ESGCT 2021 | The gene-replacement therapy landscape for CDKL5-deficiency disorder

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, discusses the current therapeutic landscape for CDKL5 deficiency disorder (CDD), a rare neurodevelopmental disorder that affects young children and is caused by loss-of-function mutations in the cyclin-dependent kinase-like 5 (CDKL5) gene. CDD is characterised by seizures and intellectual disability and current therapies are only able to ameliorate symptoms. Dr Schmid describes the rationale behind the investigation of potentially curative gene-replacement therapy approaches in order to restore CDKL5 protein expression and provides an overview of ongoing research in this area. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.