Zayed Centre for Research: Seven Leading Experts Drive Breakthroughs in Rare Disease Treatment

While most surgeries focus on a single patient, some procedures, like those conducted in vitro, involve collaboration. Professor Paolo De Coppi, based at London's Zayed Centre for Research into Rare Disease in Children (ZCR), exemplifies this. ZCR is a dedicated facility focused on genetic and regenerative breakthroughs for childhood conditions. Prof De Coppi is developing precision implants, including mini-organs that can be implanted into children suffering from various medical conditions, potentially preventing life-threatening complications before or shortly after birth.

The development of these mini-organs for implantation is a collaborative effort, drawing on the diverse expertise within ZCR. "We have in ZCR the expertise that goes from the stem-cell biologist to the ability to sequence genes," Prof De Coppi stated in an interview with The National. He further described the lab as a synergy between surgeons with innovative ideas, cell culturing specialists, and bioengineers. "Engineers help us create modelling and create the special flasks to grow the cells in a special way," he added. An innovative example highlighted is the oesophagus, where advanced techniques like spatial transcriptomics are employed. "The technology is now such that you look very deep at the gene expression in the cell and put this in the context of the tissue. Cells do certain things in context and understanding that context gives us functionality and a degree of safety," he explained.

Prof De Coppi is one of seven leading researchers at ZCR spearheading advancements in gene therapy, regenerative medicine, rare disease diagnosis, and innovative treatments. ZCR, which opened its doors to patients in October 2019, integrates pioneering research with clinical care. In partnership with the renowned Great Ormond Street Hospital, ZCR is committed to manufacturing gene and cell therapies to offer new treatments and cures for critically ill children from across the UK and internationally. The purpose-built facility, located adjacent to the UCL Great Ormond Street Institute of Child Health, enables hundreds of researchers and clinicians to collaborate closely. Prof De Coppi noted that the centre's design allows families to witness the scientists at work, fostering transparency and engagement: "We invite the family and the patient themselves … to join us with the researchers so they see what's going on and they can raise their concerns at any stage." Earlier this year, Prof De Coppi was honored as an honorary fellow by the American College of Surgeons, an accolade he attributes to the entire team: "It is really the recognition of the whole group and I feel very humble and privileged."

The centre's progress is further accelerated by AI-driven analysis of laboratory developments, leading to faster diagnoses, treatments, and cures for rare and complex diseases. The article also highlights other key researchers at ZCR: Prof Claire Booth, a paediatric immunologist leading gene therapy clinical trials; Prof Waseem Qasim, a cell and gene therapy research leader; Sara Ghorashian, a consultant paediatric haematologist; Prof Francesco Muntoni, a specialist in neuromuscular diseases; Dr Amy McTague, a consultant paediatric neurologist focusing on early-onset epilepsy; and Prof Manju Kurian, a consultant paediatric neurologist researching childhood neurological disorders. Each of these experts contributes significantly to the centre's mission.

Since its opening, ZCR has provided care for over 13,300 children in its Falcon Outpatients suite. This achievement was made possible by a significant £60 million ($80.7 million) gift in 2014 from Sheikha Fatima bint Mubarak, Mother of the Nation, alongside major contributions from Research England, The Wolfson Foundation, John Connolly & Odile Griffith, and the Mead Family Foundation. ZCR operates as a partnership between Great Ormond Street Hospital, UCL, and Great Ormond Street Hospital Children’s Charity.