Department of Health – Abu Dhabi and Children’s Hospital of Philadelphia Launch Strategic Collaboration to Advance Gene Editing Capabilities for Rare Diseases

The Department of Health – Abu Dhabi (DoH), the regulator of the healthcare sector in the emirate, and Children’s Hospital of Philadelphia (CHOP) announced the signing of a Memorandum of Understanding (MoU) during the BIO International Convention in San Diego, California, establishing a strategic collaboration focused on advancing gene editing capabilities and accelerating innovation in rare disease care.

Under the agreement, CHOP’s gene therapy pioneers will provide specialised training to Abu Dhabi clinician-scientists in innovative gene editing approaches for rare diseases. The collaboration will focus on building local expertise and further refining specialised skills in base-editing therapies targeting conditions including liver metabolic disorders, genetic clotting disorders, urea cycle disorders and central nervous system conditions, while supporting the long-term development of advanced therapies for diseases prevalent within the region.

H.E. Dr Noura Khamis Al Ghaithi, Undersecretary, Department of Health – Abu Dhabi, said “Abu Dhabi has built one of the world’s most advanced intelligent health systems, bringing together scientific discovery, clinical innovation and healthcare delivery within a single ecosystem. With nearly one million genomes sequenced through the Emirati Genome Programme, we have the foundation to translate genomic insights into life-changing treatments at population scale. Abu Dhabi is building the full gene-editing ecosystem that spans scientific research, clinical translation, regulation and talent development. Through our partnership with Children’s Hospital of Philadelphia, we are strengthening local capabilities and training the next generation of clinician-scientists to help make advanced therapies a reality for children living with rare genetic diseases. Our ambition is to ensure these innovations are developed responsibly and become more accessible to patients in Abu Dhabi, the region and beyond.”

The MoU was signed by Angela Wurster, MSN, CRNP, Vice President of Global Strategy and Business Development at Children’s Hospital of Philadelphia and Mohamed Alameri, PhD, Division Director Genome and Biobank at Department of Health, Abu Dhabi, in the presence of H.E. Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi (DoH).

With genetic conditions representing a significant healthcare challenge, Abu Dhabi has established one of the world's most comprehensive genomics ecosystems through initiatives such as the Emirati Genome Program and the Newborn Genetic Screening. Together, these efforts are enabling earlier identification of inherited conditions, generating population-scale genomic insights, and creating the foundations for more precise interventions and future therapies. 

CHOP is uniquely positioned to advance personalized, liver‑directed gene editing from bench to bedside. This approach can correct a single DNA letter in the liver to treat up to 700 inherited conditions. CHOP recently proved this potential by rapidly creating the world’s first personalized gene‑editing therapy for a baby with a rare metabolic disease, demonstrating that safe, targeted liver gene fixes can be developed quickly. The hospital system is also working closely with the FDA to help reshape gene‑editing trials and broaden clinical applicability.

“Our program will give Abu Dhabi researchers hands‑on, end‑to‑end experience,” said Beverly L. Davidson, PhD, Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and CHOP’s Chief Scientific Strategy Officer, who will help lead the effort. “From designing safe, rigorous clinical trials and caring for patients to evaluating uncertain genetic variants and planning pharmacology and toxicology studies – we will teach them how to translate promising lab discoveries into effective, potentially life-saving therapies.”

The collaboration will support the development of capabilities across the gene editing value chain, from scientific research and clinical translation to talent development and future therapeutic applications. Through Abu Dhabi’s integrated ecosystem, including the Health, Endurance, Longevity and Medicine (HELM) cluster, the emirate is creating an environment where scientific discovery, validation and implementation can happen within the same ecosystem.