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

Collaboration combines CHOP's world-leading gene editing expertise with Abu Dhabi's population-scale genomics ecosystem to accelerate the future of precision medicine

ABU DHABI, UAE, June 24, 2026 /PRNewswire/ -- 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.

About the Department of Health Abu Dhabi (DoH)

The Department of Health – Abu Dhabi is the regulator of the healthcare sector in the Emirate of Abu Dhabi. Committed to ensuring excellence in healthcare, DoH monitors the health status of the population while shaping the regulatory framework for the health system. This includes inspecting against regulations, enforcing standards and encouraging the adoption of world-class best practices and performance targets by all healthcare providers in the Emirate. Abu Dhabi remains dedicated to shaping the future of healthcare by establishing an intelligent and sustainable ecosystem that prioritises health, longevity and quality of life for all community members.  Leveraging state-of-the-art infrastructure and cutting-edge capabilities, DoH continues to develop and implement initiatives and programmes in line with its strategic focus areas, Healthy Population, Best-in-Class Care and Resilience and Innovation. These efforts reflect DoH's unwavering commitment to predicting, preventing and personalising healthcare for every individual.

For further information on DoH, visit https://www.doh.gov.ae/ and follow on X, Instagram, Facebook, LinkedIn and YouTube.

About Children's Hospital of Philadelphia:  

A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania, New Jersey, and Delaware. CHOP also operates the Middleman Family Pavilion and its dedicated pediatric emergency department in King of Prussia, the Behavioral Health and Crisis Center (including a 24/7 Crisis Response Center) and the Center for Advanced Behavioral Healthcare, a mental health outpatient facility. Its unique family-centered care and public service programs have brought Children's Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit https://www.chop.edu.

Contact: Jennifer Lee
Children's Hospital of Philadelphia
(267) 426-6084
[email protected]

SOURCE Children's Hospital of Philadelphia