoming bottlenecks in the development of gene-based therapies. The workshop convened at National Institutes of Health (Main Campus), NIH Clinical Center, in Bethesda, Maryland. There was no admission fee to attend.