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Sidra Medicine is building an integrated institution that combines paediatric care with advanced research under one roof, a model its chief research officer described as novel for the region and inspired by leading children’s hospitals worldwide. Prof Khalid Fakhro said the approach aims to close gaps in the care chain by starting with precise diagnosis and advancing toward personalised treatments. Sidra Medicine has invested heavily in these capabilities since its founding as a member of Qatar Foundation, according to Fakhro’s remarks. The strategy aligns with national goals to establish Qatar as a centre for precision medicine.
Fakhro noted that Qatar became one of the first countries in the region to implement whole genome sequencing at scale to improve diagnosis of rare paediatric conditions. Sidra Medicine’s research portfolio focuses on precision health applications for rare diseases, paediatric leukaemia, mitochondrial disorders and autism, a Sidra Medicine overview stated. The hospital’s facilities support multi-omics technologies including transcriptomics, proteomics and epigenomics to drive these efforts. Such infrastructure has enabled the development of targeted interventions tailored to local genetic profiles.
The institution currently maintains five active clinical trials, with plans to expand this programme significantly as regulatory frameworks evolve across the region, Fakhro indicated. Sidra Medicine has secured a one-million-dollar grant from the Juvenile Diabetes Research Foundation to support type 1 diabetes studies that will feed into a national paediatric diabetes registry. These trials form part of a broader push to position Qatar as a test bed for innovative therapies before scaling them regionally. Fakhro emphasised the need for advocacy to update regulations and facilitate faster approvals for such research.
Qatar offers unique advantages as a potential hub-and-spoke centre for Middle East paediatric research due to its strategic location and infrastructure, according to the chief research officer. Fakhro highlighted how the country’s role as a global logistics and travel hub facilitates the movement of patients, specialists and biological materials essential for advanced therapies. This connectivity could allow Sidra Medicine to serve as an entry point for international partners seeking to address unmet needs in regional paediatric care. The model envisions Qatar coordinating expertise and resources that radiate to neighbouring countries.
Sidra Medicine signed a memorandum of understanding with Great Ormond Street Hospital in May 2025 to collaborate on rare disease treatments, gene therapies and clinical trial design, the hospital announced at the time. The partnership leverages Qatar’s unique Arab genetic datasets alongside the London institution’s expertise in secure data environments and personalised medicine. Additional milestones include the January 2025 launch of a dedicated Gene Therapy Center and the successful administration of sepiapterin for phenylketonuria, making Qatar the third country globally to approve this treatment through regulated pathways, according to a Sidra Medicine statement. These steps demonstrate concrete progress toward the hub vision.
In paediatric oncology, Sidra Medicine operates a translational research programme that collects samples from roughly half of all young cancer patients in Qatar each year, with particular focus on acute lymphoblastic leukaemia and central nervous system tumours. The programme collaborates with international groups including the Children’s Oncology Group and the International Society of Paediatric Oncology to import global best practices. Sidra Medicine has also established a paediatric cancer biorepository to accelerate development of precision therapies, its research documentation shows. Officials expect these initiatives to improve survival rates and quality of life for children across the region.
