SynaptixBio, which is developing a therapy for H-ABC (Hypomyelination with atrophy of the basal ganglia and cerebellum) leukodystrophy, says it has identified the drug it will take forward into clinical trials.
Dan Williams PhD, CEO at SynaptixBio, said: “This is a hugely significant milestone. We are confident our candidate drug will deliver the same positive impacts that ASOs have made on other degenerative diseases such as Duchenne muscular dystrophy.
To find out more: SynaptixBio announcement
