Find out how advocacy groups, researchers and industry use the GENE TARGET framework to determine readiness for gene therapy, identify gaps in the translational research pipeline and prioritize funding and research efforts.
• Moderator: Shruti Mitkus, Ph.D., Director, Patient Services, Global Genes
• Raymond Belanger-Deloge, Translational Genomic Medicine Specialist, Rosamund
Stone Zander Translational Neuroscience Center, Boston Children’s Hospital
• Matt Fuller, Ph.D., Executive Director, Gene Therapy Research, Ultragenyx
• Ashley Point, President, Koolen De-Vries Syndrome Foundation

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