Down syndrome is the most prevalent genetic disorder, occurring in one of every 800 live births. Until recently, a therapeutic for individuals with Down syndrome seemed improbable due to the large number of genes (extra chromosome 21) that could be responsible for the developmental disorder. Studies suggest that a 33-gene region of the chromosome appears critical for the cognitive deficit and that potentially one gene, Dyrk1a, may play a direct role in the developmental cognitive impairment.

Our lead neurogenic agent for depression appears to inhibit the same Dyrk1a protein that is overproduced in Down syndrome and this inhibition may be responsible, at least in part, for the neurogenic activity observed both in vitro and in vivo. Our lead therapeutic candidate for Down syndrome represents an exciting new class of drugs that to-date has shown memory enhancement and neurogenesis in vivo. Our program for Down syndrome is in the proof-of-concept phase of development.

For more information on Down syndrome, please visit the Down Syndrome Research and Treatment Foundation at www.dsrtf.org.