GOSH celebrates second anniversary of Zayed Centre for Research into Rare Disease in Children

In addition to facilitating more than 105,000 patient visits to Falcon Outpatients since it opened in 2019, the Centre has also been involved in several globally significant research developments, hosted some very important visitors and is on track to become a central hub for vector manufacture following the announcement of a new partnership.

The Zayed Centre for Research opened shortly before the COVID-19 pandemic struck and, due to its specialist facilities, it was soon playing an instrumental role in the world’s first human challenge study of COVID-19. The study aims to establish the smallest amount of virus needed to cause infection, giving doctors greater understanding of COVID-19 and helping to support the pandemic response by aiding vaccine and treatment development.

It has also been an exciting time for gene therapy. July 2021 marked 20 years since the first patient was treated with gene therapy at GOSH and much of the pioneering gene therapy research at the hospital now takes place at the Zayed Centre for Research. Among those leading the clinical trials at the Centre are Professor Claire Booth who recently celebrated the successful results of a stem cell gene therapy clinical trial for a form of severe combined immunodeficiency called ADA-SCID, [MS2] in a joint study with UCLA. Of the 50 patients treated in the trial, 48 are no longer showing symptoms of the disease.

Meanwhile Professor Manju Kurian, who discovered the brain disorder dopamine transporter deficiency syndrome (DTDS) in 2009, has helped develop a potential gene therapy cure for the disease. Professor Kurian said: “Being in the Zayed Centre has been absolutely life-transforming for both me and my researchers. We answer the questions that families ask us: why has my child got this condition, how has this happened and what can you do to make my child’s life better? And being in the Zayed Centre has allowed us to answer them. We have the most amazing laboratory space, we have state-of-the-art tissue culture facilities and we are in a milieu of other researchers who have the same goal, propelling us forward for these children.”

GOSH’s credentials as a global leader in gene therapy have also led to a new partnership with ViroCell Biologics, a global expert in vector manufacture, which going forward will see the Zayed Centre for Research become a central hub for the production of vectors - the gene therapy ‘delivery system’ that takes the working gene into living cells.

In September, His Highness Sheikh Mohamed bin Zayed, Crown Prince of Abu Dhabi, visited the Centre along with the UK's Secretary of State for Health and Social Care and Sajid Javid, and the Secretary of State for Education, Nadhim Zahawi. The group toured the facility, meeting researchers and clinicians who briefed them on how they work to diagnose, treat and cure rare diseases in children.

And in November 2021, a team from the Abu Dhabi Stem Cell Centre met with researchers and clinicians working on a number of different disease groups to hear about their work and consider areas for future collaboration.

The Zayed Centre for Research was made possible thanks to a transformative £60 million gift from Her Highness Sheikha Fatima bint Mubarak, wife of the late Sheikh Zayed bin Sultan Al Nahyan, founding father of the United Arab Emirates, in 2014. We are also grateful to Research England, The Wolfson Foundation, John Connolly & Odile Griffith and the Mead Family Foundation whose generous support contributed to the creation of the Zayed Centre for Research. The Centre is a partnership between Great Ormond Street Hospital, UCL and Great Ormond Street Hospital Children’s Charity.