The point I was trying to make, which was perhaps not clear, is that there is no single autism gene as such. Rather, there are multiple genes (roughly 250) strongly linked to autism, according to recent research. Further, mutations in any single gene are responsible for less than 1% of non-syndromic cases of ASD.
Therefore, although ASD-related syndromes with a single gene mutation, such as Rett, Fragile X, Phelan McDermid, Angelman or Prader-Willi, could be addressed by a targeted antisense therapy, I would think that the broad spectrum disorder of ASD could not.
Stoke Therapeutics, is already working within this area. Its lead compound is being developed in the monogenic neurodevelopmental disorder of Dravet syndrome and it entered a licence agreement earlier this year with Neuren’s trofinetide partner, Acadia Therapeutics, to develop antisense therapies for Rett syndrome, SYNGAP1 syndrome and a further undisclosed monogenic neurodevelopmental disorder.
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