Novel Therapeutic Interventions to Address the Root Genetic Cause of Neurodevelopmental Disorders

Our client, a global leader in therapies for rare genetic diseases, is seeking novel approaches to address the root genetic cause of neurodevelopmental disorders. Specifically, the team is looking for interventions which promote a disease modifying effect in developmental epileptic encephalopathies and neurodevelopmental disorders - CDKL5 deficiency disorder; Fragile X Syndrome; Rett Syndrome, SYNGAP1 disorder and STXBP1 disorder.

Approaches of Interest:

• Disease modifying treatments, for example through allele-specific knockdown, protein replacement/enhancement...