Today's Edition
LONDON (WAM)
Great Ormond Street Hospital (GOSH) celebrated 25 years of pioneering gene therapy during an anniversary event hosted at the Zayed Centre for Research into Rare Disease in Children in London.
The event brought together a UAE delegation, alongside leading clinicians, researchers, and partners from around the world, to reflect on the life-changing impact of gene therapy, and reaffirm a shared commitment to advancing the field.
GOSH has been at the forefront of developing and delivering gene therapies, moving the field from the laboratory into life-changing treatments for children and young people. The therapies work by altering the genes inside a patient’s cells to treat disease, offering hope to children with conditions once thought untreatable.
At GOSH, this has included treating immunodeficiencies, leukaemia, spinal muscular atrophy, and rare genetic conditions.
To date, more than 135 children with rare and complex conditions have benefited from these pioneering treatments, with 35 different gene and cell therapy treatments delivered to babies, children, and young people. Today, the hospital is recognised internationally as a centre of excellence, delivering treatments that offer hope to patients and families worldwide, including across the Gulf region.
Among those attending the event was Rhys Evans, the UK's first gene therapy patient and one of the earliest beneficiaries of the groundbreaking treatment. Born with Severe Combined Immunodeficiency (SCID), a rare and life-threatening disorder that left him without a functioning immune system, Rhys became known as the UK's "bubble boy" before undergoing pioneering gene therapy at Great Ormond Street Hospital in 2001.
Now 25 years old, his attendance at the anniversary celebration provided a powerful reminder of the transformative impact of gene therapy, demonstrating how a once-experimental treatment has evolved into a life-changing reality for children and families around the world.
Prof Maha Barakat, Assistant Minister of Medical Affairs and Life Sciences at the UAE Ministry of Foreign Affairs stated: “This anniversary is a significant celebration of progress in medical sciences and of the dedicated teams whose work is turning discovery into hope for children and families. The Zayed Centre for Research reflects the strength of international collaboration, and we are proud to continue supporting initiatives that advance life-changing research and care.”
Barakat emphasised the longstanding relationship between the UAE and GOSH, through the generous donation of Her Highness Sheikha Fatima bint Mubarak Mother of the Nation, Chairwoman of the GWU, President of the Supreme Council for Motherhood and Childhood, and Supreme Chairwoman of the Family Development Foundation to establish the Zayed Centre for Research in 2014.
Since its opening in 2019, the Zayed Centre for Research has managed more than 89,000 patient appointments and delivered world-first breakthroughs in gene therapy, genomics, and paediatric medicine — transforming lives and offering hope to children worldwide.
The Centre is a partnership between GOSH, University College London, and GOSH Charity, dedicated to advancing research and treatment for children with rare and complex diseases.
Dr Kiki Syrad, Director of Research and Innovation at Great Ormond Street Hospital, said: “Marking 25 years of gene therapy at GOSH is both a celebration of how far we’ve come and a powerful reminder of what’s still possible. This progress has only been possible through global collaboration, innovation, and the dedication and hard work of our clinicians and researchers, alongside our patients and families who have helped shape this field.
"We are committed to building on this momentum and accelerating the development of new therapies and ensuring more children, in the UK and around the world, can benefit from gene therapies.”
Significant funding from Great Ormond Street Hospital Charity (GOSH Charity), across the research pipeline as well as for infrastructure and innovation, has provided extensive support for pioneering gene therapies at GOSH, helping to deliver more breakthroughs for seriously ill children.
During the event an esteemed panel, including Professor Manju Kurian, Consultant Paediatric Neurologist, Professor Claire Booth, Mahboubian Professor in Gene Therapy and Consultant Paediatric Immunologist and Dr Barry Flutter, Head of Product Manufacturing, explored the current gene therapy landscape, the scientific and clinical pathways that underpin treatment development, and the opportunities and challenges facing the field as it looks ahead at another 25 years.
