Afraxis: Backing PAK1

Most treatments for Fragile X syndrome only alleviate the symptoms, such as anxiolytics for anxiety, but Afraxis Inc. is attempting to modify the disease by attacking the underlying biology of synaptic dysfunction. The company is developing PAK1 inhibitors to correct abnormalities in dendritic spines on neurons, which have been associated with a range of CNS disorders including Fragile X, autism spectrum disorders, schizophrenia, Alzheimer's disease, Huntington's disease and Parkinson's disease.

"PAK1 could become the first disease-modifying target in all of these disorders, and it could become a pan-CNS target," said Jay Lichter, president and CEO of Afraxis and managing member at its sole investor, Avalon Ventures...