NEW YORK – Encoded Therapeutics on Monday said that regulators in the UK have granted it permission to begin a clinical trial of its gene therapy ETX101 for SCN1A-positive Dravet syndrome.
The UK Medicines and Healthcare Products Regulatory Agency's approval of the company's clinical trial authorization application allows for the start of the Phase I/II dose-escalation EXPEDITION study involving children with Dravet syndrome, a developmental disorder characterized by treatment-resistant seizures and neurodevelopmental stagnation and often caused by loss-of-function variants in the SCN1A gene.
South San Francisco, California-based Encoded will launch EXPEDITION in mid-2024 and focus on enrolling infants and young children with Dravet who are between 6 months and 4 years of age. This clinical trial will run concurrently with two other Phase I/II dose-escalation trials in the US and Australia in which Encoded is studying the gene therapy in other age groups.
In the UK trial, four patients will receive up to two doses of ETX101. The gene therapy uses an adeno-associated virus serotype 9 vector to deliver a transgene encoding an engineered transcription factor under the control of a cell-selective regulatory element and is designed to increase the expression of the endogenous SCN1A gene.