First p47 CGD Gene Therapy Patient Regains Normal Life at 20
Updated
Updated · Wellcome Trust · Jul 20
First p47 CGD Gene Therapy Patient Regains Normal Life at 20
1 articles · Updated · Wellcome Trust · Jul 20
Summary
Remi Pereszczak, 20, says a 2024 gene therapy trial at Great Ormond Street Hospital transformed life with p47 chronic granulomatous disease, letting him finish A-levels, start university and live more normally.
The treatment corrected his blood stem cells outside the body using a modified virus, then returned them to rebuild an immune system weakened by the rare, life-threatening disorder.
GOSH said Remi was the first patient in the world to receive this particular therapy for p47 CGD, a condition that had previously left him with frequent hospital visits, daily pain and tight limits on ordinary childhood activities.
The advance builds on decades of UCL and GOSH research into inherited immune disorders, but wider use still faces major barriers including specialist delivery needs and treatment costs that can run into millions.
Remi's cells were edited outside his body. How close are we to safely fixing genes directly inside a patient?
What is the biggest hurdle preventing multi-million dollar gene therapies from becoming accessible to everyone who needs a cure?
July 2026: The First Successful Gene Therapy for p47 Chronic Granulomatous Disease—A New Era for Rare Genetic Immunodeficiencies
Overview
In July 2026, the world-first successful gene therapy for p47 Chronic Granulomatous Disease (CGD) was announced, marking a monumental achievement in medical science. This breakthrough offered a new lease on life and dramatically transformed the future for patients with this rare genetic immunodeficiency. Remi Pereszczak, the first patient to receive the therapy, exemplified its profound human impact. Before treatment, Remi faced severe infections and chronic inflammation that limited his childhood and adolescence. The success of this therapy not only changed Remi's life but also set a new standard for treating rare genetic diseases.