Great Ormond Street Hospital leads gene therapy for children

Great Ormond Street Hospital is keeping pace with the fast‑moving field of gene therapy for children, a niche that has moved from experimental to routine in just a few decades.

Quarter‑century of pioneering work

In 2024 the institution marked 25 years since it first helped shape the modern setting of genetic treatments. The early work focused on viral vectors, a delivery method that has become a backbone for many later successes. Since the first patient received the approach in 2001, the centre has moved from lab benches to bedside at a scale few other children’s hospitals can match.

Clinical milestones and patient impact

More than 90 experimental programmes have passed through its dedicated Cell and Gene Therapy Facility, with 35 of those reaching clinical trial status. To date, over 135 young patients have benefited from the modality, while nearly 100 others have been treated with advanced cell‑based interventions.

Recognition came in 2026 when a leading clinician and a patient were listed among TIME’s TIME100 Health honorees. The patient, a child with an otherwise untreatable form of T‑cell leukaemia, became the first to receive a base‑editing therapy that rewrites DNA inside immune cells. She has remained in remission for more than three years, a result that shows the practical impact of the treatment.

During the 2025/26 reporting period the centre reported 67 phase 1 and 2 trials of experimental medicines and nine world‑first in‑human or in‑child applications, a volume that suggests the pipeline is far from drying up.

Outcomes have begun to show up in everyday life: many recipients are now back in school, some live independently, and developmental milestones that once seemed out of reach are being met. The institution also played a key role in the development of a therapy for spinal muscular atrophy, where early intervention dramatically improves long‑term prognosis.

The partnership with University College London, highlighted by the Zayed Centre for Research into Rare Disease in Children, blends clinicians, scientists and families in a single workspace. This model speeds the shift from discovery to bedside, expanding the menu of options for children with the most complex conditions.

Looking ahead, the centre’s track record suggests it will keep shaping the field, but the path is not without hurdles. Scaling up manufacturing, handling regulatory settings and ensuring equitable access will test its capacity to turn breakthroughs into standard care. If the current momentum holds, the next wave of precision medicines could reach patients faster than ever before.

Beyond research, the institution collaborates with health providers worldwide, including several in the Middle East. Visiting‑consultant programmes, fellowships, education initiatives and joint clinical projects aim to build local expertise while preserving a route to the specialist care available in London.

Annually, the hospital handles roughly 260 000 visits across 67 specialties and sub‑specialties, with more than 6 000 of those coming through its International and Private Care service. Such volume reflects both its reputation and the demand for highly specialised paediatric care.

A multidisciplinary team approach ensures that children with rare and complex conditions receive coordinated expertise.

Families receive dedicated support from referral through discharge, a seamless service that aims to reduce the stress of handling rare‑disease care. The institution’s blend of innovative research, rigorous outcome measurement and a patient‑first ethos continues to set benchmarks that other centres look to emulate.