An in vivo therapy being developed to treat X-linked chronic granulomatous disease (X-CGD) has shown positive early results. 

EN-374, developed by cellular engineering company Ensoma, has demonstrated tolerability in the first participant being evaluated in the Phase I/II trial. 

The therapy is a first-in-class in vivo haematopoietic stem cell (HSC)-directed therapy for X-CGD that employs virus-like particles (VLPs) to deliver payloads having a CYBB transgene to HSCs.  

Neutrophils arising from the engineered HSC then express the protein product of the CYBB transgene. As such, EN-374 is designed to restore function of the infection-fighting NADPH oxidase enzyme complex critical for immune defence in humans. 

Data on EN-374 was presented by Ensoma at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, taking place 11-15 May in Boston, US. 

“We are excited to discuss encouraging initial safety data from the first participant in our Phase I/II clinical trial of EN-374 for X-CGD, the first-ever in vivo HSC gene insertion therapy in the clinic,” said Jim Burns, CEO of Ensoma. 

“While these are early data from a single participant, they mark an important first step in evaluating a new approach to engineering haematopoietic stem cells directly in vivo, and we look forward to continuing to assess safety and potential markers of clinical activity as the study progresses.”