Great Ormond Street Hospital: Continuing to lead the future of gene therapy for children

Transforming possibility in paediatric medicine

For families facing rare genetic disorders, and for clinicians seeking options where conventional treatments have reached their limits, gene therapy is redefining what is possible in paediatric healthcare. At the forefront of this transformation is Great Ormond Street Hospital for Children (GOSH), a globally recognised leader in paediatric healthcare, research and innovation based in London, United Kingdom.

25 years of pioneering innovation
This year marks 25 years since GOSH helped pioneer gene therapy, laying the foundations for treatments that are now changing, and saving, young lives. Work­ing with international partners, GOSH has led advances in delivery systems, par­ticularly viral vectors, enabling safer and more effective treatment of conditions once considered incurable, including se­vere immune deficiencies, degenerative muscle disorders and inherited blindness.

Since delivering its first gene therapy in 2001, GOSH has translated scientific dis­covery into clinical practice at scale. More than 90 therapies have been developed through its Cell and Gene Therapy Facil­ity, with 35 gene and cell therapies deliv­ered through clinical trials. To date, over 135 children have received gene therapy at GOSH, alongside nearly 100 patients treated with advanced cell therapies.

Global recognition for breakthrough science
In 2026, this leadership was further recog­nised when GOSH clinician Professor Was­eem Qasim and patient Alyssa Tapley were named in TIME’s prestigious TIME100 Health list. Their inclusion reflects a world-first breakthrough in base editing technol­ogy, developed and delivered at GOSH.

Alyssa, who had an otherwise untreatable form of T-cell leukaemia, became the first patient to receive this pioneering therapy, in which DNA within immune cells is precise­ly rewritten to target and eliminate cancer. Now in sustained remission for over three years, her story demonstrates both the clini­cal impact of this innovation and GOSH’s capability to safely deliver highly complex, next-generation therapies. In 2025/26 this included 67 phase 1 and 2 clinical trials of experimental medicines, and 9 world-first, in man or child treatments.

Delivering measurable outcomes for children
Gene therapy at GOSH is already trans­forming outcomes, enabling children to attend school, live independently and achieve key developmental milestones. The hospital has also contributed to the development of globally significant thera­pies, including treatment for spinal mus­cular atrophy, where early intervention is critical to improving long-term outcomes.

Driving the next generation of therapies
Innovation continues at pace through GOSH’s partnership with University College London, most visible in the Zayed Centre for Research into Rare Disease in Children. This integrated model, bringing clinicians, scien­tists, academics and patients together, accel­erates the translation of discovery into clini­cal care. Advances in genomics, cell therapy and precision medicine are rapidly expanding treatment options for children with the most complex conditions.

A trusted global partner
GOSH works in partnership with health­care providers worldwide, including across the Middle East, to strengthen paediat­ric services and improve outcomes closer to home. Through visiting consultant programmes, fellowships to London, education programmes and clinical col­laborations, the hospital supports capabil­ity-building while maintaining access to highly specialised care in London.

Great Ormond Street Hospital is one of the world’s leading specialist children’s hos­pitals, delivering care across 67 specialties and sub-specialties. Each year, it receives around 260,000 visits from patients, includ­ing more than 6,000 visits to its dedicated International and Private Care service.

At the core of GOSH’s model is its mul­tidisciplinary team approach, ensuring that children with rare and complex conditions benefit from coordinated, world-leading expertise. This supports robust clinical de­cision-making and consistently strong out­comes, underpinned by a commitment to transparency. GOSH publishes more patient-reported outcome measures (PROMs) data than any other paediatric hospital globally.

Care is personalised to each child, with dedicated international teams providing families with seamless support from refer­ral through to discharge.

With a proven track record of innova­tion, rigorous outcomes measurement and a commitment to excellence, GOSH de­livers world-leading care for the most com­plex paediatric conditions, consistently achieving results that set international benchmarks.

For more information or to refer a patient:
Email: GulfOffice@gosh.nhs.uk
Phone: +971 4 3624722
Website: www.gosh.ae