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Workshop: Gene-based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders


January 21, 2021




On January 21, 2021, the National Institute of Mental Health conducted a virtual workshop that provided insights into the potential challenges and opportunities for gene-based therapeutic strategies for individuals with rare genetic neurodevelopmental disorders. This workshop brought together a multi-disciplinary group of experts from academia, government, and industry to provide a review of the current state of science in gene-targeted therapeutics and characteristics of suitable gene/molecular targets and mechanisms amenable to gene-based therapies. This workshop also featured some of the gene-targeted approaches that are currently in the clinic or in development to highlight the scientific rationale for each treatment and the milestones for reaching key stages of therapeutics development, and the challenges and lessons learned along the way.