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 neurogenic therapeutic candidate for depression and post-traumatic stress disorder, NNI-351, inhibits the same Dyrk1a protein that is overproduced in Down syndrome. This inhibition of the protein appears key to how NNI-351 increases the production of new neurons in the hippocampus, reduces the overall hyperactivity and increases the learning and memory ability in Down syndrome mice. Of great importance, is that these beneficial effects of NNI-351 are disease-modifying and do not just mask the symptoms in this well established mouse model of Down syndrome. As stated elsewhere, a patent for NNI-351 has now been issued to Neuronascent by the United States patent office.

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