A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

May 9, 2024

Primary Completion Date

September 30, 2030

Study Completion Date

September 30, 2030

Conditions
Dravet Syndrome
Interventions
DRUG

ETX101

ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)

Trial Locations (3)

G51 4TF

RECRUITING

Queen Elizabeth Hospital, Glasgow

WC1N3JH

RECRUITING

Great Ormond Street Hospital, London

S10 2TH

RECRUITING

Sheffield Children's Hospital, Sheffield

Sponsors
All Listed Sponsors
lead

Encoded Therapeutics

INDUSTRY