Great Ormond Street Hospital celebrates 25 years of gene therapy at international anniversary event

Great Ormond Street Hospital for Children (GOSH) has marked 25 years since the delivery of its first gene therapy, celebrating a quarter-century of medical innovation at a special anniversary event held in the Zayed Centre for Research into Rare Disease in Children. 

The event brought together international guests, including world-leading clinicians, researchers and partners, alongside representatives from the United Arab Emirates. Together, they reflected on the life-changing impact of gene therapy and reaffirmed a shared global 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.  

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. 

Also in attendance was Rhys Evans, the UK’s first gene therapy patient, who received treatment for Severe Combined Immunodeficiency as a baby. Now aged 25, Rhys represents the extraordinary, life-long impact of these treatments.  

At the heart of this milestone celebration is the Zayed Centre for Research, a world-leading facility, funded by GOSH Charity, and made possible by a landmark £60 million gift in 2014 from Her Highness Sheikha Fatima bint Mubarak, wife of the late Sheikh Zayed bin Sultan Al Nahyan, the founding father of the United Arab Emirates - exemplifying international collaboration in advancing children’s health. 

The UAE delegation was welcomed by Karl Munslow Ong, Chief Executive of GOSH, at the Zayed Centre for Research where he highlighted the pioneering work taking place at the Zayed Centre for Research, which brings together innovative research and clinical care under one roof. This world first centre drives forward new treatments and cures for seriously ill children from across the UK and international patients. 

On this occasion, Her Excellency 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.” 

The visit reinforces the long-standing relationship between GOSH, University College London (UCL), and Her Highness Sheikha Fatima bint Mubarak, whose generous support made the creation of the Zayed Centre for Research possible. Since opening in 2019, the Zayed Centre for Research has hosted 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.     

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.”   

Aoife Regan, GOSH Charity’s Director of Impact & Charitable Programmes, said: “As we mark 25 years since the first gene therapy was administered at the hospital, GOSH Charity is proud to have played such a key role in supporting the research and clinical work that has helped transform the lives of seriously ill children and their families.”  

“Over the past 25 years, GOSH Charity has provided sustained funding for groundbreaking gene therapy work at GOSH, helping to advance treatments for some of the rarest and most complex inherited conditions affecting children.”  

“Gene therapy has the potential to be truly transformative, offering targeted treatments for rare diseases and creating new possibilities for children at GOSH and beyond, helping them have the best chance, and best childhood possible. Supporting this pioneering research remains a key priority for GOSH Charity, so we can ensure that more children can benefit from these advances in the future.”