LONDON, 10th November, 2026 (WAM) -- The Zayed Centre for Research into Rare Disease in Children (ZCR) in London is marking its seventh anniversary, highlighting significant advances in paediatric research and innovative treatments for children with rare and complex diseases.
Made possible thanks to a £60 million contribution from H.H. Sheikha Fatima bint Mubarak, the “Mother of the Nation”, Chairwoman of the General Women's Union (GWU), President of the Supreme Council for Motherhood and Childhood (SCMC), and Supreme Chairwoman of the Family Development Foundation (FDF), ZCR began treating young patients in 2019 by bringing together scientists, clinicians and researchers under one roof to accelerate scientific discoveries and develop new approaches to diagnosing and treating childhood diseases.
Dr. Maha Barakat, Assistant Minister of Foreign Affairs for Medical Affairs and Life Sciences, said, “Seven years on, the Zayed Centre for Research reflects the lasting impact of Her Highness Sheikha Fatima bint Mubarak’s extensive support. The Centre’s progress demonstrates how investment in scientific research and innovation can turn scientific discoveries into better outcomes for children diagnosed with rare and complex diseases worldwide.”
She commended “the dedicated teams at the Zayed Centre for Research and Great Ormond Street Hospital for their continued commitment and achievements.”
The Zayed Centre 2026 Annual Report highlights its most recent research breakthroughs and treatment innovations led by 46 principal investigators and their research teams, who together contributed to 61 peer-reviewed scientific publications in the past year. During the 2025/26 financial year, the Centre launched 23 new research projects, including 11 focused on gene and cell therapies.
Among the notable developments are advances in gene therapy for rare genetic conditions, including the Centre's contribution to manufacturing a pioneering treatment for Hunter syndrome, administered to a three-year-old patient as part of a world-first clinical trial. Other achievements include advances in regenerative medicine and innovative approaches to treating childhood cancers.
To mark its seventh anniversary, the Centre is highlighting the work of seven leading researchers whose contributions span neurological disorders, regenerative medicine, gene therapy and innovative cancer treatments.
The work of Professor Manju Kurian, a Consultant Paediatric Neurologist at GOSH and Professor of Neurogenetics at UCL identifies the genetic causes of childhood neurological disorders, including movement disorders and neurodegeneration.
Her work focuses on understanding how genetic changes affect brain development, and developing precision therapies, including innovative gene therapy approaches. By combining genetic research with clinical expertise, her team aims to improve diagnosis and treatment options for children affected by complex neurological conditions.
A surgeon, scientist and pioneer of regenerative medicine, Professor Paolo De Coppi has spent his career pushing the boundaries of what medicine can achieve for children born with complex conditions. As Consultant Paediatric Surgeon at GOSH and leader of regenerative medicine research at UCL Great Ormond Street Institute of Child Health, his work spans stem cells, tissue engineering and innovative surgical techniques.
From developing new approaches to repairing damaged tissues, even before birth, to helping pioneer tissue-engineered solutions for congenital anomalies, Professor De Coppi's research aims to give children treatment options that previously did not exist. His work exemplifies the Zayed Centre's commitment to turning breakthrough science into real-world patient benefit. He is co-lead of the Tissue Engineering and Regenerative Medicine theme of the NIHR GOSH Biomedical Research Centre.
Dr. Amy McTague, Consultant Paediatric Neurologist and researcher, is working to improve the diagnosis and treatment of severe childhood epilepsy through advances in genomic research.
Her work includes the Gene-STEPS study, which has demonstrated how rapid genome sequencing can help identify genetic causes of epilepsy within weeks rather than months or years. She is also developing stem-cell models to support research into future therapies for genetic epilepsies.
Professor Francesco Muntoni, Director of the Dubowitz Neuromuscular Centre, has dedicated his career to advancing research into rare neuromuscular conditions, including Duchenne muscular dystrophy and spinal muscular atrophy (SMA).
His work has bridged the gap between genetic discovery and treatment, helping to lay the foundations for therapies that are transforming outcomes for children with previously untreatable conditions. For families affected by SMA and other neuromuscular disorders, the progress achieved over recent years, including recent announcements to included SMA in UK newborn screening, is a testament to decades of dedicated translational research.
Few areas of medicine are advancing as rapidly as cell and gene therapy, and Professor Waseem Qasim has been at the centre of that revolution. His research focuses on harnessing and engineering immune cells to treat children with cancer.
Professor Qasim led pioneering work in genome-edited CAR-T cell therapies, including world-first treatments for children with otherwise untreatable leukaemia. His team's innovations are helping create "off-the-shelf" cellular therapies that could make life-saving treatments available to more children around the world.
Professor Sara Ghorashian, Consultant Paediatric Haematologist and clinician scientist, is advancing the development of cellular immunotherapies for children with difficult-to-treat blood cancers.
Her research focuses on improving the safety and effectiveness of CAR-T cell therapies and translating laboratory discoveries into clinical treatments. She is also leading clinical trials, exploring new treatment approaches for children with aggressive blood cancers.
For children born with severe immune disorders, gene therapy can offer the possibility of a cure rather than a lifetime of treatment. Professor Claire Booth, Consultant Paediatric Immunologist and Professor of Gene Therapy and Paediatric Immunology, has helped establish GOSH as a global leader in this field.
Leading multiple gene therapy clinical trials, Professor Booth works to develop and deliver innovative treatments for children with rare immune deficiencies and other inherited disorders. Her research spans everything from laboratory development to first-in-human clinical studies, ensuring discoveries reach patients as quickly and safely as possible.
Dr. Kiki Syrad, Director of Research and Innovation at Great Ormond Street Hospital, said, “Seven years ago, the Zayed Centre for Research was established with a bold ambition: to bring together scientists, clinicians, patients and families to accelerate discoveries for children with rare and complex conditions. Today, it stands as a world-leading centre of excellence where groundbreaking research is improving diagnosis, pioneering new treatments and giving hope to families around the world. The seven researchers featured here represent just a fraction of the extraordinary talent, collaboration and determination that make the ZCR so special. As we celebrate this anniversary, we also look ahead to the next generation of discoveries that will transform children's lives.”
The Centre is a partnership between Great Ormond Street Hospital (GOSH), University College London (UCL) and Great Ormond Street Hospital Children's Charity, bringing together scientific and clinical expertise to advance research into rare and complex childhood diseases.