JAG201

JAG201 is an investigational gene therapy developed to treat neurodevelopmental disorders caused by SHANK3 haploinsufficiency, including Phelan-McDermid syndrome (PMS) and a genetic form of autism spectrum disorder (ASD). The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional SHANK3 minigene under the control of a neuron-specific promoter (human synapsin 1), ensuring expression only in neuronal cells. Administered as a one-time intracerebroventricular injection, JAG201 aims to restore normal SHANK3 protein levels in the central nervous system, thereby durably restoring synaptic function required for learning, memory, cognitive development, and motor skills. Preclinical studies in mice and non-human primates have shown that JAG201 can achieve widespread delivery throughout the brain with significant improvements in neurobehavioral outcomes. The therapy has received FDA Fast Track and Rare Pediatric Disease designations[1][2][3][5][8].