The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has cleared Ensoma’s clinical trial authorisation (CTA) application for its in vivo haematopoietic stem cell (HSC)-directed gene insertion therapy, EN-374, for X-linked chronic granulomatous disease (X-CGD), a genetic disorder.
Ensoma recently announced the dosing of the first US patient in the multinational Phase I/II trial of EN-374.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
The authorisation will enable the company to initiate the Phase I/II trial at sites in the UK.
The multicentre, open-label Phase I/II trial is aimed at assessing the tolerability, pharmacodynamics, safety, and efficacy biomarkers of the therapy.
Its primary goal is to identify a dose for further clinical development in this disorder.
Incidence of treatment-related, treatment-emergent and serious adverse events are the key safety endpoints of the trial.
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalDataEfficacy biomarkers focus on changes in functional dihydrorhodamine positive (DHR+) neutrophils and the proportion of subjects reaching predefined DHR+ neutrophil thresholds, ranging from 10% to 50%.
The dose-escalation portion of the study will enrol adult subjects with X-CGD.
Upon completion of the adult cohorts, paediatric patients are expected to be enrolled in a dose-expansion cohort.
Ensoma CEO Jim Burns said: “This is a defining moment for Ensoma. With regulatory clearance to initiate our Phase I/II trial in the UK so quickly following successful dosing of the first patient at a US site, we are well positioned to generate meaningful clinical data for our first-in-class in vivo HSC-directed therapy.
“These milestones underscore the potential of our technology and commitment of our team to deliver breakthrough genetic medicines to patients.”
Earlier this year, the company secured orphan drug and rare paediatric disease designations for EN-374 from the US Food and Drug Administration (FDA).
EN-374 is described as a first-in-class in vivo HSC-directed therapy for X-CGD. It uses virus-like particles (VLPs) for delivering payloads that have a cytochrome b-245 beta chain (CYBB) transgene to HSCs.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
