Neuren Pharmaceuticals Adds SYNGAP1 Gene-Related Disorder to Neurodevelopmental Disorders Pipeline for Drug Candidate

MT Newswires Live

08/08

Neuren Pharmaceuticals (ASX:NEU) added a SYNGAP1-related disorder into its neurodevelopmental disorders pipeline for its NNZ-2591 drug candidate, after treatment with the candidate reversed the

neuronal dysfunction caused by SYNGAP1 haploinsufficiency in an in vitro model of the disorder in human iPSC-derived neurons, according to a Friday Australian bourse filing.

The disorder is caused by a variant on the SYNGAP1 gene located on chromosome 6, responsible for producing the SYNGAP1 protein, which acts as a regulator in the synapses.

The firm is also preparing to begin a phase three clinical trial in Phelan-McDermid syndrome.

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