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Studies in rats suggest oxytocin could help treat the autism-like symptoms of Phelan-McDermid syndrome (PMDS), which is caused by SHANK3 mutations. In a SHANK3-knockout rat model of Phelan-McDermid syndrome, intraventricular injection of oxytocin increased social memory and decreased attention and synaptic plasticity deficits compared with vehicle. Next steps could include testing oxytocin at various stages of development in the PMDS models.

OptiNose Inc. has intranasal oxytocin (OPN-300), delivered using the OptiNose bidirectional nasal delivery device, in Phase II testing for autism...