Great Ormond Street Hospital for Children (GOSH) is a global leader in pediatric healthcare, research, and innovation. Based in London, it has been at the forefront of gene therapy for 25 years, transforming the lives of children with rare genetic disorders. This year marks a significant milestone, celebrating a quarter-century of pioneering work that has laid the foundation for treatments once thought impossible.
Pioneering Gene Therapy
GOSH has played a key role in advancing gene therapy, particularly in the development of viral vectors for safer and more effective treatments. Since 2001, the hospital has delivered over 90 therapies through its Cell and Gene Therapy Facility, with 35 gene and cell therapies administered in clinical trials. To date, more than 135 children have received gene therapy at GOSH, alongside nearly 100 patients treated with advanced cell therapies. These therapies have addressed conditions such as severe immune deficiencies, degenerative muscle disorders, and inherited blindness, offering hope where conventional treatments had failed.
The hospital’s work has been recognized globally, with GOSH clinician Professor Waseem Qasim and patient Alyssa Tapley named in TIME’s prestigious TIME100 Health list in 2026. Alyssa, who had an otherwise untreatable form of T-cell leukemia, became the first patient to receive a pioneering base editing therapy, which precisely rewrites DNA within immune cells to target and eliminate cancer. She has been in sustained remission for over three years. This breakthrough not only highlights GOSH’s innovative capabilities but also its commitment to delivering highly complex, next-generation therapies safely. In the 2025/26 period alone, GOSH conducted 67 phase 1 and 2 clinical trials and introduced 9 world-first treatments.
Transforming Outcomes for Children
Gene therapy at GOSH is already making a significant impact, enabling children to attend school, live independently, and achieve key developmental milestones. The hospital has contributed to the development of globally significant therapies, including treatment for spinal muscular atrophy, where early intervention is critical. GOSH’s multidisciplinary team approach ensures that children with rare and complex conditions receive coordinated, world-leading expertise. This model 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 pediatric hospital globally.
The hospital’s partnership with University College London has led to the establishment of the Zayed Centre for Research into Rare Disease in Children, which accelerates the translation of discovery into clinical care. This integrated model brings together clinicians, scientists, academics, and patients to drive innovation in genomics, cell therapy, and precision medicine. By supporting collaboration across disciplines, the Zayed Centre is rapidly expanding treatment options for children with the most complex conditions.
A Trusted Global Partner
GOSH works with healthcare providers worldwide, including across the Middle East, to strengthen pediatric services and improve outcomes. Through visiting consultant programs, fellowships, education programs, and clinical collaborations, the hospital supports capability-building while maintaining access to highly specialized care in London. Each year, GOSH receives around 260,000 visits from patients, including more than 6,000 visits to its dedicated International and Private Care service. The hospital’s international teams provide personalized care, offering seamless support to families from referral through to discharge, ensuring that every child receives the best possible treatment tailored to their needs.
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