Paths to autism treatment: Validating targets for syndromic ASD

Progress in unraveling the genetics of the autism spectrum, along with baby steps toward accessible genetic testing, are pointing a path for drug developers in a disease whose diverse etiology has made it hard to crack.

The pipeline of genetically based compounds is still sparse given the size of the unmet need, with eight companies pursuing seven targets. Still, while some of the targets — such as SCN2A and SHANK3 have been around for a while, genetic and small molecule therapies are emerging for new targets like GRIN2B and SYNGAP1 and likely represent the beginning of a new wave arising from large scale genomics efforts. ...