Six Years of Discovery and Impact at the Zayed Centre for Research
This year marks a major milestone for the Zayed Centre for Research into Rare Disease in Children (ZCR) as we celebrate six years of world-leading science, innovation and hope.
Since opening in 2019, the centre has become a global hub for pioneering research and advanced therapies, bringing together clinicians, scientists and manufacturing experts under one roof. Their shared mission is to transform the lives of children with the world’s rarest and most complex conditions.
GOSH Charity recently released its Impact Report 2024/25, which highlights another exceptional year of breakthroughs and innovations that are shaping the future of children’s healthcare in the UK, the UAE and around the world.
Below is a look back at some of the year’s most significant achievements.
2024 to 2025 Impact Timeline
April 2024: Leading a new model for global access to gene therapy
In a UK first, Great Ormond Street Hospital (GOSH), supported by GOSH Charity and LifeArc, announced plans to hold the licence for a proven gene therapy.
Led by Professor Claire Booth at the ZCR, the pilot project aims to make life-changing treatments for rare diseases available to more children worldwide.
May 2024: ZCR expertise recognised on the TIME100 Health list
Professor Paolo De Coppi, renowned for his work in regenerative and prenatal medicine, was named to the TIME100 Health list. His contributions, from foetal surgery for spina bifida to developing diagnostic organoids grown from amniotic fluid, demonstrate the ZCR’s commitment to turning scientific discovery into clinical impact.
July 2024: Advancing understanding of childhood heart disease
For the first time, ZCR researchers applied Hierarchical Phase-Contrast Tomography (HiP-CT) to study congenital heart defects.
Led by Professor Andrew Cook, this work is offering new insight into how complex cardiac abnormalities develop and how they might be treated more precisely in the future.
October 2024: Launch of a world-first CAR T-cell therapy trial
A major highlight of the year was the launch of a £2.4 million GOSH Charity funded clinical trial testing a new CAR T-cell therapy for aggressive childhood blood cancers.
Led by Dr Sara Ghorashian, it is the first study to run adult and paediatric trials in parallel. This approach helps ensure that children have faster access to promising treatments.
February 2025: International collaboration speeds up epilepsy diagnoses
Within the International Precision Child Health Partnership, Dr Amy McTague and colleagues are using rapid genome sequencing to diagnose children with severe, unexplained epilepsy.
This method helps clinicians begin effective treatment sooner and improves outcomes for families.
April 2025: New partnership supports the next generation of CAR T therapies
A new collaboration between the ZCR and ViroCell Biologics is helping to address the global shortage of viral vectors, which are essential for many gene and cell therapies.
These vectors are now being manufactured on-site at the ZCR’s Gene and Cell Therapy Facility, the largest academic unit of its kind in the UK. This is accelerating the development of future CAR T-cell treatments.
Meet Gunreet: The youngest child to receive pioneering AADC gene therapy
The GOSH Charity Impact Report also highlights the inspiring story of Gunreet, who in February 2024 became the youngest NHS patient to receive a groundbreaking gene therapy for AADC deficiency. This rare neurological condition affects movement, blood pressure and heart rate.
Since receiving treatment at GOSH, her progress has been remarkable.
The Zayed Centre for Research was established thanks to a visionary £60 million gift from Her Highness Sheikha Fatima bint Mubarak, wife of the late Sheikh Zayed bin Sultan Al Nahyan.
We also extend our deepest thanks to Research England, the Wolfson Foundation, John Connolly and Odile Griffith, the Mead Family Foundation, and the Rachel Charitable Trust. Their generosity continues to drive discoveries that are transforming young lives every day.
