February 21, 2026-Emirates Health Services (EHS) has unveiled two advanced gene therapy initiatives aimed at treating rare neurological and genetic disorders in children — marking a strategic shift from symptom management to targeting the genetic root causes of disease.
The first program focuses on monogenic autism and epilepsy-linked conditions, including rare neurodegenerative disorders such as Beta-Propeller Protein-Associated Neurodegeneration (BPAN). The approach will use enhanced gene delivery systems designed to target specific brain cells, with the goal of improving precision, reducing required dosage, and enhancing safety. The platform could also create pathways for treating other inherited pediatric neurological diseases.
The initiative is being developed in collaboration with Children’s Hospital of Philadelphia, combining global research expertise with UAE-based clinical development to expand treatment access for affected children.
A second project will develop advanced genetic interventions for broader neurodegenerative conditions, including gene repair strategies, disease modeling, and cell-based therapies. Researchers will also identify brain biomarkers to better monitor disease progression and treatment response.
Artificial intelligence and data analytics will support the program by leveraging insights from the Emirati Genome Programme to identify at-risk children earlier and enable precision intervention.
EHS leadership described the initiative as a long-term investment in innovation-driven healthcare, aiming to build domestic capabilities in advanced biotechnology while improving quality of life for families affected by rare genetic disease.
